Clinical Trials

Inclusion Criteria:

• 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.
• Patient 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.
• Having been enrolled and is still receiving treatment with pelabresib in a parent clinical study.
• Is receiving clinical benefit from treatment with pelabresib in the parent study, as assessed by the investigator.
• Willingness and ability to comply with scheduled visits, treatment plans, and any other study procedures indicated in this protocol.
• Agrees to avoid pregnancy or fathering children based on the criteria below:
  • Male patients 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 patients must refrain from donating sperm during this period. Permitted methods that are at least 99% effective in preventing pregnancy (see Section 7.13) should be communicated to the patients and their understanding confirmed.
  • Women of childbearing potential (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 (see Section 7.13) should be communicated to the patients and their understanding confirmed.
  • 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 patients 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 (see Section 7.13). Patients may consider seeking information from the study investigator regarding donation and cryopreservation of germ cells prior to treatment. Male patients should be informed of the risk of testicular toxicity and provided with adequate advice regarding sperm preservation.

Eligibility for Survival Follow-up (Overall Survival and Leukemia-Free Survival) Patients are eligible to be included in the study for continued Survival Follow-up if all the following criteria are fulfilled:

• 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.
• Patient 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.
• Patient has been enrolled in a clinical study with pelabresib.
• Willingness and ability to comply with ongoing follow-up as indicated in this protocol.

Exclusion Criteria:

• Patients who are legally institutionalized or under judicial protection.
• Concurrent enrollment in another interventional clinical trial (other than the parent study).
• History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical class.
• Any other medical condition that, in the investigator’s opinion, makes the patient unsuitable for the study.
• Patient is able to access pelabresib outside the study 6. Patient with an uncontrolled intercurrent illness or any concurrent condition that, in the investigator’s opinion, would jeopardize the safety of the patient or compliance with the protocol.
• Female patients who are pregnant or breastfeeding or not on adequate contraceptive therapy specified in inclusion criteria.
• A male patient 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

Eligibility for Survival Follow-up (Overall Survival and Leukemia-Free Survival) Patients are excluded from the study for Survival Follow-up if the following criterion applies:

• Patients who are legally institutionalized or under judicial protection.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 4/29/2024. Questions regarding updates should be directed to the study team contact.