Clinical trial • Phase II/III • Oncology|Immunology

ELENESTINIB PHOSPHATE for Indolent systemic mastocytosis|Smoldering systemic mastocytosis

Phase II/III trial of ELENESTINIB PHOSPHATE for Indolent systemic mastocytosis|Smoldering systemic mastocytosis.

Overview

Trial Therapeutic Area
Oncology|Immunology
Trial Disease
Indolent systemic mastocytosis|Smoldering systemic mastocytosis
Trial Stage
Phase II/III
Drug Modality
Small molecule
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
08-10-2024
First CTIS Authorization Date
05-11-2024

Trial design

Randomised, open-label, placebo tablets + symptom-directed therapy (matching placebo control); dose/schedule not specified for placebo, crossover, adaptive Phase II/III trial across 50 sites in Portugal, France, Denmark and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo tablets + symptom-directed therapy (matching placebo control); dose/schedule not specified for placebo
Adaptive
True, includes dose-finding/dose-escalation in Part 1 to determine recommended dose(s) of elenestinib, crossover of placebo patients to active RD after Week 12, PK groups enrolled to evaluate PK; select personnel unblinded for safety/IDMC. No detailed stopping rules provided in available text.
Crossover
Yes
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
187
Trial Duration For Participant
1825

Eligibility

Recruits 187 paediatric patients.

Vulnerable Population
Vulnerable populations are selected. If a patient is under 18 years, a parent or legal guardian provides signed informed consent when necessary; pediatric assent documents ("Pediatric Assent_16 to 17 Years") and Parental ICFs are provided in some countries (e.g., Belgium, Portugal).

Inclusion criteria

  • {"criterion_text":"- All patients. 1. Patient must be ≥ 18 years of age at the time of signing the informed consent/assent.\n- Patients in Part K Previously Treated With Approved Selective KIT Inhibitors: 12. Patient has a centrally confirmed diagnosis of ISM. In consultation with the Sponsor, archival biopsy may be used if completed within the past 12 months and there is no evidence of progressive disease.\n- 13. Patients with 14-day average TSS obtained no earlier than 15 days after the discontinuation of the prior approved selective KIT inhibitor may be enrolled.\n- Patients with ISM in Part 1, 2 and PK groups. 4. Patient has confirmed diagnosis of ISM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria. Archival biopsy may be used if completed within the past 12 months.\n- 2. Patient must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. With ISM in Part 1 and Part 2\n- Patients with ISM in Part 1. 3. Patient must have moderate-to-severe symptoms based on minimum mean total symptom score (TSS) of the ISM Symptom Assessment Form (ISM-SAF) over the 14-day eligibility screening period. With ISM in Part 1, Part 2 and PK groups\n- 5. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigators best clinical judgment, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab.\n- 6. Patients must have SDT for ISM symptom management stabilized without any new or worsening of symptoms and/or AEs for at least 14 days prior to starting 14-day ISM-SAF TSS eligibility period (Part 1 only).\n- Patients in Part 2: 17. Patient has a centrally confirmed diagnosis of ISM. Archival biopsy may be used if completed within the past 12 months.\n- 7. For patients receiving corticosteroids, the dose must be ≤ 20 mg/d prednisone or equivalent, and the dose must be stable for ≥ 14 days before beginning the 14-day eligibility Screening Period for assessment of ISM-SAF for Part 1 or PK.\n- 8. The patient’s ISM symptom-directed therapies (eg, H1 and H2 blockers) must be stable (same dose, no new medications ≥ 14 days before beginning the 14-day eligibility Screening Period for the assessment of ISM-SAF).\n- 15. Treatment with a prior approved selective KIT inhibitor for ISM.\n- 14. Patients must have a washout period of their prior approved selective KIT inhibitor for ISM of at least 28 days prior to the first elenestinib dose.\n- 19. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms as determined by the Investigator’s best clinical judgment, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab.\n- 18. Patient must have moderate to severe symptoms during the eligibility period.\n- 21. If the patient is receiving corticosteroids, the dose must be ≤ 20 mg/day prednisone or equivalent, and the dose must be stable for ≥ 14 days before beginning the 14-day eligibility Screening Period for assessment of ISM-SAF.\n- 22. If on a bone-targeted therapy other than calcium and vitamin D, including hormone replacement therapy, patients must be on a stable dose for least 24 weeks prior to first treatment day unless the administration frequency of the drug is less frequent than once every 24 weeks for which at least 2 consecutive doses must have been administrated to the patient following the schedule.\n- 10. Accrual may be limited to patients who have specific disease manifestations (ie, GI involvement) to better explore the impact of these features on PK.\n- 20. The patient’s ISM symptom-directed therapies (e.g., H1 and H2 blockers) must be stable (same dose, no new medications ≥ 14 days before beginning the 14-day eligibility Screening Period for the assessment of ISM-SAF).\n- Patients with SSM in Part S: 11.Patient has confirmed diagnosis of SSM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria . No archival BM biopsies will be accepted without approval from the Sponsor."}

Exclusion criteria

  • {"criterion_text":"- Patient has been diagnosed with any of the following WHO SM sub-classifications: cutaneous mastocytosis only, SM-AHN, ASM, MCL, Mast cell sarcoma.\n- Patient has been diagnosed with another myeloproliferative disorder.\n- Patient has organ damage attributable to SM.\n- Patient has a QT interval corrected using Fridericia's formula (QTcF) > 470 msec (for females) or > 450 msec (for males).\n- Patient has clinically significant, uncontrolled, cardiovascular disease.\n- Patient has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.\n- Time since any cytoreductive therapy including mastinib and midostaurin should be at least 5 half-lives or 14 days (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy < 28 days or 5 half-lives of the drug (whichever is longer), before beginning the screening assessments. For omalizumab, washout is not necessary, and patients can continue on omalizumab therapy.\n- Patient has received radiotherapy or PUVA therapy < 14 days before beginning the screening assessments."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Part 1: Safety and tolerability as determined by adverse events, serious treatment-emergent adverse events, and changes in safety laboratory parameters, vital signs, and ECG evaluations PK and PD data The mean change in ISM-SAF TSS from Baseline at Week 13.","definition_or_measurement_approach":"Safety and tolerability assessed by adverse events (AEs), serious AEs, changes in safety laboratory parameters, vital signs, ECG evaluations, PK and PD data; efficacy measured as mean change in ISM-SAF Total Symptom Score (TSS) from Baseline at Week 13."}
  • {"endpoint_text":"- Part 2: Mean change in ISM-SAF TSS from Baseline to after 48 weeks of treatment (Week 49), as compared to placebo","definition_or_measurement_approach":"Mean change in ISM-SAF TSS from Baseline to Week 49 (after 48 weeks treatment) compared between elenestinib + SDT and placebo + SDT."}
  • {"endpoint_text":"- Part 3: 1. Safety and tolerability determined by AEs, SAEs, and changes in safety laboratory parameters, vital signs, and ECG evaluations. 2. Change in ISM-SAF TSS from elenestinib Baseline (T the last available observation prior to the first dose of elenestinib)","definition_or_measurement_approach":"Safety/tolerability by AEs/SAEs and changes in lab parameters, vitals, ECG; ISM-SAF TSS change measured from elenestinib Baseline (last observation prior to first dose)."}

Secondary endpoints

  • {"endpoint_text":"- Part 1: The mean change in the following measures from Baseline at Week 13: • Serum tryptase; • KIT D816V allele fraction in blood; • BM mast cells. The mean change in ISM-SAF individual symptom scores from Baseline at 12 weeks of treatment (Week 13). The time to achieve a 30% reduction in ISM-SAF TSS, ISM-SAF GSS, ISM-SAF SSS, and ISM-SAF Neurocognitive Symptom Cluster Score from randomization among patients who achieve such a reduction on or before 12 weeks of treatment (Week 13).\"","definition_or_measurement_approach":"Measures include serum tryptase, PB KIT D816V allele fraction, bone marrow mast cell counts; ISM-SAF individual symptom scores; time to 30% reduction in specified ISM-SAF scores assessed up to Week 13."}
  • {"endpoint_text":"- Part 2: 1. Proportion of patients achieving a normalized tryptase after 48weeks of treatment (Week 49) as compared to placebo","definition_or_measurement_approach":"Proportion of patients achieving normalized serum tryptase at Week 49 (after 48 weeks) compared between active and placebo arms."}
  • {"endpoint_text":"- Part 2: 4. Mean percent change in lumbar BMD from Baseline to after 48 weeks of treatment (Week 49), compared to placebo among patients with baseline osteopenia or osteoporosis","definition_or_measurement_approach":"Mean percent change in lumbar bone mineral density (BMD) from Baseline to Week 49 in patients with baseline osteopenia/osteoporosis compared between arms; measured by DXA."}
  • {"endpoint_text":"- Part 2: 5. Mean change in the annualized rate of anaphylaxis events between the Screening Period and Weeks 25 to 48 of treatment compared to placebo.","definition_or_measurement_approach":"Mean change in annualized rate of anaphylaxis events comparing Screening Period to Weeks 25–48 between treatment arms."}
  • {"endpoint_text":"- Part 2: 6. Mean change in QoL score from Baseline to after 48 weeks of treatment (week 49) as compared to placebo.","definition_or_measurement_approach":"Mean change in quality-of-life (QoL) score from Baseline to Week 49 compared between active treatment and placebo."}
  • {"endpoint_text":"- Part 3: 1. Proportion of patients achieving symptom control as defined by achieving mild symptoms 2. Change in ISM-SAF domain scores including the ISM-SAF GSS, ISM-SAF SSS, and ISM-SAF NSS 3. Change in BMD 4. Proportion of patients achieving a normalized tryptase 5. Change in the annualized rate of anaphylaxis events","definition_or_measurement_approach":"Proportion achieving symptom control (mild symptoms); changes in ISM-SAF domain scores (GSS, SSS, NSS); BMD change; proportion with normalized tryptase; annualized anaphylaxis event rate change."}
  • {"endpoint_text":"- Part 2 and Part 3 Shared: 1. Change in the following measures: Serum tryptase; KIT D816V allele fraction in blood, and BM mast cells. 2. Proportion of patients achieving controlled disease 3. Change in skin lesions as assessed by the fractional body surface area of the most affected skin area 4. Change in the number of concomitant medications identified as SDT 5. Change in ISM-SAF Individual Symptom Scores 6. Change in ISM-SAF Lead (most severe) Symptom Score","definition_or_measurement_approach":"Shared endpoints include changes in serum tryptase, PB KIT D816V VAF, BM mast cells; proportion with controlled disease; change in skin lesion BSA; change in number of SDT medications; ISM-SAF individual and lead symptom score changes."}
  • {"endpoint_text":"- Part 2 and Part 3 Shared (translations continued)","definition_or_measurement_approach":"Additional specification of shared endpoints: change in concomitant SDT medications and individual/lead ISM-SAF symptom score changes."}
  • {"endpoint_text":"- Part 2: 2. Proportion of patients achieving an undetectable level or ≥ 50% reduction in KIT D816V-VAF Baseline to after 48 weeks of treatment (Week 49) as compared to placebo among patients with detectable mutation at Baseline","definition_or_measurement_approach":"Proportion achieving undetectable KIT D816V VAF or ≥50% reduction from Baseline to Week 49 among patients with detectable baseline mutation compared between arms."}
  • {"endpoint_text":"- Part 2: 3. Proportion of patients symptom control as defined by achieving mild symptoms after 48 weeks of treatment as compared to placebo.","definition_or_measurement_approach":"Proportion of patients achieving symptom control (mild symptoms) at Week 49 compared between active and placebo arms."}

Recruitment

Planned Sample Size
187
Recruitment Window Months
137
Consent Approach
Adults (≥18) provide informed consent. If patient is under 18, a parent or legal guardian provides signed informed consent when necessary; pediatric assent documents are provided for 16- to 17-year-olds ("Pediatric Assent_16 to 17 Years" in Belgium). Multiple country-specific ICFs and parental ICFs are provided (documents in English, Portuguese, French, German, Dutch, Spanish, Italian, Greek, Polish, Swedish, Norwegian, Czech and other local languages are available per country-specific documentation).

Geography

Total Number Of Sites
50
Total Number Of Participants
379

Portugal

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
08-09-2025
Processing Time Days
314
Number Of Sites
4
Number Of Participants
20

Sites

Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Hematology
Principal Investigator Name
Renata Cabral
Contact Person Name
Renata Cabral
Site Name
Unidade Local de Saude de Sao Joao E.P.E.
Department Name
Immunoallergology Department
Principal Investigator Name
Leonor Leão
Principal Investigator Email
leonor.leao@ulssjoao.min-saude.pt
Contact Person Name
Leonor Leão
Site Name
Unidade Local De Saude De Matosinhos E.P.E.
Department Name
Immunoallergology Department
Principal Investigator Name
Tiago Rama
Principal Investigator Email
tiago.rama@ulsm.min-saude.pt
Contact Person Name
Tiago Rama
Contact Person Email
tiago.rama@ulsm.min-saude.pt
Site Name
Unidade Local De Saude De Sao Jose E.P.E.
Department Name
Hematology
Principal Investigator Name
Patricia Ribeiro
Principal Investigator Email
patricia.m.ribeiro@ulssjose.min-saude.pt
Contact Person Name
Patricia Ribeiro

France

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
08-09-2025
Processing Time Days
314
Number Of Sites
10
Number Of Participants
127

Sites

Site Name
Centre Hospitalier Universitaire Grenoble Alpes
Department Name
Département de Médecine interne
Principal Investigator Name
Laurence Bouillet
Principal Investigator Email
lbouillet@chu-grenoble.fr
Contact Person Name
Laurence Bouillet
Contact Person Email
lbouillet@chu-grenoble.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Service de médecine interne 2
Principal Investigator Name
Stephane Barete
Principal Investigator Email
stephane.barete@gmail.com
Contact Person Name
Stephane Barete
Contact Person Email
stephane.barete@gmail.com
Site Name
Centre Hospitalier Et Universitaire De Limoges
Department Name
Service d'Hématologie Clinique et Thérapie Cellulaire
Principal Investigator Name
Marie-Pierre Gourin
Principal Investigator Email
marie-pierre.gourin@chu-limoges.fr
Contact Person Name
Marie-Pierre Gourin
Site Name
Centre Hospitalier Universitaire De Nantes
Department Name
Service de Médecine interne
Principal Investigator Name
Antoine Neel
Principal Investigator Email
antoine.neel@chu-nantes.fr
Contact Person Name
Antoine Neel
Contact Person Email
antoine.neel@chu-nantes.fr
Site Name
Centre Hospitalier Universitaire Amiens Picardie
Department Name
Département d'hématologie clinique et thérapie cellulaire
Principal Investigator Name
Clement Gourguechon
Principal Investigator Email
gourguechon.clement@chu-amiens.fr
Contact Person Name
Clement Gourguechon
Site Name
Hopital Necker Enfants Malades
Department Name
Département d'hématologie adultes
Principal Investigator Name
Olivier Hermine
Principal Investigator Email
olivier.hermine@nck.aphp.fr
Contact Person Name
Olivier Hermine
Contact Person Email
olivier.hermine@nck.aphp.fr
Site Name
CHU Besancon
Department Name
Département de myologie
Principal Investigator Name
Florence CASTELAIN
Principal Investigator Email
fcastelain@chu-besancon.fr
Contact Person Name
Florence CASTELAIN
Contact Person Email
fcastelain@chu-besancon.fr
Site Name
Centre Hospitalier Universitaire De Poitiers
Department Name
Département de Dermatologie
Principal Investigator Name
Ewa Wierzbicka Hainaut
Principal Investigator Email
ewa.hainaut@chu-poitiers.fr
Contact Person Name
Ewa Wierzbicka Hainaut
Contact Person Email
ewa.hainaut@chu-poitiers.fr
Site Name
Centre Hospitalier Universitaire De Caen Normandie
Department Name
Institut d’Hématologie de Basse Normandie
Principal Investigator Name
Gandhi Laurent DAMAJ
Principal Investigator Email
damaj.gl@chu-caen.fr
Contact Person Name
Gandhi Laurent DAMAJ
Contact Person Email
damaj.gl@chu-caen.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
CEREMAST, Service de Dermatologie
Principal Investigator Name
Cristina LIVIDEANU
Principal Investigator Email
livideanu.cl@chu-caen.fr
Contact Person Name
Cristina LIVIDEANU
Contact Person Email
livideanu.cl@chu-caen.fr

Denmark

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
04-09-2025
Processing Time Days
310
Number Of Sites
1
Number Of Participants
7

Sites

Site Name
Odense University Hospital
Department Name
Department of Dermatology and Allergy Center, Odense University Hospital
Principal Investigator Name
Sigurd Broesby-Olsen
Principal Investigator Email
sigurd.broesby-olsen@rsyd.dk
Contact Person Name
Sigurd Broesby-Olsen
Contact Person Email
sigurd.broesby-olsen@rsyd.dk

Austria

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
05-09-2025
Processing Time Days
311
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Kepler Universitätsklinikum Linz - Med Campus III
Department Name
Hämatologie und Internistische Onkologie
Principal Investigator Name
Clemens Schmitt
Principal Investigator Email
clemens.schmitt@kepleruniklinikum.at
Contact Person Name
Clemens Schmitt

Norway

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
08-09-2025
Processing Time Days
314
Number Of Sites
1
Number Of Participants
12

Sites

Site Name
Oslo University Hospital
Department Name
Oslo Universitetssykehus HF Rikshospitalet
Principal Investigator Name
Ingunn Dybedal
Principal Investigator Email
idybedal@ous-hf.no
Contact Person Name
Ingunn Dybedal
Contact Person Email
idybedal@ous-hf.no

Sweden

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
05-09-2025
Processing Time Days
311
Number Of Sites
2
Number Of Participants
13

Sites

Site Name
Karolinska University Hospital
Department Name
Karolinska universitetssjukhuset-Huddinge,Tema cancer/Studieenheten & cancerstudieenheten
Principal Investigator Name
Cecilia Karlström
Principal Investigator Email
cecilia.karlstrom@regionstockholm.se
Contact Person Name
Cecilia Karlström
Site Name
Uppsala University Hospital
Department Name
Akademiska sjukhuset , Blod- och Tumörsjukdomar, KFUE - Kliniska forsknings- och utvecklingsenheten
Principal Investigator Name
Mattias Mattson
Principal Investigator Email
mattias.mattsson@akademiska.se
Contact Person Name
Mattias Mattson
Contact Person Email
mattias.mattsson@akademiska.se

Spain

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
05-09-2025
Processing Time Days
311
Number Of Sites
3
Number Of Participants
51

Sites

Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Servicio de Alergia
Principal Investigator Name
David Gonzalez de Olano
Principal Investigator Email
dgolano@yahoo.es
Contact Person Name
David Gonzalez de Olano
Contact Person Email
dgolano@yahoo.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Servicio de Oncologia Medica
Principal Investigator Name
Mar Guilarte
Principal Investigator Email
mar.guilarte@vallhebron.cat
Contact Person Name
Mar Guilarte
Contact Person Email
mar.guilarte@vallhebron.cat
Site Name
Hospital Virgen Del Valle
Department Name
Instituto de Mastocitosis de Castilla La Mancha
Principal Investigator Name
Ivan Alvarez-Twose
Principal Investigator Email
ivana@sescam.jccm.es
Contact Person Name
Ivan Alvarez-Twose
Contact Person Email
ivana@sescam.jccm.es

Italy

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
17-09-2025
Processing Time Days
323
Number Of Sites
7
Number Of Participants
29

Sites

Site Name
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania
Department Name
Presidio Ospedaliero Gaspare Rodolico
Principal Investigator Name
Di Raimondo
Principal Investigator Email
diraimon@unict.it
Contact Person Name
Di Raimondo
Contact Person Email
diraimon@unict.it
Site Name
Azienda Ospedaliera Universitaria Integrata Verona
Department Name
Unità Operativa di Allergologia
Principal Investigator Name
Patrizia Bonadonna
Principal Investigator Email
patrizia.bonadonna@aovr.veneto.it
Contact Person Name
Patrizia Bonadonna
Site Name
Careggi University Hospital
Department Name
Dipartimento di Medicina Sperimentale e Clinica
Principal Investigator Name
Francesco Mannelli
Principal Investigator Email
francesco.mannelli@unifi.it
Contact Person Name
Francesco Mannelli
Contact Person Email
francesco.mannelli@unifi.it
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Unità di Ematologia
Principal Investigator Name
Cristina Papayannidis
Principal Investigator Email
cristina.papayannidis@unibo.it
Contact Person Name
Cristina Papayannidis
Contact Person Email
cristina.papayannidis@unibo.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
S.C. Ematologia
Principal Investigator Name
Chiara Elena
Principal Investigator Email
c.elena@smatteo.pv.it
Contact Person Name
Chiara Elena
Contact Person Email
c.elena@smatteo.pv.it
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
Dipartimento Medicina Interna, Centro Emofilia E Trombosi Angelo Bianchi Bonomi
Principal Investigator Name
Bruno Fattizzo
Principal Investigator Email
bruno.fattizzo@policlinico.mi.it
Contact Person Name
Bruno Fattizzo
Site Name
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d'Aragona
Department Name
SSD Immunologia Clinica e Allergologia
Principal Investigator Name
Mssimo Triggiani
Principal Investigator Email
mtriggiani@unisa.it
Contact Person Name
Mssimo Triggiani
Contact Person Email
mtriggiani@unisa.it

Greece

Earliest CTIS Part Ii Submission Date
03-12-2025
Latest Decision Or Authorization Date
13-01-2026
Processing Time Days
41
Number Of Sites
2
Number Of Participants
10

Sites

Site Name
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara
Department Name
Allergy Unit “Dimitrios Kalogeromitros”, 2nd Department of Dermatology and Venereology NKUA
Principal Investigator Name
Michael Makris
Principal Investigator Email
mmakris.allergy@gmail.com
Contact Person Name
Michael Makris
Contact Person Email
mmakris.allergy@gmail.com
Site Name
Geniko Nosokomeio Thessalonikis George Papanikolaou
Department Name
Department of Hematology and Bone Marrow Transplantation Unit
Principal Investigator Name
Ioanna Sakellari
Principal Investigator Email
ioannamarilena@gmail.com
Contact Person Name
Ioanna Sakellari
Contact Person Email
ioannamarilena@gmail.com

Czechia

Earliest CTIS Part Ii Submission Date
27-11-2025
Latest Decision Or Authorization Date
19-01-2026
Processing Time Days
53
Number Of Sites
2
Number Of Participants
10

Sites

Site Name
Fakultni Nemocnice Kralovske Vinohrady
Department Name
Hematologická klinika 3. LF UK v Praze a FNKV
Principal Investigator Name
Tomáš Kozák
Principal Investigator Email
tomas.kozak@fnkv.cz
Contact Person Name
Tomáš Kozák
Contact Person Email
tomas.kozak@fnkv.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Interní hematologická a onkologická klinika
Principal Investigator Name
Michael Doubek
Principal Investigator Email
doubek.michael@fnbrno.cz
Contact Person Name
Michael Doubek
Contact Person Email
doubek.michael@fnbrno.cz

Ireland

Earliest CTIS Part Ii Submission Date
27-11-2025
Latest Decision Or Authorization Date
23-01-2026
Processing Time Days
57
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Cork University Hospital
Department Name
Haematology
Principal Investigator Name
Clodagh Keohane
Principal Investigator Email
ckeohane@muh.ie
Contact Person Name
Clodagh Keohane
Contact Person Email
ckeohane@muh.ie

Poland

Earliest CTIS Part Ii Submission Date
04-12-2025
Latest Decision Or Authorization Date
26-01-2026
Processing Time Days
53
Number Of Sites
2
Number Of Participants
10

Sites

Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Poradnia Hematologiczna/Oddział Kliniczny Hematologii i Chorób Wewnętrznych
Principal Investigator Name
Tomasz Sacha
Principal Investigator Email
sachatom@gmail.com
Contact Person Name
Tomasz Sacha
Contact Person Email
sachatom@gmail.com
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Alergologii
Principal Investigator Name
Marek Niedoszytko
Principal Investigator Email
marek.niedoszytko@gumed.edu.pl
Contact Person Name
Marek Niedoszytko
Contact Person Email
marek.niedoszytko@gumed.edu.pl

Germany

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
17-11-2025
Processing Time Days
384
Number Of Sites
8
Number Of Participants
54

Sites

Site Name
Universitaetsklinikum Schleswig-Holstein AöR
Department Name
Medizinische Klinik I, Hämatologie/ Onkologie
Principal Investigator Name
Friederike Wortmann
Principal Investigator Email
friederike.wortmann@uksh.de
Contact Person Name
Friederike Wortmann
Contact Person Email
friederike.wortmann@uksh.de
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Institute of Allergology Campus Benjamin Franklin
Principal Investigator Name
Frank Siebenhaar
Principal Investigator Email
frank.siebenhaar@charite.de
Contact Person Name
Frank Siebenhaar
Contact Person Email
frank.siebenhaar@charite.de
Site Name
Universitaetsklinikum Mannheim GmbH
Department Name
III. Medizinische Klinik Hämatologie und Internistische Onkologie
Principal Investigator Name
Juliana Schwaab
Principal Investigator Email
juliana.schwaab@medma.uni-heidelberg.de
Contact Person Name
Juliana Schwaab
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Klinik und Poliklinik für Onkologie, Hämatologie und KMT mit der Abteilung für Pneumologie
Principal Investigator Name
Philippe Schafhausen
Principal Investigator Email
schafhausen@uke.de
Contact Person Name
Philippe Schafhausen
Contact Person Email
schafhausen@uke.de
Site Name
Universitaetsklinikum Aachen AöR
Department Name
Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation
Principal Investigator Name
Jens Panse
Principal Investigator Email
jpanse@ukaachen.de
Contact Person Name
Jens Panse
Contact Person Email
jpanse@ukaachen.de
Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Dermato-Allergologisches Studienzentrum Campus Innenstadt
Principal Investigator Name
Anne-Charlotte Kuna
Principal Investigator Email
AnneCharlotte.Niesert@med.uni-muenchen.de
Contact Person Name
Anne-Charlotte Kuna
Site Name
Universitaetsklinikum Erlangen AöR
Department Name
Hautklinik
Principal Investigator Name
Nicola Wagner
Principal Investigator Email
nicola.wagner@uk-erlangen.de
Contact Person Name
Nicola Wagner
Contact Person Email
nicola.wagner@uk-erlangen.de
Site Name
Klinikum rechts der Isar der TU Muenchen AöR
Department Name
Klinik und Poliklinik für Dermatologie und Allergologie am Biederstein
Principal Investigator Name
Knut Brockow
Principal Investigator Email
knut.brockow@tum.de
Contact Person Name
Knut Brockow
Contact Person Email
knut.brockow@tum.de

Netherlands

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
05-01-2026
Processing Time Days
433
Number Of Sites
3
Number Of Participants
12

Sites

Site Name
Universitair Medisch Centrum Groningen
Department Name
Internal Medicine (Dept of Allergology)
Principal Investigator Name
Hanneke Oude Elberink
Principal Investigator Email
j.n.g.oude.elberink@umcg.nl
Contact Person Name
Hanneke Oude Elberink
Contact Person Email
j.n.g.oude.elberink@umcg.nl
Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Internal Medicine
Principal Investigator Name
Paul van Daele
Principal Investigator Email
p.l.a.vandaele@erasmusmc.nl
Contact Person Name
Paul van Daele
Contact Person Email
p.l.a.vandaele@erasmusmc.nl
Site Name
Academisch Ziekenhuis Maastricht
Department Name
Hematology
Principal Investigator Name
Floor Heubel-Moenen
Principal Investigator Email
floor.moenen@mumc.nl
Contact Person Name
Floor Heubel-Moenen
Contact Person Email
floor.moenen@mumc.nl

Belgium

Earliest CTIS Part Ii Submission Date
29-10-2024
Latest Decision Or Authorization Date
20-02-2026
Processing Time Days
479
Number Of Sites
3
Number Of Participants
16

Sites

Site Name
UZ Leuven
Department Name
Allergy and Clinical Immunology
Principal Investigator Name
Christine Breynaert
Principal Investigator Email
christine.breynaert@uzleuven.be
Contact Person Name
Christine Breynaert
Site Name
Antwerp University Hospital
Department Name
Immunology
Principal Investigator Name
Vito Sabato
Principal Investigator Email
vito.sabato@uza.be
Contact Person Name
Vito Sabato
Contact Person Email
vito.sabato@uza.be
Site Name
Centre hospitalier universitaire de Tivoli Institut medical des Mutualites socialistes
Department Name
Hematology
Principal Investigator Name
Mélanie Vaes
Principal Investigator Email
mvaes@chu-tivoli.be
Contact Person Name
Mélanie Vaes
Contact Person Email
mvaes@chu-tivoli.be

Sponsor

Primary sponsor

Full Name
Blueprint Medicines Corp.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
PPD Development LP
Responsibilities
Multiple operational responsibilities (listed sponsor duties including clinical operations, medical monitoring, data management etc. in sponsor duties array)
Name
ICON Clinical Research Limited
Responsibilities
KIT D816V-related assays & duties
Name
Pharmaceutical Product Development LLC
Responsibilities
Clinical Chemistry and related sponsor duties
Name
Syneos Health Clinique Inc.
Responsibilities
PK Analysis

Third parties

  • {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"Biomarker Analysis","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Biotel Research LLC","duties_or_roles":"CT/MRI Imaging","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Qualitymetric Incorporated LLC","duties_or_roles":"SF-12 QoL Owner","organisation_type":"Pharmaceutical company"}
  • {"country":"Canada","full_name":"Syneos Health Clinique Inc.","duties_or_roles":"PK Analysis","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United Kingdom","full_name":"Phlexglobal Limited","duties_or_roles":"eTMF","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Frontage Laboratories Inc.","duties_or_roles":"PK Analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Mayo Clinic Hospital Rochester","duties_or_roles":"urinary Mastocytosis biomarkers","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"IRT","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"KIT D816V","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"DXA scans","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Canfield Scientific Inc.","duties_or_roles":"Imaging","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Primevigilance USA Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"Coagulation","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"Trilogy Writing & Consulting GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Transperfect Translations International Inc.","duties_or_roles":"QoL Translations","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Bone biomarkers","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"Tryptase Analysis","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Clinical Chemistry","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Mde Services Group Limited","duties_or_roles":"Patient Primary Travel and accomodation vendor","organisation_type":"Non-Pharmaceutical company"}

Investigational products

Investigational Product Name
ELENESTINIB
Active Substance
ELENESTINIB PHOSPHATE
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
MIA (IMP) 20377 (investigational medicinal product)
Orphan Designation
Yes
Frequency
QD (once daily) where specified (e.g., Part K: 75 mg QD)
Maximum Dose
100 mg/day
Investigational Product Name
Placebo tablets
Modality
Other
Combination Treatment
Yes

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