NO_LONGER_AVAILABLENot Applicable

Compassionate Use Individual Request Program for GSK525762 in NUT Midline Carcinoma

Plain-language summary

Compassionate use access to molibresib/GSK525762 for eligible participant with NUT Midline Carcinoma; indication is a seriously debilitating or life-threatening disease.

Who can participate

Age range

16 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Participants meeting all three of the following criteria will be eligible:
. Written informed consent can be obtained from the participant or legally authorised representative.
. Participant must be ≥16 years of age, at the time of signing the informed consent.
. Demonstrate adequate organ function defined as :Hematological Absolute neutrophil count (ANC) ≥1.5 X 109/liter (L) ; Platelets ≥100 X 109/L ; Hemoglobin≥9.0 g/deciliter ; total bilirubin ≤ 1.5 x X ULN (upper limit of normal)(isolated bilirubin \>1.5 X ULN is acceptable if bilirubin is fractionated and direct bilirubin \<35% or participant has a diagnosis of Gilbert's syndrome) ; Alanine transaminase ≤ 2.5 X ULN OR ≤ 5 X ULN with documented liver metastases/tumor infiltration; Prothrombin Time (PT) or Partial Thromboplastin Time (PTT) ≤ 1.5 X upper limit of normal (ULN); Renal creatinine OR calculated creatinine clearance \[calculated or directly measured\] ≤1.5 X ULN or ≥ 50 mL/min; Ejection fraction ≥ Lower limit of normal (LLN); Troponin ≤ ULN
. Female participants who are not pregnant, breastfeeding and at least one of the following applies:
. Female participants of childbearing potential: Negative highly sensitive serum pregnancy test (as required by local regulations) within 72 hours prior to the first dose of molibresib.
. NOTE: The investigator is responsible for review of medical history, menstrual history and recent sexual activity to decrease risk for inclusion of a woman with an early undetected pregnancy.
. Male participants are eligible to participate if agree to the following during intervention period and for at least 16 weeks after the last dose of molibresib:

Exclusion criteria

. Use of anticoagulants (e.g., warfarin, heparin) at therapeutic levels within 7 days prior to the first dose of molibresib. Low dose (prophylactic) anticoagulants (e.g., low molecular weight heparin (LMWH) or oral anticoagulants) is permitted. In addition, INR must be monitored in accordance with local institutional practices, as appropriate.
. Concurrent use of non-steroidal anti-inflammatory drugs (NSAIDs) except for cases where NSAIDs provide benefit over other analgesics or high dose aspirin (allowed up to 100 mg PO daily).
. Participants with a history of known bleeding disorder(s) or history of clinically significant hemorrhage (e.g., GI, neurologic) within the past 6 months.
. Evidence of cardiac abnormalities as evidenced by any of the following:
. Current active liver or biliary disease (with the exception of Gilbert's syndrome or asymptomatic gallstones, liver metastases or otherwise stable chronic liver disease per investigator assessment).
. Evidence of severe or uncontrolled systemic diseases (e.g., unstable or uncompensated respiratory, hepatic, renal, cardiac disease, or clinically significant bleeding episodes). Any serious and/or unstable pre-existing medical or psychiatric disorder, or other conditions that could interfere with participant's safety, obtaining informed consent or compliance in the opinion of the investigator.
. Presence of hepatitis B surface antigen (HBsAg) or positive hepatitis C antibody test result at screening.
. History of known HIV infection.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

Trial details

NCT IDNCT03702036

SponsorGlaxoSmithKline

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

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