Solid TumoursNCT03702036
Compassionate Use Individual Request Program for GSK525762 in NUT Midline Carcinoma
A study testing molibresib in people with Solid Tumours, run by GlaxoSmithKline.
Trial summary
Compassionate use access to molibresib/GSK525762 for eligible participant with NUT Midline Carcinoma; indication is a seriously debilitating or life-threatening disease. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Solid Tumours
- What is being tested?
- molibresib
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Participants meeting all three of the following criteria will be eligible:
- Diagnosis of NUT Midline Carcinoma based on ectopic expression of NUT protein as determined by IHC and/or detection of NUT gene translocation as determined by FISH.
- there is no satisfactory alternative treatment; and
- there is reason to believe that the benefit:risk ratio for molibresib in the indication is positive.
- 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.
Show all 15Show fewer
- 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:
- Is not a woman of childbearing potential (WOCBP) OR Is a WOCBP and using a contraceptive method that is highly effective (with a failure rate of <1% per year, see Appendix 5), preferably with low user dependency, during the intervention period and for at least 7 months after the last dose of molibresib. The investigator should evaluate the effectiveness of the contraceptive method in relationship to the first dose of molibresib.
- 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:
- Refrain from donating sperm
- PLUS, if have a female partner of childbearing potential, either:
- Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent -OR- Agree to use a male condom and if female partner is not pregnant, partner to use an additional highly effective contraceptive method with a failure rate of <1% per year.
Who cannot join
- 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:
- History or current clinically significant conduction abnormalities, uncontrolled arrhythmias or hypertension.
- History or evidence of current ≥Class II congestive heart failure as defined by New York Heart Association (NYHA).
Show all 15Show fewer
- Recent history (within the past 3 months) of acute coronary syndromes (including unstable angina and myocardial infarction), coronary angioplasty, or stenting
- Baseline QTcF interval ≥480 msec
- 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.
- NOTE: Participants with positive Hepatitis C antibody due to prior resolved disease can be enrolled only if a confirmatory negative Hepatitis C RNA PCR is obtained.
- History of known HIV infection.
- Hemoptysis >1 teaspoon in 24 hours within the last 28 days.
- GSK525762 is a benzodiazepine class molecule. Any serious known immediate or delayed hypersensitivity reaction(s) to molibresib or idiosyncrasy to drugs chemically related to molibresib.
About this study
Compassionate use access to molibresib/GSK525762 for eligible participant with NUT Midline Carcinoma; indication is a seriously debilitating or life-threatening disease.
Read the detailed description
Investigational Product GSK525762 is an orally bioavailable small molecule that is a potent inhibitor of the binding of BET proteins to acetylated histones. Binding of GSK525762 induces squamous differentiation and inhibits proliferation of patient-derived NMC cell lines both in culture and in murine xenografts.
Compassionate use access to molibresib is restricted to patients with confirmed diagnosis of NUT Midline Carcinoma.
- Conditions
- Solid Tumours
- Being tested
- molibresib
- Keywords
- Nuclear protein in testis (NUT), NUT Midline Carcinoma (NMC)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
molibresibDrug
molibresib 20 mg will be supplied for compassionate use for the identified individual subject.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Solid TumoursStudies of the same condition, open and under way.
- More trials run by GlaxoSmithKlineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- GlaxoSmithKline
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- GSK Clinical Trials, Study director, GlaxoSmithKline
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03702036
- Sponsor's protocol ID
- 209447
- Status last verified
- Apr 2020
- First submitted
- 4 Oct 2018
- Last updated
- 13 Apr 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use Individual Request Program for GSK525762 in NUT Midline Carcinoma
- ClinicalTrials.gov ID
- NCT03702036
- Acronym / sponsor ID
- 209447
- Conditions
- Solid Tumours
- Keywords
- Nuclear protein in testis (NUT), NUT Midline Carcinoma (NMC)
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- GlaxoSmithKlineIndustry (pharma, biotech, devices)
- First posted
- 10 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Compassionate use access to molibresib/GSK525762 for eligible participant with NUT Midline Carcinoma; indication is a seriously debilitating or life-threatening disease.
Detailed description
Investigational Product GSK525762 is an orally bioavailable small molecule that is a potent inhibitor of the binding of BET proteins to acetylated histones. Binding of GSK525762 induces squamous differentiation and inhibits proliferation of patient-derived NMC cell lines both in culture and in murine xenografts.
Compassionate use access to molibresib is restricted to patients with confirmed diagnosis of NUT Midline Carcinoma.
Study design
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- 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:
Key exclusion criteria (15 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| molibresibmolibresib 20 mg will be supplied for compassionate use for the identified individual subject. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2020.
Related trials for Solid Tumours
RecruitingPhase 1
A Study of DS-1103a Combination Therapy in Participants With Advanced Solid Tumors
Advanced Solid Tumor
RecruitingPhase 1/2
Docetaxel and SX-682 in Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma, Salivary Gland Carcinoma, and Advanced Prostate Cancer
Head and Neck Cancer, Head and Neck Squamous Cell Carcinoma
Recruiting
Virtual Reality Intervention to Improve Psychological Symptom Burden for Cancer Patients Undergoing Treatment
Adjustment Disorder, Anxiety Disorder
RecruitingPhase 1
Personalized KXV01 Lentinvivo (TCR) Injection as the Therapy for Advanced Solid Tumors
Advanced Solid Tumors
RecruitingObservational study
Obtaining Solid Tumor Tissue From People Having Biopsy or Surgery for Certain Types of Cancer
Colorectal Neoplasms, Gastric Neoplasms
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
GSK Clinical Trials study director
GlaxoSmithKline
Questions about this trial
What is this clinical trial studying?
A study testing molibresib in people with Solid Tumours, run by GlaxoSmithKline.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 13 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2020.