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MD1003-AMN MD1003 in Adrenomyeloneuropathy

NCT ID: NCT02961803

Last Updated: 2017-10-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

67 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-10-31

Study Completion Date

2017-06-30

Brief Summary

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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

Detailed Description

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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.

Conditions

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Adrenomyeloneuropathy Adrenoleukodystrophy AMN

Keywords

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AMN MD1003 Adrenomyeloneuropathy ALD Adrenoleukodystrophy Biotin 2MWT Two-minute walk test TW25 Timed 25-Foot Walk

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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MD1003

MD1003 100mg capsules, 1 capsule tid for 24 months

Group Type EXPERIMENTAL

MD1003 100 mg capsule

Intervention Type DRUG

Placebo

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

Group Type PLACEBO_COMPARATOR

MD1003 100 mg capsule

Intervention Type DRUG

Placebo

Intervention Type DRUG

Interventions

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MD1003 100 mg capsule

Intervention Type DRUG

Placebo

Intervention Type DRUG

Other Intervention Names

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High Dose Biotin

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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MedDay Pharmaceuticals SA

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Patrick Aubourg, MD

Role: PRINCIPAL_INVESTIGATOR

Hopital Le Kremlin-Bicêtre

Frederic Sedel, MD

Role: STUDY_DIRECTOR

Medday Pharmeuticals

Locations

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Hôpital Bicêtre

Le Kremlin-Bicêtre, , France

Site Status

Groupe Hospitalier Pitié-Salpêtrière

Paris, , France

Site Status

MS-Ambulanz Fachkrankenhaus Hubertusburg

Wermsdorf, , Germany

Site Status

Hospital Duran i Reynals / Bellvitge

Barcelona, , Spain

Site Status

Countries

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France Germany Spain

Other Identifiers

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MD1003CT2014-01AMN

Identifier Type: -

Identifier Source: org_study_id