Clinical trial • Phase IV • Oncology

Ruxolitinib for Thalassemia | Chronic graft versus host disease | Acute graft versus host disease | Chronic graft versus host disease in skin | Chronic graft versus host disease in liver | Chronic graft versus host disease in intestine | Acute graft versus host disease in skin | Acute graft versus host disease in liver | Acute graft versus host disease in intestine | Post essential thrombocythaemia myelofibrosis | Post essential thrombocythemia myelofibrosis | Post polycythaemia vera myelofibrosis | Polycythaemia vera

Phase IV trial of Ruxolitinib for Thalassemia | Chronic graft versus host disease | Acute graft versus host disease | Chronic graft versus host disease in…

Overview

Trial Therapeutic Area
Oncology
Trial Disease
Thalassemia | Chronic graft versus host disease | Acute graft versus host disease | Chronic graft versus host disease in skin | Chronic graft versus host disease in liver | Chronic graft versus host disease in intestine | Acute graft versus host disease in skin | Acute graft versus host disease in liver | Acute graft versus host disease in intestine | Post essential thrombocythaemia myelofibrosis | Post essential thrombocythemia myelofibrosis | Post polycythaemia vera myelofibrosis | Polycythaemia vera
Trial Stage
Phase IV
Drug Modality
Small molecule
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
09-08-2024
First CTIS Authorization Date
11-09-2024

Trial design

open-label, none/not specified-controlled Phase IV trial across 15 sites in Germany, Italy, Sweden and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
106

Eligibility

Recruits 106 paediatric patients.

Vulnerable Population
The study includes vulnerable populations (isVulnerablePopulationSelected = true). Age-specific consent/assent documents are provided: Parent Legal Guardian ICF, Adolescent Assent, Child Assent, and Main ICF for adults. Site-specific informed consent and assent procedures are implemented (parent/guardian consent for minors and assent for adolescents/children). Country-specific ICF/assent documents are included for participating countries.

Inclusion criteria

  • {"criterion_text":"- Patient is currently enrolled in a Novartis-sponsored GDD or GMA study or Incyte-sponsored study (where Incyte can delegate the sponsorship to a preferred CRO, if applicable) and are receiving either ruxolitinib or combination of ruxolitinib and panobinostat, or combinations of ruxolitinib and siremadlin, or ruxolitinib and rineterkib and fulfilled all the requirements of the parent protocol. Please refer to the list of parent studies in Appendix 2."}
  • {"criterion_text":"- Patient is currently benefitting from the treatment with ruxolitinib monotherapy or combination of ruxolitinib and panobinostat, or combinations of ruxolitinib and siremadlin or ruxolitinib and rineterkib as determined by the investigator."}

Exclusion criteria

  • {"criterion_text":"- Patient has been permanently discontinued from study treatment in parent study due to any reason."}
  • {"criterion_text":"- Patient’s indication is currently approved and reimbursed in the corresponding country for ruxolitinib monotherapy or combination of ruxolitinib with panobinostat, or ruxolitinib and siremadlin or ruxolitinib and rineterkib (if the patient is receiving combination treatment in the parent study)."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Frequency and severity of SAEs/AEs","definition_or_measurement_approach":"To evaluate long term safety data, i.e. SAEs and AEs"}

Secondary endpoints

  • {"endpoint_text":"- Proportion of patients with clinical benefit as assessed by the investigator at scheduled visits","definition_or_measurement_approach":"Clinical benefit assessed by the investigator at scheduled visits"}
  • {"endpoint_text":"- Frequency and severity of AEs / SAEs","definition_or_measurement_approach":"Frequency and severity of AEs/SAEs as recorded"}
  • {"endpoint_text":"- Proportion of patients with clinical benefit and assessed by the investigators at scheduled visits","definition_or_measurement_approach":"Clinical benefit assessed by the investigators at scheduled visits"}

Recruitment

Planned Sample Size
106
Recruitment Window Months
146
Consent Approach
Informed consent is obtained from adult participants. For minors, parent/legal guardian consent is required and adolescent and child assent documents are provided. Country-specific informed consent and assent documents are available (documents listed for Italy, Poland, Sweden, Germany and English versions). Additional documents include follow-up ICFs for pregnant participants and information sheets for female partners; Poland includes a separate data protection consent form.

Geography

Total Number Of Sites
15
Total Number Of Participants
33

Germany

Earliest CTIS Part Ii Submission Date
28-08-2024
Latest Decision Or Authorization Date
17-09-2024
Processing Time Days
20
Number Of Sites
4
Number Of Participants
5

Sites

Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
2005: III. Medizinische Klinik und Poliklinik
Contact Person Name
Daniel Sasca
Site Name
Universitaetsklinikum Magdeburg AöR
Department Name
2006: Klinik für Hämatologie und Onkologie
Contact Person Name
Denise Walther
Contact Person Email
Denise.Walther@med-ovgu.de
Site Name
Universitaetsklinikum Jena KöR
Department Name
2009: Klinik für Innere Medizin II Abteilung Hämatologie und Internistische Onkologie
Contact Person Name
Andreas Hochhaus
Site Name
Universitaetsklinikum Aachen AöR
Department Name
2001: Medizinische Klinik VI
Contact Person Name
Steffen Koschmieder
Contact Person Email
skoschmieder@ukaachen.de

Italy

Earliest CTIS Part Ii Submission Date
28-08-2024
Latest Decision Or Authorization Date
16-09-2024
Processing Time Days
19
Number Of Sites
5
Number Of Participants
19

Sites

Site Name
Grande Ospedale Metropolitano Bianchi Melacrino Morelli
Department Name
3004: U.O.C. Ematologia
Contact Person Name
Caterina Alati
Contact Person Email
caterina.alati@gmail.com
Site Name
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Department Name
3015: U.S.D. Trapianto Midollo Adulti
Contact Person Name
Domenico Russo
Contact Person Email
domenico.russo@unibs.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
3007: S.C. Ematologia 1 Dipartimento Oncologia
Contact Person Name
Luca Arcaini
Contact Person Email
luca.arcaini@unipv.it
Site Name
Careggi University Hospital
Department Name
3001: Dipartimento di Medicina Sperimentale e Clinica S.O.D.C. di Ematologia
Contact Person Name
Alessandro Maria Vannucchi
Contact Person Email
a.vannucchi@unifi.it
Site Name
Casa Sollievo Della Sofferenza
Department Name
3023: S.S.D. di Ematologia e Terapie Cellulari
Contact Person Name
Angelo Michele Carella
Contact Person Email
am.carella@operapadrepio.it

Sweden

Earliest CTIS Part Ii Submission Date
28-08-2024
Latest Decision Or Authorization Date
11-09-2024
Processing Time Days
14
Number Of Sites
4
Number Of Participants
5

Sites

Site Name
NU Hospital Group-Vastra Gotalandsregionen
Department Name
9501: Uddevalla Hospital, Hematologmottagningen, 54180 Uddevalla
Contact Person Name
Björn Andreasson
Contact Person Email
bjorn.andreasson@vgregion.se
Site Name
Region Norrbotten
Department Name
9504: Hematologisk dagvård, Medicinkliniken, Sunderby sjukhus, 97180 Luleå
Contact Person Name
Birgitta Lauri
Contact Person Email
birgitta.lauri@norrbotten.se
Site Name
Karolinska University Hospital
Department Name
9502: Hematologiskt Centrum
Contact Person Name
Per-Anders Broliden
Site Name
Region Skane Skanes Universitetssjukhus
Department Name
9503: VO Hematologi, Kliniska forskningsenheten, 221 85 Lund
Contact Person Name
Lars Nilsson
Contact Person Email
lars.nilsson@skane.se

Poland

Earliest CTIS Part Ii Submission Date
28-08-2024
Latest Decision Or Authorization Date
16-10-2024
Processing Time Days
49
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Department Name
2401: Klinika Hematologii, Terapii Komórkowych i Chorób Wewnetrznych
Contact Person Name
Tomasz Wróbel
Contact Person Email
tomasz.wrobel@umed.wroc.pl
Site Name
Narodowy Instytut Onkologii Im. Marii Sklodowskiej-Curie Panstwowy Instytut Badawczy
Department Name
2402: Klinika Transplantacji Szpiku i Onkohematologii
Contact Person Name
Sebastian Giebel
Contact Person Email
sgiebel@io.gliwice.pl

Sponsor

Primary sponsor

Full Name
Novartis Pharma Services AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
Syneos Health Inc.
Responsibilities
code 1
Name
Parexel International (IRL) Limited
Responsibilities
code 12
Name
Icon Clinical Research Limited
Responsibilities
code 1
Name
IQVIA Limited
Responsibilities
code 1

Third parties

  • {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"TMF archive, Activation sites activities","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"code 12","organisation_type":"Pharmaceutical company"}
  • {"country":"Italy","full_name":"Mipharm S.p.A.","duties_or_roles":"Local drug supply, Local equipment storage","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
  • {"country":"Italy","full_name":"Phardis S.r.l.","duties_or_roles":"Local drug supply, Local equipment storage","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Ruxolitinib (Jakavi)
Active Substance
Ruxolitinib
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
Authorised
Dose Levels
5 mg, 10 mg, 15 mg, 20 mg
Maximum Dose
50 mg (max daily dose amount)
Investigational Product Name
Panobinostat (Farydak)
Active Substance
Panobinostat
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
Authorised
Orphan Designation
Yes
Dose Levels
10 mg, 15 mg
Maximum Dose
30 mg (max daily dose amount)
Investigational Product Name
Siremadlin (HDM201)
Active Substance
Siremadlin
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
Investigational
Maximum Dose
40 mg (max daily dose amount)
Combination Treatment
Yes

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