MD1003-AMN MD1003 in Adrenomyeloneuropathy

Sponsor

MedDay Pharmaceuticals SA (Industry)

Overall Status

Completed

CT.gov ID

NCT02961803

Collaborator

(none)

Enrollment

Locations

Arms

Duration (Months)

16.8

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Adrenomyeloneuropathy Adrenoleukodystrophy AMN	Drug: MD1003 100 mg capsule Drug: Placebo	Phase 2/Phase 3

Detailed 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.

Study Design

Study Type:

Interventional

Actual Enrollment :

67 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study

Study Start Date :

Oct 1, 2014

Actual Primary Completion Date :

Jun 1, 2016

Actual Study Completion Date :

Jun 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: MD1003 MD1003 100mg capsules, 1 capsule tid for 24 months	Drug: MD1003 100 mg capsule Other Names: High Dose Biotin
Placebo Comparator: Placebo Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months	Drug: MD1003 100 mg capsule Other Names: High Dose Biotin Drug: Placebo

Arm

Intervention/Treatment

Experimental: MD1003

MD1003 100mg capsules, 1 capsule tid for 24 months

Drug: MD1003 100 mg capsule

Other Names:

High Dose Biotin

Placebo Comparator: Placebo

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

Drug: MD1003 100 mg capsule

Other Names:

High Dose Biotin

Drug: Placebo

Outcome Measures

Primary Outcome Measures

Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline [Baseline and 12 Months]

Secondary Outcome Measures

Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20% [Baseline, 9 months, 12 months]
at Months 9 and Months 12 compared to the best value among screening and baseline.
Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20% [Baseline, 9 months, 12 months]
at Months 9 and Months 12 compared to the best value among screening and baseline
Mean Change in TW25 (time to walk 25 feet) [Baseline and 12 months]
Timed up and Go test (TUG) [12 Months]
Euroqol EQ-5D questionnaire [12 months]
Quality of Life questionnaire
Qualiveen Questionnaire [12 Months]
Qualiveen to evaluate urinary function

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

Male

Accepts 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

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Hôpital Bicêtre	Le Kremlin Bicêtre	France	94275 Cedex
2	Groupe Hospitalier Pitié-Salpêtrière	Paris	France	75651
3	MS-Ambulanz Fachkrankenhaus Hubertusburg	Wermsdorf	Germany	04779
4	Hospital Duran i Reynals / Bellvitge	Barcelona	Spain	08908