Clinical trial • Phase II • Oncology

SABATOLIMAB for Myelodysplastic syndromes (MDS) | Chronic myelomonocytic leukemia (CMML-2) | Acute myeloid leukemia (AML)

Phase II trial of SABATOLIMAB for Myelodysplastic syndromes (MDS) | Chronic myelomonocytic leukemia (CMML-2) | Acute myeloid leukemia (AML).

Overview

Trial Therapeutic Area
Oncology
Trial Disease
Myelodysplastic syndromes (MDS) | Chronic myelomonocytic leukemia (CMML-2) | Acute myeloid leukemia (AML)
Trial Stage
Phase II
Drug Modality
Monoclonal antibody | Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
13-08-2024
First CTIS Authorization Date
16-09-2024

Trial design

open-label, none/not specified-controlled Phase II trial across 12 sites in Spain, Greece, France and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
27

Eligibility

Recruits 27 paediatric patients.

Pregnancy Exclusion
Pregnant or nursing (lactating) women. Where pregnancy is defined as the state of a female after conception confirmed by a positive serum hCG laboratory test and until the termination of gestation.
Vulnerable Population
Vulnerable population selected. The trial includes provisions for minors/adolescents (Adolescent Assent and Parent/Legal Guardian consent forms are present). Written informed consent is required prior to enrolment; where participants are minors an assent form plus parent/legal guardian consent is provided.

Inclusion criteria

  • {"criterion_text":"- Participant is currently enrolled in a Novartis-sponsored study with sabatolimab, is being treated with sabatolimab, and has fulfilled all requirements in the parent study."}
  • {"criterion_text":"- Participant is currently benefiting from the treatment with sabatolimab as determined by guidelines of the parent protocol and investigator's judgment."}
  • {"criterion_text":"- Participant has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements."}
  • {"criterion_text":"- Willingness and ability to comply with scheduled visits, treatment plan and any other study procedures."}
  • {"criterion_text":"- Written informed consent obtained prior to enrolling in the roll-over study."}

Exclusion criteria

  • {"criterion_text":"- Participants in cohorts or treatment groups not receiving sabatolimab in the parent protocol."}
  • {"criterion_text":"- Participant has been permanently discontinued from sabatolimab treatment in the parent study due to unacceptable toxicity, noncompliance to study procedures, withdrawal of consent or any other reason."}
  • {"criterion_text":"- Participant currently has unresolved toxicities for which sabatolimab dosing has been interrupted in the parent study (participants meeting all other eligibility criteria may be enrolled once toxicities have resolved to allow sabatolimab dosing to resume)."}
  • {"criterion_text":"- Pregnant or nursing (lactating) women. Where pregnancy is defined as the state of a female after conception confirmed by a positive serum hCG laboratory test and until the termination of gestation."}
  • {"criterion_text":"- Participant not willing to comply with the contraception requirements outlined in the exclusion criteria of the parent protocol."}
  • {"criterion_text":"- Local access to commercially available sabatolimab for parent protocol indications."}
  • {"criterion_text":"- (other exclusion criteria not provided in the Part I principalExclusionCriteria list)"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Frequency and severity of AEs and SAEs","definition_or_measurement_approach":"Assessment of frequency and severity of adverse events (AEs) and serious adverse events (SAEs) as reported during exposure to sabatolimab (no further measurement detail provided in the Part I data)."}

Secondary endpoints

  • {"endpoint_text":"- Duration of exposure to sabatolimab","definition_or_measurement_approach":"Collection of duration of exposure (time on treatment) to sabatolimab (no further measurement detail provided in the Part I data)."}

Recruitment

Planned Sample Size
27
Recruitment Window Months
60
Consent Approach
Written informed consent is required prior to enrolment. Age-specific documents are provided: adult ICFs, Parent/Legal Guardian consent forms and Adolescent assent forms. ICFs/assent documents are available in multiple languages (examples in the dossier include German, French, Italian, Spanish, Czech, Greek and English).

Geography

Total Number Of Sites
12
Total Number Of Participants
18

Spain

Earliest CTIS Part Ii Submission Date
09-09-2024
Latest Decision Or Authorization Date
04-06-2025
Processing Time Days
268
Number Of Sites
3
Number Of Participants
7

Sites

Site Name
Institut Catala D'oncologia
Department Name
1803: Hematología
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 Clinic De Barcelona
Department Name
1800: Hematología
Principal Investigator Name
Jordi Esteve Reyner
Principal Investigator Email
JESTEVE@clinic.cat
Contact Person Name
Jordi Esteve Reyner
Contact Person Email
JESTEVE@clinic.cat
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
1805: Hematología
Principal Investigator Name
Patricia Font Lopez
Principal Investigator Email
patricia.font@salud.madrid.org
Contact Person Name
Patricia Font Lopez
Contact Person Email
patricia.font@salud.madrid.org

Greece

Earliest CTIS Part Ii Submission Date
09-09-2024
Latest Decision Or Authorization Date
30-09-2025
Processing Time Days
386
Number Of Sites
2
Number Of Participants
2

Sites

Site Name
General University Hospital Of Patras
Department Name
1300: Hematology Division, Hematology Department
Principal Investigator Name
Alexandros Spyridonidis
Principal Investigator Email
spyridonidis@upatras.gr
Contact Person Name
Alexandros Spyridonidis
Contact Person Email
spyridonidis@upatras.gr
Site Name
University General Hospital Of Alexandroupoli
Department Name
1301: Hematology
Principal Investigator Name
Ioannis Kotsianidis
Principal Investigator Email
ikotsian@med.duth.gr
Contact Person Name
Ioannis Kotsianidis
Contact Person Email
ikotsian@med.duth.gr

France

Earliest CTIS Part Ii Submission Date
09-09-2024
Latest Decision Or Authorization Date
02-06-2025
Processing Time Days
266
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Oncopole Claudius Regaud
Department Name
1001: Hematology
Principal Investigator Name
Christian Recher
Principal Investigator Email
Recher.Christian@iuct-oncopole.fr
Contact Person Name
Christian Recher

Czechia

Earliest CTIS Part Ii Submission Date
09-09-2024
Latest Decision Or Authorization Date
03-06-2025
Processing Time Days
267
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
1100: I. Interní klinika - klinika hematologie
Principal Investigator Name
Anna Jonášová
Principal Investigator Email
anna.jonasova@vfn.cz
Contact Person Name
Anna Jonášová
Contact Person Email
anna.jonasova@vfn.cz

Germany

Earliest CTIS Part Ii Submission Date
09-09-2024
Latest Decision Or Authorization Date
20-09-2024
Processing Time Days
11
Number Of Sites
2
Number Of Participants
1

Sites

Site Name
Medical Center - University Of Freiburg
Department Name
2211: Klinik fuer Innere Medizin I
Principal Investigator Name
Robert Zeiser
Principal Investigator Email
Robert.zeiser@uniklinik-freiburg.de
Contact Person Name
Robert Zeiser
Site Name
Universitaet Leipzig
Department Name
2200: Klinik und Poliklinik fuer Haematologie, Zelltherapie und Haemostaseologie
Principal Investigator Name
Uwe Platzbecker
Principal Investigator Email
uwe.platzbecker@medizin.uni-leipzig.de
Contact Person Name
Uwe Platzbecker

Italy

Earliest CTIS Part Ii Submission Date
09-09-2024
Latest Decision Or Authorization Date
09-07-2025
Processing Time Days
303
Number Of Sites
3
Number Of Participants
5

Sites

Site Name
Azienda Ospedaliera Policlinico Universitario Tor Vergata
Department Name
2404: Oncoematologia
Principal Investigator Name
Adriano Venditti
Principal Investigator Email
adriano.venditti@uniroma2.it
Contact Person Name
Adriano Venditti
Contact Person Email
adriano.venditti@uniroma2.it
Site Name
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Department Name
2408: U.S.D. Trapianto Midollo Osseo per Adulti
Principal Investigator Name
Domenico Russo
Principal Investigator Email
domenico.russo@unibs.it
Contact Person Name
Domenico Russo
Contact Person Email
domenico.russo@unibs.it
Site Name
Careggi University Hospital
Department Name
2403: Hematology
Principal Investigator Name
Valeria Santini
Principal Investigator Email
valeria.santini@unifi.it
Contact Person Name
Valeria Santini
Contact Person Email
valeria.santini@unifi.it

Sponsor

Primary sponsor

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

Contract research organisations

Name
Icon Clinical Research Limited
Responsibilities
sponsorDuties codes: 1
Name
Syneos Health Inc.
Responsibilities
sponsorDuties codes: 1
Name
PRA Hellas CRO A.E.
Responsibilities
sponsorDuties codes: 1
Name
Parexel International (IRL) Limited
Responsibilities
sponsorDuties codes: 12
Name
Medidata Solutions International Limited
Responsibilities
sponsorDuties codes: 15 (quality control); 6
Name
IQVIA Limited
Responsibilities
sponsorDuties codes: 1; 15 (drug dispensing/reconciliation cycle visits); 3
Name
Opis S.r.l.
Responsibilities
sponsorDuties codes: 15 (TMF archive)

Third parties

  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"sponsorDuties codes: 1","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"sponsorDuties codes: 1","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"sponsorDuties codes: 1","organisation_type":"Pharmaceutical company"}
  • {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"sponsorDuties codes: 15 (TMF archive)","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties codes: 12","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Medidata Solutions International Limited","duties_or_roles":"sponsorDuties codes: 15 (quality control); 6","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties codes: 1; 15 (drug dispensing/reconciliation cycle visits); 3","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
MBG453
Active Substance
SABATOLIMAB
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS USE
Route
INTRAVENOUS
Authorisation Status
euMpNumber: PRD7812649, prodAuthStatus: 1
Maximum Dose
800 mg
Investigational Product Name
VENETOCLAX
Active Substance
VENETOCLAX
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
ORAL
Authorisation Status
marketingAuthNumber: - , prodAuthStatus: 2
Maximum Dose
400 mg
Investigational Product Name
AZACITIDINE
Active Substance
AZACITIDINE
Modality
Small molecule
Routes Of Administration
INTRAVENOUS (IV) OR SUBCUTANEOUS (SC)
Route
INTRAVENOUS or SUBCUTANEOUS
Authorisation Status
euMpNumber: SUB05624MIG, prodAuthStatus: 2
Maximum Dose
75 mg/m2
Combination Treatment
Yes

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