Roll-over study for patients who have completed a prior Novartis sponsored sabatolimab (MBG453) study and are judged by the investigator to benefit from continued treatment with sabatolimab

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

13 Feb 2023 → ongoing

Decision date (initial)

2024-09-20

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2024-515281-14-00

EudraCT number

2021-004422-30

ClinicalTrials.gov

NCT05201066

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Others

To evaluate safety, including SAE and AE

Secondary objectives 1

1. To collect exposure to sabatolimab

Conditions and MedDRA coding

intermediate, high or very high risk MDS in adult patients chronic Myelomonocytic Leukemia - 2 (CMML-2) Acute Myeloid Leukemia (AML) in adult patients unfit for chemotherapy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 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.
2. Participant is currently benefiting from the treatment with sabatolimab as determined by guidelines of the parent protocol and investigator's judgment.
3. Participant has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements.
4. Willingness and ability to comply with scheduled visits, treatment plan and any other study procedures.
5. Written informed consent obtained prior to enrolling in the roll-over study.

Exclusion criteria 6

1. Participants in cohorts or treatment groups not receiving sabatolimab in the parent protocol.
2. 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.
3. 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).
4. 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.
5. Participant not willing to comply with the contraception requirements outlined in the exclusion criteria of the parent protocol.
6. Local access to commercially available sabatolimab for parent protocol indications.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Frequency and severity of AEs and SAEs

Secondary endpoints 1

1. Duration of exposure to sabatolimab

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 5

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 7

Locations

6 EU/EEA countries · 12 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 59 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications