MD1003-AMN MD1003 in Adrenomyeloneuropathy

MedDay Pharmaceuticals

Status and phase: Completed; Phase 3; Phase 2

Conditions: Adrenoleukodystrophy; Adrenomyeloneuropathy; AMN

Treatments: Drug: Placebo; Drug: MD1003 100 mg capsule

Study type: Interventional

Funder types: Industry

Identifiers

NCT02961803

MD1003CT2014-01AMN

Details and patient eligibility

About

The primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy

Full description

AMN and progressive multiple sclerosis share some similarities including progressive spastic paraparesis and secondary energy failure leading to progressive axonal degeneration. Therefore, it was hypothesized that high doses of biotin might be efficient in patients with AMN.

Enrollment: 67 patients

Sex: Male

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• ABCD1 gene mutation identified
• Elevated plasma VLCFA
• Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk
• EDSS score ≥ 3.5 and ≤ 6.5
• Normal brain MRI or brain MRI showing :
• abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4
• and/or stable (≥6 months) cerebral demyelination without gadolinium enhancement with a Loes score ≤12.
• Appropriate steroid replacement if adrenal insufficiency is present
• Likely to be able to participate in all scheduled evaluation visits and complete all required study procedures
• Signed and dated written informed consent to participate in the study in accordance with local regulations
• Affiliated to a Health Insurance

Exclusion criteria

• Brain MRI abnormalities with a Loes score > 12 or with gadolinium enhancement
• Any progressive neurological disease other than AMN
• Impossibility to perform the walk tests and the TUG test
• Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or any progressive malignancy
• Any new medication for AMN including Fampridine initiated less than 1 month prior to inclusion
• Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants.
• Inclusion in another therapeutic clinical trial for ALD
• Not easily contactable by the investigator in case of emergency or not capable to call the investigator

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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67 participants in 2 patient groups, including a placebo group

MD1003

Experimental group

Description:

MD1003 100mg capsules, 1 capsule tid for 24 months

Treatment:

Drug: MD1003 100 mg capsule

Placebo

Placebo Comparator group

Description:

Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months

Treatment:

Drug: MD1003 100 mg capsule

Drug: Placebo