BridgeMD
A clinical study to assess the efficacy and safety of leriglitazone in adult male subjects with cerebral adrenoleukodystrophy.
EU CTIS ID: 2024-515104-39-00
What this study is testing
To evaluate the efficacy of leriglitazone compared to placebo at increasing survival
- Therapeutic confirmatory (Phase III)
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
- Subject is able to read and understand the ICF and has provided written informed consent to participate in the study.
- Subject is surgically sterilized. If subject is not surgically sterilized, they must be willing to use adequate contraception when engaging in sexual intercourse with a partner who is pregnant or has the potential to become pregnant, and not donate sperm from the first dose of the study drug until at least 90 days after the last dose. Adequate contraception is defined as a diaphragm or cervical cap, or a male condom; this should be combined with hormonal contraceptives or an intrauterine device for a nonpregnant partner with the potential to become pregnant. Total abstinence, in accordance with the lifestyle of the subject, is also acceptable.
- Subject is male and aged ≥18 years.
- Subject has genetic confirmation of X-ALD.
- Subject has progressive cALD, defined as GdE+ brain lesions.
- Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT.
You likely can't join if
- Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel).
- Subject with chronic kidney disease (CKD) of stage 3 or higher (according to the Renal Association CKD staging)
- Subject has previous or current history of cancer, unless surgically resected and without evidence of recurrence for a minimum of 5 years.
- Subject has contraindications for MRI such as having paramagnetic material in the body (e.g., aneurysm clips, pacemakers, intraocular metal, or cochlear implants).
- Subject with conditions that could modify absorption of the study drug.
- Subject with current participation in another interventional clinical study or within 1 month (or within five half-lives, whichever is longer) prior to Screening.
See the full eligibility criteria
Who can join
- Subject is able to read and understand the ICF and has provided written informed consent to participate in the study.
- Subject is surgically sterilized. If subject is not surgically sterilized, they must be willing to use adequate contraception when engaging in sexual intercourse with a partner who is pregnant or has the potential to become pregnant, and not donate sperm from the first dose of the study drug until at least 90 days after the last dose. Adequate contraception is defined as a diaphragm or cervical cap, or a male condom; this should be combined with hormonal contraceptives or an intrauterine device for a nonpregnant partner with the potential to become pregnant. Total abstinence, in accordance with the lifestyle of the subject, is also acceptable.
- Subject is male and aged ≥18 years.
- Subject has genetic confirmation of X-ALD.
- Subject has progressive cALD, defined as GdE+ brain lesions.
- Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT.
- Subject has a Loes score ≥0.5 and ≤12 at Screening.
- Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), other than “wheelchair bound” or “total incontinence”, which will be allowed as these are considered expected symptoms of AMN in the time course of the disease
- Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening
- Subject has normal adrenal function or appropriate steroid replacement if adrenal insufficiency is present.
What rules you out
- Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel).
- Subject with chronic kidney disease (CKD) of stage 3 or higher (according to the Renal Association CKD staging)
- Subject has previous or current history of cancer, unless surgically resected and without evidence of recurrence for a minimum of 5 years.
- Subject has contraindications for MRI such as having paramagnetic material in the body (e.g., aneurysm clips, pacemakers, intraocular metal, or cochlear implants).
- Subject with conditions that could modify absorption of the study drug.
- Subject with current participation in another interventional clinical study or within 1 month (or within five half-lives, whichever is longer) prior to Screening.
- Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.
- Subject has known type 1 or type 2 diabetes.
- Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione or any of the ingredients of the study drug or placebo.
- Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening.
- Subject has a requirement for treatment with a prohibited concomitant medication.
- Subject has a previous or current history of congestive heart failure.
- Subject has reduced left-ventricular ejection fraction < 50% or other clinically significant cardiac abnormalities on echocardiogram (including ventricular dilatation; aortic or mitral stenosis greater than or equal to moderate; or ventricular wall abnormality) that in the opinion of the investigator could predispose the subject to volume overload or its associated consequences.
- Subjects with clinically significant anemia (hemoglobin <12.5 g/dL), abnormal liver enzyme tests for aspartate transaminase (AST) or alanine transaminase (ALT) of >2.5 × the upper limit of normal (ULN) at Screening.
- Subject has moderate or severe hepatic impairment (Child-Pugh classification groups B or C).
The study team makes the final eligibility decision.
Where it's taking place
- Argentina
- Brazil
- India
- United States
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, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Argentina; Brazil; India; United States. Enter your location above to see the nearest site and check your eligibility.
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.