Clinical trial • Phase III • Haematology

Luspatercept for Myelofibrosis | Anemia associated with myeloproliferative neoplasm-associated myelofibrosis

Phase III trial of Luspatercept for Myelofibrosis | Anemia associated with myeloproliferative neoplasm-associated myelofibrosis.

Overview

Trial Therapeutic Area
Haematology
Trial Disease
Myelofibrosis | Anemia associated with myeloproliferative neoplasm-associated myelofibrosis
Trial Stage
Phase III
Drug Modality
Peptide/protein/enzyme | Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
21-12-2023
First CTIS Authorization Date
04-03-2024

Trial design

Randomised, placebo arm: 0.9 % w/v sodium chloride injection (solution for injection) administered subcutaneously as placebo comparator. active arm: luspatercept (reblozyl; product presentations: reblozyl 25 mg and reblozyl 75 mg powder for solution for injection) administered subcutaneously (dose units mg/kg indicated; specific dosing schedule not specified in provided data).-controlled Phase III trial in Hungary, Ireland, Belgium and others.

Randomised
Yes
Comparator
Placebo arm: 0.9 % w/v Sodium Chloride Injection (solution for injection) administered subcutaneously as placebo comparator. Active arm: Luspatercept (Reblozyl; product presentations: Reblozyl 25 mg and Reblozyl 75 mg powder for solution for injection) administered subcutaneously (dose units mg/kg indicated; specific dosing schedule not specified in provided data).
Target Sample Size
74
Trial Duration For Participant
169

Eligibility

Recruits 74 Vulnerable population selected. Only adults (≥18 years) are eligible. Informed consent must be provided by the participant via the Main ICF (country-specific versions available). Specific provisions for females of childbearing potential and male contraception/abstinence requirements are specified in the ICFs. No assent/parental consent procedures (minors are excluded)..

Pregnancy Exclusion
Have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the study, and after end of IP.
Vulnerable Population
Vulnerable population selected. Only adults (≥18 years) are eligible. Informed consent must be provided by the participant via the Main ICF (country-specific versions available). Specific provisions for females of childbearing potential and male contraception/abstinence requirements are specified in the ICFs. No assent/parental consent procedures (minors are excluded).

Inclusion criteria

  • {"criterion_text":"- 1. Subject is ≥18 years of age at the time of signing the informed consent form (ICF).\n- 2. Subject has a diagnosis of primary myelofibrosis (PMF) according to the 2016 World Health Organization (WHO) criteria or diagnosis of postET or post-PV myelofibrosis according to the IWG-MRT 2007 criteria, confirmed by the most recent local pathology report.\n- 3. Subject is requiring RBC transfusions as defined as: a. Average RBC-transfusion frequency: 4 to 12 RBC units/12 weeks immediately up to randomization. There must be no interval > 6 weeks (42 days) without ≥ 1 RBC transfusion. b. RBC transfusions are scored in determining eligibility when given for treatment of: - Symptomatic (ie, fatigue or shortness of breath) anemia with a pretransfusion Hgb ≤ 9.5 g/dL or- Asymptomatic anemia with a pretransfusion Hgb ≤ 7 g/dL c. RBC transfusions given for worsening of anemia due to bleeding or infections are not scored in determining eligibility.\n- 4. Subjects on continuous (eg, absent of dose interruptions lasting ≥ 2 consecutive weeks) JAK2 inhibitor therapy as approved in the country of the study site for the treatment for MPN-associated MF as part of their standard-of-care therapy for at least 32 weeks, on stable daily dose for at least 16 weeks immediately up to the date of randomization and anticipated to be on a stable daily dose of that JAK2 inhibitor for at least 24 weeks after randomization.\n- 5. Subject has an Eastern Cooperative Oncology Group (ECOG) performance score of ≤2.\n- 6. A female of childbearing potential (FCBP) for this study is defined as a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (eg, has had menses at any time in the preceding 24 consecutive months). FCBP participating in the study must: a. Have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the study, and after end of IP. This applies even if the subject practices true abstinence* from heterosexual contact. b. Either commit to true abstinence* from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with, effective contraception** without interruption, 28 days prior to starting IP, during the study therapy (including dose interruptions), and for 12 weeks (approximately 5 times the mean terminal half-life of IP based on multiple-dose pharmacokinetics [PK] data) after discontinuation of study therapy.\n- 7. Male subjects must: Practice true abstinence* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential** while participating in the study, during dose interruptions and for at least 12 weeks (approximately 5 times the mean terminal half-life of IP based on multiple-dose PK data) following IP discontinuation, even if he has undergone a successful vasectomy."}

Exclusion criteria

  • {"criterion_text":"- 1. Subject with anemia from cause other than MPN-associated MF or JAK2 inhibitor therapy (eg, iron deficiency, vitamin B12 and/or folate deficiencies, autoimmune or hemolytic anemia, infection, or any type of known clinically significant bleeding or sequestration).\n- 2. Subject use of hydroxyurea, immunomodulatory compounds such as pomalidomide, thalidomide, ESAs, androgenic steroids or other drugs with potential effects on hematopoiesis ≤ 8 weeks immediately up to the date of randomization. a. Systemic corticosteroids are permitted for nonhematological conditions providing the subject is receiving a constant dose equivalent to ≤ 10 mg prednisone for the 4 weeks immediately up to randomization. b. Iron chelation therapy (ICT) is permitted providing the subject is receiving a stable dose for the 8 weeks immediately up to randomization.\n- 3. Subject with any of the following laboratory abnormalities at screening: a. Neutrophils: < 1 x 10^9/L b. White blood count (WBC): > 100 x 10^9/L c. Platelets: the lowest allowable level as approved for the concomitant JAK2 inhibitor but not < 25 x 10^9/L or > 1000 x 10^9/L d. Peripheral blood myeloblasts: > 5% e. Estimated glomerular filtration rate: < 30 mL/min/1.73 m2 (via the 4variable modification of diet in renal disease [MDRD] formula) or nephrotic subjects (eg, urine albumin-tocreatinine ratio> 3500 mg/g) f. Aspartate aminotransferase (AST) or alanine aminotransferase: (ALT) > 3.0 x upper limit of normal (ULN) g. Direct bilirubin: ≥ 2 x ULN - Higher levels are acceptable if these can be attributed to active red blood cell precursor destruction within the bone marrow (eg, ineffective erythropoiesis)\n- 4. Subject with uncontrolled hypertension, defined as repeated elevations of systolic blood pressure ≥ 140 mmHg or diastolic blood pressure ≥ 90 mmHg, that is not resolved at the time of randomization.\n- 5. Subject with prior history of malignancies, other than disease under study, unless the subject has been free of the disease for ≥ 3 years. However, subject with the following history/concurrent conditions is allowed: a. Basal or squamous cell carcinoma of the skin b. Carcinoma in situ of the cervix c. Carcinoma in situ of the breast d. Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system)\n- 6. Subject with prior hematopoietic cell transplant or subject anticipated to receive a hematopoietic cell transplant during the 24 weeks from the date of randomization.\n- 7. Subject with stroke, myocardial infarction, deep venous thrombosis, pulmonary or arterial embolism within 6 months immediately up to the date of randomization."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- The Primary end point of RBC-transfusion is defined as the proportion of subjects who become RBC transfusion free during any consecutive 12-week period","definition_or_measurement_approach":"The Primary end point is defined as the proportion of subjects who become RBC transfusion free during any consecutive 12-week period (RBC-transfusion independence over any consecutive 12-week period)."}

Secondary endpoints

  • {"endpoint_text":"- The key secondary endpoint, defined as the proportion of subjects who become RBC-transfusion free over any consecutive 16-week period starting during the Blinded Core Treatment Period (the time from randomization up to and including Week 24) and referred to as RBC-TI 16...","definition_or_measurement_approach":"Defined as the proportion of subjects who become RBC-transfusion free over any consecutive 16-week period starting during the Blinded Core Treatment Period (from randomization up to and including Week 24); referred to as RBC-TI 16."}
  • {"endpoint_text":"- .....will be tested in the same manner as the primary efficacy endpoint once superiority ofluspatercept on the primary endpoint is demonstrated.","definition_or_measurement_approach":"This secondary endpoint will be tested in the same manner as the primary efficacy endpoint contingent on demonstration of superiority on the primary endpoint."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
74
Recruitment Window Months
139
Consent Approach
Informed consent obtained from each participant (participants must be ≥18 years). Country-specific Main ICFs and related consent documents are available (multiple language versions listed: English, German, French, Spanish, Italian, Polish, Romanian, Czech, Greek, Hungarian, etc.). ICF requirements include specific pregnancy testing and contraception requirements for females of childbearing potential and requirements for males; optional/optional research consent and data privacy forms are provided where applicable.

Methods

  • HCP brochures (materials for healthcare professionals) — country-specific HCP brochures documented (e.g., K2_HCP-Brochure_BE, EL, IT).
  • Patient brochures / patient invitation materials — country-specific patient brochures and patient invitation letters (e.g., K2_Patient-Brochure_BE, Patient-Invitation-Letter_EL).
  • GP / Physician referral letters and GP outreach — country-specific GP/physician letters and referral cards (e.g., K2_GP-Letter_CZ, Referral-Letter_CZ, Physician_Referral-Card_EL).
  • Social posts — documented social media posts for recruitment (e.g., K2_HCP_Social-Posts_EL).
  • Scout/email communication and prepaid ScoutPass cards for targeted outreach (e.g., L2_Scout-EmailCommunication_EL, ScoutPass_PrepaidCard).
  • Site-level materials and local centre information (List_of_center_information_for_patient_information) and patient-facing transfusion diary to support participation and retention.

Geography

Total Number Of Sites
59
Total Number Of Participants
150

Hungary

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
06-03-2024
Processing Time Days
40
Number Of Sites
2
Number Of Participants
1

Sites

Site Name
Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz
Department Name
II. Belgyógyászat, Haematológiai Osztály
Principal Investigator Name
Eszter Sari
Principal Investigator Email
sarieszter@gmail.com
Contact Person Name
Eszter Sari
Contact Person Email
sarieszter@gmail.com
Site Name
Del-Pesti Centrumkorhaz Orszagos Hematologiai Es Infektologiai Intezet
Department Name
Haematológiai és Őssejt-transzplantációs Osztály
Principal Investigator Name
Gabor Mikala
Principal Investigator Email
gmikala@dpckorhaz.hu
Contact Person Name
Gabor Mikala
Contact Person Email
gmikala@dpckorhaz.hu

Ireland

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
04-03-2024
Processing Time Days
38
Number Of Sites
3
Number Of Participants
5

Sites

Site Name
St James's Hospital
Principal Investigator Name
Catherine Flynn
Principal Investigator Email
cmflynn@stjames.ie
Contact Person Name
Catherine Flynn
Contact Person Email
cmflynn@stjames.ie
Site Name
Mater Misericordiae University Hospital
Principal Investigator Name
Anne Fortune
Principal Investigator Email
afortune@mater.ie
Contact Person Name
Anne Fortune
Contact Person Email
afortune@mater.ie
Site Name
Cork University Hospital
Principal Investigator Name
Clodagh Keohane
Principal Investigator Email
ckeohane@muh.ie
Contact Person Name
Clodagh Keohane
Contact Person Email
ckeohane@muh.ie

Belgium

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
05-03-2024
Processing Time Days
39
Number Of Sites
4
Number Of Participants
3

Sites

Site Name
CHR Verviers
Department Name
Onco-haematology
Principal Investigator Name
Gaetan Vanstraelen
Principal Investigator Email
etudes.gvanstraelen@chrverviers.be
Contact Person Name
Gaetan Vanstraelen
Site Name
Centre hospitalier universitaire de Liege
Principal Investigator Name
Gaëlle Vertenoeil
Principal Investigator Email
gaelle.vertenoeil@chuliege.be
Contact Person Name
Gaëlle Vertenoeil
Contact Person Email
gaelle.vertenoeil@chuliege.be
Site Name
UZ Leuven
Department Name
Haematology
Principal Investigator Name
Timothy Devos
Principal Investigator Email
timothy.devos@uzleuven.be
Contact Person Name
Timothy Devos
Contact Person Email
timothy.devos@uzleuven.be
Site Name
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur
Department Name
Haematology
Principal Investigator Name
Julien Depaus
Principal Investigator Email
julien.depaus@chuuclnamur.uclouvain.be
Contact Person Name
Julien Depaus

Germany

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
06-03-2024
Processing Time Days
40
Number Of Sites
3
Number Of Participants
20

Sites

Site Name
Universitaetsklinikum Halle (Saale) AöR
Department Name
Universitätsklinik und Poliklinik für Innere Medizin IV
Principal Investigator Name
Haifa Kathrin Al-Ali
Principal Investigator Email
haifa.al-ali@uk-halle.de
Contact Person Name
Haifa Kathrin Al-Ali
Contact Person Email
haifa.al-ali@uk-halle.de
Site Name
Universitaetsklinikum Jena KöR
Department Name
Klinik für Innere Medizin II Abteilung Hämatologie und Internistische Onkologie
Principal Investigator Name
Carl Crodel
Principal Investigator Email
carl.crodel@med.uni-jena.de
Contact Person Name
Carl Crodel
Contact Person Email
carl.crodel@med.uni-jena.de
Site Name
Johannes Wesling Klinikum Minden
Department Name
Hämatologie, Onkologie
Principal Investigator Name
Martin Griesshammer
Contact Person Name
Martin Griesshammer

Austria

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
25-03-2024
Processing Time Days
59
Number Of Sites
1
Number Of Participants
4

Sites

Site Name
Ordensklinikum Linz GmbH
Department Name
interne 1 - Hämatologie mit Stammzelltransplantation, Hämostaseologie und medizinische Onkologie
Principal Investigator Name
Veronika Buxhofer-Ausch
Principal Investigator Email
veronika.buxhofer-ausch@ordensklinikum.at
Contact Person Name
Veronika Buxhofer-Ausch

Czechia

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
07-03-2024
Processing Time Days
41
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
I. interní klinika - klinika hematologie
Principal Investigator Name
Anna Jonášová
Principal Investigator Email
atjonas@hotmail.com
Contact Person Name
Anna Jonášová
Contact Person Email
atjonas@hotmail.com

Greece

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
22-03-2024
Processing Time Days
56
Number Of Sites
6
Number Of Participants
20

Sites

Site Name
University General Hospital Attikon
Department Name
Hematology Unit
Principal Investigator Name
Vasiliki Pappa
Principal Investigator Email
vas_pappa@yahoo.com
Contact Person Name
Vasiliki Pappa
Contact Person Email
vas_pappa@yahoo.com
Site Name
Geniko Nosokomeio Thessalonikis George Papanikolaou
Department Name
Hematology Clinic
Principal Investigator Name
Damianos Sotiropoulos
Principal Investigator Email
dsotiro@otenet.gr
Contact Person Name
Damianos Sotiropoulos
Contact Person Email
dsotiro@otenet.gr
Site Name
General University Hospital Of Patras
Department Name
Hematology Department-Pathology Clinic
Principal Investigator Name
Alexandros Spyridonidis
Principal Investigator Email
spyridonidis@upatras.gr
Contact Person Name
Alexandros Spyridonidis
Contact Person Email
spyridonidis@upatras.gr
Site Name
Evangelismos S.A.
Department Name
Hematology and Lymphoma Clinic - Bone Marrow Transplantation Unit
Principal Investigator Name
Maria Pagoni
Principal Investigator Email
marianpagoni@yahoo.com
Contact Person Name
Maria Pagoni
Contact Person Email
marianpagoni@yahoo.com
Site Name
University General Hospital Of Alexandroupoli
Department Name
Hematology Department
Principal Investigator Name
Ioannis Kotsianidis
Principal Investigator Email
ikotsian@med.duth.gr
Contact Person Name
Ioannis Kotsianidis
Contact Person Email
ikotsian@med.duth.gr
Site Name
Olympion Therapeftirio General Clinic Of Patras S.A.
Department Name
Hematology Department
Principal Investigator Name
Αnargyros Symeonidis
Principal Investigator Email
argiris.symeonidis@yahoo.gr
Contact Person Name
Αnargyros Symeonidis
Contact Person Email
argiris.symeonidis@yahoo.gr

France

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
05-03-2024
Processing Time Days
39
Number Of Sites
11
Number Of Participants
23

Sites

Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Service Hématologie Clinique
Principal Investigator Name
Michael Loschi
Principal Investigator Email
loschi.m@chu-nice.fr
Contact Person Name
Michael Loschi
Contact Person Email
loschi.m@chu-nice.fr
Site Name
Centre Hospitalier Universitaire Grenoble Alpes
Principal Investigator Name
Frederic Garban
Principal Investigator Email
fgarban@chu-grenoble.fr
Contact Person Name
Frederic Garban
Contact Person Email
fgarban@chu-grenoble.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Service Oncologie Hématologie
Principal Investigator Name
Shanti NATARAJAN-AME
Principal Investigator Email
s.ame@icans.eu
Contact Person Name
Shanti NATARAJAN-AME
Contact Person Email
s.ame@icans.eu
Site Name
Centre Hospitalier Universitaire De Nimes
Department Name
Service Hématologie Clinique
Principal Investigator Name
Stefan Wickenhauser
Principal Investigator Email
stefan.wickenhauser@chu-nimes.fr
Contact Person Name
Stefan Wickenhauser
Site Name
Hopital Saint Louis
Principal Investigator Name
Jean-Jacques Kiladjian
Principal Investigator Email
jean-jacques.kiladjian@aphp.fr
Contact Person Name
Jean-Jacques Kiladjian
Contact Person Email
jean-jacques.kiladjian@aphp.fr
Site Name
University Hospital Of Clermont-Ferrand
Department Name
Service d'hématologie clinique adultes et de thérapie cellulaire
Principal Investigator Name
Gaspar Aspas Requena
Principal Investigator Email
gaspasrequena@chu-clermontferrand.fr
Contact Person Name
Gaspar Aspas Requena
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Service des Maladies du Sang
Principal Investigator Name
Mathieu Wemeau
Principal Investigator Email
mathieu.wemeau@chru-lille.fr
Contact Person Name
Mathieu Wemeau
Contact Person Email
mathieu.wemeau@chru-lille.fr
Site Name
Hopital Haut Leveque
Department Name
Service d'hématologie clinique et Thérapie cellulaire
Principal Investigator Name
Clémence Médiavilla
Principal Investigator Email
clemence.mediavilla@chu-bordeaux.fr
Contact Person Name
Clémence Médiavilla
Site Name
CHRU de Poitiers La Miletrie
Department Name
Service Onco-Hématologie et Thérapie Cellulaire
Principal Investigator Name
Jose Miguel Torregrosa-Diaz
Contact Person Name
Jose Miguel Torregrosa-Diaz
Site Name
Centre Leon Berard
Principal Investigator Name
Franck Nicolini
Principal Investigator Email
franck-emmanuel.nicolini@lyon.unicancer.fr
Contact Person Name
Franck Nicolini
Site Name
Centre Hospitalier Universitaire D'Angers
Department Name
Département des maladies du Sang
Principal Investigator Name
Françoise Boyer
Principal Investigator Email
frboyer-perrard@chu-angers.fr
Contact Person Name
Françoise Boyer
Contact Person Email
frboyer-perrard@chu-angers.fr

Spain

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
06-03-2024
Processing Time Days
40
Number Of Sites
11
Number Of Participants
23

Sites

Site Name
Hospital Universitario De Salamanca
Department Name
Hematology
Principal Investigator Name
Jesus Maria Hernandez-Rivas
Principal Investigator Email
jmhr@usal.es
Contact Person Name
Jesus Maria Hernandez-Rivas
Contact Person Email
jmhr@usal.es
Site Name
Hospital Clinico Universitario De Valencia
Department Name
Hematology and Oncology
Principal Investigator Name
Juan Carlos Hernandez Boluda
Principal Investigator Email
hernandez_jca@gva.es
Contact Person Name
Anne Fortune
Contact Person Email
afortune@mater.ie
Site Name
El Hospital Universitario De Gran Canaria Dr. Negrin
Department Name
Hematology
Principal Investigator Name
Maria Teresa Gomez Casares
Principal Investigator Email
mgomcasf@gobiernodecanarias.org
Contact Person Name
Maria Teresa Gomez Casares
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
Hematology and Hemotherapy
Principal Investigator Name
Manuel Mateo Perez Encinas
Principal Investigator Email
manuel.mateo.perez.encinas@sergas.es
Contact Person Name
Manuel Mateo Perez Encinas
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Centro de Actividades Ambulatorias
Principal Investigator Name
Maria Isabel Montero Cuadrado
Principal Investigator Email
imonterohuvr@gmail.com
Contact Person Name
Maria Isabel Montero Cuadrado
Contact Person Email
imonterohuvr@gmail.com
Site Name
Hospital Universitario Virgen De Las Nieves
Department Name
Hematology
Principal Investigator Name
Francisca Hernandez Mohedo
Principal Investigator Email
paquihernandez@yahoo.es
Contact Person Name
Francisca Hernandez Mohedo
Contact Person Email
paquihernandez@yahoo.es
Site Name
Hospital Germans Trias I Pujol
Department Name
Hematology
Principal Investigator Name
Blanca Xicoy Cirici
Principal Investigator Email
bxicoy@iconcologia.net
Contact Person Name
Blanca Xicoy Cirici
Contact Person Email
bxicoy@iconcologia.net
Site Name
Hospital Universitario 12 De Octubre
Department Name
Centro de Actividades Ambulatorias
Principal Investigator Name
Rosa Ayala Diaz
Principal Investigator Email
rayaladiaz12@gmail.com
Contact Person Name
Rosa Ayala Diaz
Contact Person Email
rayaladiaz12@gmail.com
Site Name
Hospital Clinic De Barcelona
Department Name
Hematology
Principal Investigator Name
Alberto Alvarez-Larrán
Principal Investigator Email
aalvar@clinic.cat
Contact Person Name
Alberto Alvarez-Larrán
Contact Person Email
aalvar@clinic.cat
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
UTMO (Unidad de Transplante de Médula Ósea)
Principal Investigator Name
Jose Valentin Garcia Gutierrez
Principal Investigator Email
jvalentin.garcia@salud.madrid.org
Contact Person Name
Jose Valentin Garcia Gutierrez
Site Name
El Hospital Universitario De Gran Canaria Dr. Negrin (duplicate entry in list?)

Italy

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
29-05-2024
Processing Time Days
124
Number Of Sites
12
Number Of Participants
36

Sites

Site Name
Azienda Ospedaliero Universitaria Delle Marche
Department Name
Clinica di Ematologia
Principal Investigator Name
Antonella Poloni
Principal Investigator Email
a.poloni@univpm.it
Contact Person Name
Antonella Poloni
Contact Person Email
a.poloni@univpm.it
Site Name
Azienda Ospedaliero-Universitaria Maggiore Della Carita
Department Name
SCDU Ematologia
Principal Investigator Name
Andrea Patriarca
Principal Investigator Email
andrea.patriarca@uniupo.it
Contact Person Name
Andrea Patriarca
Contact Person Email
andrea.patriarca@uniupo.it
Site Name
Azienda Ospedale-Universita Padova
Department Name
U.O.C Ematologia
Principal Investigator Name
Gianni Binotto
Principal Investigator Email
gianni.binotto@aopd.veneto.it
Contact Person Name
Gianni Binotto
Contact Person Email
gianni.binotto@aopd.veneto.it
Site Name
Azienda Ospedaliero Ospedale Di Circolo E Fondazione Macchi
Department Name
S.C. di Ematologia
Principal Investigator Name
Marco Brociner
Principal Investigator Email
marco.brociner@asst-settelaghi.it
Contact Person Name
Marco Brociner
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
S.C. Ematologia
Principal Investigator Name
Francesco Passamonti
Principal Investigator Email
francesco.passamonti@policlinico.mi.it
Contact Person Name
Francesco Passamonti
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
U.O.C. Ematologia e Trapianti di Midollo
Principal Investigator Name
Fabrizio Pane
Principal Investigator Email
fabpane@unina.it
Contact Person Name
Fabrizio Pane
Contact Person Email
fabpane@unina.it
Site Name
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
Principal Investigator Name
Alessandro Lucchesi
Principal Investigator Email
a.lucchesi@irst.emr.it
Contact Person Name
Alessandro Lucchesi
Contact Person Email
a.lucchesi@irst.emr.it
Site Name
Centro Ricerche Cliniche Di Verona S.r.l.
Principal Investigator Name
Massimiliano Bonifacio
Principal Investigator Email
massimiliano.bonifacio@univr.it
Contact Person Name
Massimiliano Bonifacio
Site Name
Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco Di Catania
Department Name
Divisione Clinicizzata di Ematologia con Trapianto di Midollo Osseo
Principal Investigator Name
Giuseppe Alberto Maria Palumbo
Principal Investigator Email
studiclinicimpn.unict@gmail.com
Contact Person Name
Giuseppe Alberto Maria Palumbo
Site Name
Ospedale S. Eugenio
Department Name
U.O.C Ematologia
Principal Investigator Name
Elisabetta Abruzzese
Principal Investigator Email
elisabetta.abruzzese@uniroma2.it
Contact Person Name
Elisabetta Abruzzese
Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
S.C. Ematologia Universitaria
Principal Investigator Name
. Giulia Benevolo
Principal Investigator Email
gbenevolo@cittadellasalute.to.it
Contact Person Name
. Giulia Benevolo
Site Name
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Department Name
U.O. Ematologia
Principal Investigator Name
Mariella D’Adda
Principal Investigator Email
mariella.dadda@asst-spedalicivili.it
Contact Person Name
Mariella D’Adda

Romania

Earliest CTIS Part Ii Submission Date
26-01-2024
Latest Decision Or Authorization Date
06-03-2024
Processing Time Days
40
Number Of Sites
3
Number Of Participants
2

Sites

Site Name
Spitalul Clinic Municipal Filantropia Craiova
Department Name
Haematology Department
Principal Investigator Name
Luminita Ocroteala
Principal Investigator Email
diaconu_luminita@yahoo.com
Contact Person Name
Luminita Ocroteala
Contact Person Email
diaconu_luminita@yahoo.com
Site Name
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca
Department Name
Haematology Department
Principal Investigator Name
Ciprian Tomuleasa
Principal Investigator Email
ciprian.tomuleasa@gmail.com
Contact Person Name
Ciprian Tomuleasa
Contact Person Email
ciprian.tomuleasa@gmail.com
Site Name
Onco Card S.R.L.
Department Name
Haematology Department
Principal Investigator Name
Mihaela Lazaroiu
Principal Investigator Email
ellalaz@yahoo.com
Contact Person Name
Mihaela Lazaroiu
Contact Person Email
ellalaz@yahoo.com

Poland

Earliest CTIS Part Ii Submission Date
18-03-2024
Latest Decision Or Authorization Date
02-04-2024
Processing Time Days
15
Number Of Sites
2
Number Of Participants
10

Sites

Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Oddział Kliniczny Hematologii
Principal Investigator Name
Tomasz Sacha
Principal Investigator Email
sachatom@gmail.com
Contact Person Name
Tomasz Sacha
Contact Person Email
sachatom@gmail.com
Site Name
Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego
Department Name
Oddział Hematologii
Principal Investigator Name
Aleksandra Butrym
Principal Investigator Email
aleksandra.butrym@gmail.com
Contact Person Name
Aleksandra Butrym
Contact Person Email
aleksandra.butrym@gmail.com

Sponsor

Primary sponsor

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

Contract research organisations

Name
Q Squared Solutions Limited
Responsibilities
Clinical chemistry, Clinical haematology, Analytical chemistry
Name
Endpoint Clinical Inc.
Responsibilities
IVRS30 – treatment randomization and drug accountability.
Name
PPD Global Central Labs
Responsibilities
Clinical chemistry, Clinical haematology, Serology/ endocrinology, Analytical chemistry and Sample management.
Name
PPD Global Limited
Responsibilities
Monitoring
Name
Medidata Solutions Inc.
Responsibilities
Patient reported outcomes - ePRO
Name
Pharmaceutical Product Development LLC
Responsibilities
Clinical chemistry, Clinical haematology, Serology/ endocrinology, Analytical chemistry and Sample management.
Name
MLL Dx GmbH
Responsibilities
Clinical chemistry, Clinical haematology, Analytical chemistry
Name
Azenta US Inc.
Responsibilities
Long term storage facilities used samples

Third parties

  • {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"Clinical chemistry, Clinical haematology, Analytical chemistry","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"IVRS30 – treatment randomization and drug accountability.","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Clinical chemistry, Clinical haematology, Serology/ endocrinology, Analytical chemistry and Sample management.","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"PPD Global Limited","duties_or_roles":"Monitoring","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Patient reported outcomes - ePRO","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Clinical chemistry, Clinical haematology, Serology/ endocrinology, Analytical chemistry and Sample management.","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"MLL Dx GmbH","duties_or_roles":"Clinical chemistry, Clinical haematology, Analytical chemistry","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"Long term storage facilities used samples","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Reblozyl 25 mg powder for solution for injection
Active Substance
Luspatercept
Modality
Peptide/protein/enzyme
Routes Of Administration
Subcutaneous use
Route
Subcutaneous
Authorisation Status
Authorised (marketing authorisation numbers present)
Orphan Designation
Yes
Dose Levels
25 mg
Maximum Dose
14 mg/kg
Investigational Product Name
Reblozyl 75 mg powder for solution for injection
Active Substance
Luspatercept
Modality
Peptide/protein/enzyme
Routes Of Administration
Subcutaneous use
Route
Subcutaneous
Authorisation Status
Authorised (marketing authorisation numbers present)
Orphan Designation
Yes
Dose Levels
75 mg
Maximum Dose
14 mg/kg
Investigational Product Name
0.9 % w/v Sodium Chloride Injection
Active Substance
Sodium chloride
Modality
Small molecule
Routes Of Administration
Subcutaneous use
Route
Subcutaneous
Authorisation Status
Authorised (marketing authorisation numbers present)
Maximum Dose
14 mg/kg
Combination Treatment
Yes

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