An extension study for patients previously enrolled in studies with pelabresib

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

1 Oct 2024 → ongoing

Decision date (initial)

2024-08-29

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2023-508950-24-00

ClinicalTrials.gov

NCT06401356

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety, Efficacy

• To evaluate long-term safety in patients who are receiving pelabresib
• To evaluate survival
• To evaluate clinical benefit per investigator assessment

Conditions and MedDRA coding

Myelofibrosis

Study design 1 period

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. Eligibility for Ongoing Pelabresib Treatment (criteria 1-6 ) 1. Capable of giving signed informed consent as described in Section 12.2.2, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
2. Participant must be at least 18 years of age and the legal age of consent in the jurisdiction in which the study is taking place at the time of signing the informed consent
3. Was previously enrolled and is still receiving treatment with pelabresib in a parent clinical study
4. Is receiving clinical benefit from treatment with pelabresib in the parent study, as assessed by the investigator
5. Willingness and ability to comply with scheduled visits, treatment plans, and any other study procedures indicated in this protocol
6. Agrees to avoid pregnancy or fathering children based on the criteria below: a. Male participants and their female partners of childbearing potential must agree to take appropriate precautions to avoid fathering children (with at least 99% certainty) from the time of the eligibility check through 94 days after the last dose of pelabresib. Additionally, male participants must refrain from donating sperm during this period. Permitted methods that are at least 99% effective in preventing pregnancy (Section 7.13.) should be communicated to the participants and their understanding confirmed. b. WOCBP must have a negative serum pregnancy test at the time of the eligibility check and must agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from the time of the eligibility check until the end of relevant systemic exposure (ie, through 184 days after the last dose of pelabresib). They must also agree to regular urine pregnancy testing through the study treatment period and monthly pregnancy testing up to 184 days after the last dose of pelabresib. They must also refrain from breastfeeding and donating oocytes during the course of study and for 184 days after the last dose of pelabresib. Permitted methods that are at least 99% effective in preventing pregnancy (Section 7.13) should be communicated to the participants and their understanding confirmed. c. Women without childbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal with ≥12 months of amenorrhea without an alternative medical cause) are eligible. NOTE: Female participants who have been amenorrheic for at least 12 months resulting from chemo/radiotherapy are considered of childbearing potential and should agree to use adequate contraceptive measures (Section 7.13).
7. Eligibility for Survival Follow-up (1): Capable of giving signed informed consent as described in Section 12.2.2, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol
8. Eligibility for Survival Follow-up (2) : Participant must be at least 18 years of age and the legal age of consent in the jurisdiction in which the study is taking place at the time of signing the ICF
9. Eligibility for Survival Follow-up (3): Participant was previously enrolled in a clinical study with pelabresib
10. Eligibility for Survival Follow-up (4): Willingness and ability to comply with ongoing follow-up as indicated in this protocol

Exclusion criteria 15

1. Eligibility for Ongoing Pelabresib Treatment (criteria 1-14 ): 1. Participants who are legally institutionalized or under judicial protection
2. Concurrent enrollment in another interventional clinical trial (other than the parent study)
3. History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical class
4. Any other medical condition that, in the investigator’s opinion, makes the participant unsuitable for the study
5. participant with an uncontrolled intercurrent illness or any concurrent condition that, in the investigator’s opinion, would jeopardize the safety of the participant or compliance with the protocol
6. Female participants who are pregnant or breastfeeding or not on adequate contraceptive therapy specified in inclusion criteria
7. A male participants who does not agree to use contraception as specified in the inclusion criteria during the treatment period and for at least 94 days after the last dose of pelabresib (if they have a heterosexual partner who is a WOCBP) and who does not refrain from donating sperm during this period
8. Eligibility for Survival Follow-up : 1. participants who are legally institutionalized or under judicial protection
9. Participants with impaired gastrointestinal function or gastrointestinal disease, including active IBD, that could significantly alter the absorption of study drug, including any unresolved nausea, vomiting, or diarrhea > Grade 1
10. Had systemic anticancer treatment or treatment with investigational agents, with the exception of the study drug in the parent study or hormonal therapy, less than 2 weeks (or 5 half-lives, whichever is longer) before the first dose of study drug (Section 7.10). NOTE: Hydroxyurea and anagrelide are permitted up to 24 hours prior to start of study drug.
11. Had hematopoietic growth factor (granulocyte growth factor, erythropoiesis stimulating agent, thrombopoietin mimetic) or androgenic steroids less than 4 weeks before the first dose of study drug (Section 7.10)
12. Had a strong CYP3A4 inhibitor or inducer within 2 weeks prior to the first dose of study drug, including St. John’s wort (Section 7.10). Initiation of treatment or concomitant use of a strong CYP3A4 inhibitor or inducer during study treatment is prohibited.
13. Had immunosuppressive agents with systemic activity (>10 mg QD prednisone or equivalent) less than 4 weeks before the first dose of study drug. Participants who received topical, nasal, intra-articular, inhaled, and other forms of corticosteroids without systemic activity are eligible (Section 7.10 ).
14. Had a live vaccination within 30 days prior to the first dose of study drug, except for COVID-19 vaccination (Section 7.10)
15. Participants who are unwilling or unable to comply with this study protocol or study requirements

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Occurrence and severity of treatment-emergent adverse events (TEAEs) and serious TEAEs among patients who are receiving pelabresib
2. Survival and leukemia-free survival
3. Time-to-event endpoints (eg, duration of response, progression-free survival)

Investigational products

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

Test 2

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

3 EU/EEA countries · 6 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 24 files

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

Application history

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