Trial on patients with adrenomyeloneuropathy to assess PXL770's behaviour, a new potential medicine, in the body
- Conditions
- Adrenomyeloneuropathy (AMN)Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2021-006223-18-DE
- Lead Sponsor
- Poxel S.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 24
1. Male subjects with either a confirmed diagnosis of AMN by genetic testing or a family history of ALD together with an elevation in VLCFA obtained from overnight fasting plasma sample at V1 (Screening Visit).
2. Age: = 18 to = 65 years at informed consent signature.
3. Normal brain MRI or brain MRI showing non-specific abnormalities that can be observed in AMN subjects without signs of C-ALD
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2
Target disease exclusions
1. Any progressive neurological disease other than AMN.
2. Arrested or progressing C-ALD as defined by cerebral lesions.
3. Prior receipt of an allogeneic hematopoietic stem cell transplant or gene therapy.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method