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New treatment option for Primary Biliary Cholangitis (PBC)
Official title A Two-part Study to Investigate the Effects in Adults of Two Doses of Golexanolone in Patients With Primary Biliary Cholangitis (PBC) With Fatigue and Cognitive Dysfunction
ClinicalTrials.gov ID: NCT07304843
What this study is testing
What is golexanolone?
golexanolone is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for primary biliary cholangitis (pbc).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The present phase 1b/2 randomised, double-blind, placebo-controlled, two-part study is designed to evaluate the safety, tolerability, pharmacokinetic characteristics and preliminary efficacy of two dose levels of golexanolone compared with placebo among subjects with a history of non-cirrhotic or Child-Pugh class A cirrhotic Primary Biliary Cholangitis (PBC) with clinically significant fatigue and cognitive symptoms on stable background standard of care (SoC) PBC medication. The objectives of this research study are to assess the safety and tolerability as well the pharmacokinetic (PK) characteristics of golexanolone administered 40 mg BID for 5 days in the target population (part A) and to assess the safety and tolerability, the effects of golexanolone on health-related quality of life (HRQoL), including fatigue, day-time sleepiness and cognitive function as well as Investigator's overall impression of treatment effect of 28 days twice per day (BID) treatment with two dose levels of golexanolone versus placebo (part B).
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Male and female people age ≥ 18 years
- Diagnosis of PBC based on the presence of ≥2 of 3 key disease characteristics
- Clinically significant fatigue defined for the purposes of this study as a PBC-40 fatigue domain score of ≥29 at screening
- Clinically significant cognitive symptoms, defined for the purposes of this study as a PBC-40 cognitive domain ≥16 at screening
- Stable PBC SoC therapy (if any),for at least 3 months prior to randomisation
You likely can't join if
- Child-Pugh class B or C cirrhosis
- Clinical evidence of hepatic decompensation (e.g. current or prior HE, ascites, or variceal bleeding)
- History of hepatocellular carcinoma
- Bilirubin \>1.5 x ULN
- Glomerular filtration rate (GFR) \<35 mL/min/1.73m2
- Low Haemoglobin (HB), i.e. people with moderate/severe anaemia
See the full eligibility criteria
- Male and female people age ≥ 18 years
- Diagnosis of PBC based on the presence of ≥2 of 3 key disease characteristics
- Clinically significant fatigue defined for the purposes of this study as a PBC-40 fatigue domain score of ≥29 at screening
- Clinically significant cognitive symptoms, defined for the purposes of this study as a PBC-40 cognitive domain ≥16 at screening
- Stable PBC SoC therapy (if any),for at least 3 months prior to randomisation
- For all women of childbearing potential (WOCBP) a negative pregnancy test at screening and a negative urine dip-stick pregnancy test at baseline, prior to first dose of IMP
- WOCBP must be willing to use a contraceptive method with a failure rate of \< 1% and agree to continue use of this method for the duration of the study and thereafter for 1 month after the last dosing of the IMP
- Females of non-childbearing potential must have documented tubal ligation or hysterectomy; or be post-menopausal
- Fertile male people must be willing to use condom and assure that their female partner will use contraceptive methods with a failure rate of \< 1%
- Willing and able to give informed consent
- The subject should be judged by the Investigator to be lucid and oriented to person, place, time, and situation when giving the informed consent
- Child-Pugh class B or C cirrhosis
- Clinical evidence of hepatic decompensation (e.g. current or prior HE, ascites, or variceal bleeding)
- History of hepatocellular carcinoma
- Bilirubin \>1.5 x ULN
- Glomerular filtration rate (GFR) \<35 mL/min/1.73m2
- Low Haemoglobin (HB), i.e. people with moderate/severe anaemia
- Low S-B12 or low P-folate
- Evidence of biliary obstruction
- Any positive result on screening for human immunodeficiency virus (HIV), or hepatitis B (serum hepatitis B surface antigen positive)
- Prolonged QTcF (\>500 ms), or any clinically significant abnormality in the resting ECG, as judged by the Investigator (at screening)
- Concomitant disease characterised by chronic fatigue and/or cognitive impairment
- Clinically significant bowel disease, including obstruction, active inflammatory bowel disease, or malabsorption
- Clinically significant sleep apnoea
- An uncontrolled thyroid disorder
- people with a history of or currently active immune disorders (i.e. uncontrolled) other that PBC (including autoimmune disease) and/or diseases requiring immunosuppressive drugs
- Clinical diagnosis of autoimmune hepatitis overlap
- The presence, as judged by the Investigator, of clinically significant concomitant illness which would jeopardise safe participation in the study and /or the interpretation of study findings
- Regular use of prescribed or over the counter (OTC) medications known to cause fatigue or cognitive dysfunction
- Use of prohibited medications within 14 days prior to randomisation
- Anticipated change in PBC medication and/or significant medical or surgical intervention within the duration of the study
- Regular (more than 1 week per month) alcohol consumption in excess of 14 units per week
- Administration of another new chemical entity or has participated in any other clinical study that included drug treatment with the last administration within 3 months prior to administration of IMP in this study
- Females who are pregnant, nursing or actively trying to conceive a child
- Expected inability to swallow the required number of IMP capsules at the applicable dose level
- History of severe allergy/hypersensitivity or on-going allergy/hypersensitivity, as judged by the Investigator
The study team makes the final eligibility decision.
Where it's taking place
- Düsseldorf, Germany
- Leipzig, Germany
- Athens, Greece
- Pátrai, Greece
- Gyula, Hungary
- Kistarcsa, Hungary
- Milan, Italy
- Monza, Italy
- Padova, Italy
- Palermo, Italy
- Roma, Italy
- Rozzano, Italy
- Udine, Italy
- Belgrade, Serbia
- Barcelona, Spain
- Madrid, Spain
- Málaga, Spain
- Pontevedra, Spain
- Santander, Spain
- Seville, Spain
+ 17 more site(s).
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Düsseldorf, Germany; Leipzig, Germany; Athens, Greece; Pátrai, Greece; Gyula, Hungary; Kistarcsa, Hungary and 31 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.