BridgeMD
AN OPEN-LABEL, MULTICENTER STUDY IN MALE PEDIATRIC PATIENTS WITH CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY (CALD) TO ASSESS THE EFFECTS OF MIN-102 TREATMENT ON DISEASE PROGRESSION PRIOR TO HUMAN STEM CELL TRANSPLANT (HSCT)
EU CTIS ID: 2024-513774-21-00
What this study is testing
To evaluate whether MIN-102 can arrest disease progression of cALD at week 96, as determined by serial clinical and magnetic resonance imaging (MRI) investigations in pediatric subjects.
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Written informed consent by parent/legal guardian, or authorized legal representative to participate in the study
- Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD based on genetic testing; or, in absence of genetic testing, elevation of VLCFA and confirmed by family history of X-ALD with clinical symptoms and elevation of VLCFA or by genetic testing of a family member.
- White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
- Major Functional Disabilities (MFD) score of 0, as determined by key measures in the Neurological Function Scale (NFS).
- Baseline Loes score >0 and ≤10.
- Baseline Gadolinium Intensity Score (GIS) ≤3.
You likely can't join if
- Other chronic neurological disease.
- Permanent indwelling urinary catheter or catheter port. (Only applicable in France)
- Smoking with 25 cigarettes per day over the past 2 years until Screening (V-1). (Only applicable in France).
- Previous or current history of congestive heart failure.
- Clinically significant anemia with hemoglobin <10 g/dL.
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level >2 times the ULN or total bilirubin >1.5 times the ULN (unless due to Gilbert's syndrome).
See the full eligibility criteria
Who can join
- Written informed consent by parent/legal guardian, or authorized legal representative to participate in the study
- Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD based on genetic testing; or, in absence of genetic testing, elevation of VLCFA and confirmed by family history of X-ALD with clinical symptoms and elevation of VLCFA or by genetic testing of a family member.
- White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
- Major Functional Disabilities (MFD) score of 0, as determined by key measures in the Neurological Function Scale (NFS).
- Baseline Loes score >0 and ≤10.
- Baseline Gadolinium Intensity Score (GIS) ≤3.
- No signs or symptoms of adrenal insufficiency and morning cortisol and aldosterone levels within normal laboratory ranges for age, or appropriate steroid replacement if adrenal insufficiency is present. A history of adrenal insufficiency is not exclusionary if the foregoing is currently met.
- Glycated hemoglobin (HbA1c) within normal range.
What rules you out
- Other chronic neurological disease.
- Permanent indwelling urinary catheter or catheter port. (Only applicable in France)
- Smoking with 25 cigarettes per day over the past 2 years until Screening (V-1). (Only applicable in France).
- Previous or current history of congestive heart failure.
- Clinically significant anemia with hemoglobin <10 g/dL.
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level >2 times the ULN or total bilirubin >1.5 times the ULN (unless due to Gilbert's syndrome).
- Moderate or severe hepatic impairment (groups B and C according to Child-Pugh classification).
- eGFR < 90 ml/min or any evidence of renal disease or impairment, including proteinuria or hematuria.
- Pulmonary disease or cardiac disease of sufficient severity to limit participation in the study and/or completion of study procedures.
- Reduced left-ventricular ejection fraction or other clinically significant cardiac abnormalities on echocardiogram that, in the investigator's opinion, could predispose the subject to volume overload or its attendant consequences.
- Hereditary Fructose Intolerance.
- Known intolerance to pioglitazone or other thiazolidinediones.
- History of diabetes, or glycated hemoglobin (HbA1c) levels >6.4% and fasting blood glucose levels ≤ 0.9 times the lower limit of normal and ≥ 1.1 times the upper limit of normal at Screening
- A positive result on laboratory tests for hepatitis B surface antigen, hepatitis C antibody or human immunodeficiency virus antibody. (Only applicable in France)
- Contraindication to MRI procedure, such as presence of ferromagnetic materials (aneurysm clips, pacemaker, intraocular metal, cochlear implant) in the body.
- Conditions that could modify the absorption of the study drug.
- Inability or unwillingness of parent/legal guardian or subject to comply with the study procedures.
- Inability or unwillingness of parent/legal guardian or subject to resume standard of care at a local center once study is complete or criteria for HSCT is met and treatment available.
- Other medical, neurologic, psychiatric, or social condition that, in the opinion of the investigator, is likely to unfavorably alter risk-benefit of study participation, confound interpretation of safety or efficacy results, or interfere with the satisfactory completion of study requirements.
- Use of pioglitazone or other thiazolidinediones within the past 6 months prior to screening.
- Use of biotin at a daily dose of >50 mg per day within the past 3 months prior to screening.
- Current participation in another interventional clinical study or participation in such a study within 6 months prior to screening.
- Previous HSCT.
- Requirement for a prohibited concomitant medication.
- Previous or current history of bladder polyps, bladder cell hyperplasia, or cancer (other than successfully treated basal cell carcinoma).
- Chronic or recurrent symptomatic urinary infections (≥2 per year over the past 2 years until Screening [V-1]). (Only applicable in France)
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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 male, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.