SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study

NCT ID: NCT02967679

Last Updated: 2020-11-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-12-05
• Study Completion Date: 2019-03-18

Brief Summary

The single-center, open-label Phase II study has the objective of assess the effect of MD1003 on motor and sensory conduction in patients suffering from demyelinating polyneuropathies in 15 subjects.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy; Peripheral Neuropathy; Charcot-Marie-Tooth Disease; Charcot-Marie-Tooth Disease Type 1A; Charcot-Marie-Tooth Disease, Type 1B; Anti-MAG Neuropathy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

MD1003

MD1003 100mg capsules, 1 capsule tid for 48 weeks

Group Type: EXPERIMENTAL

MD1003

Intervention Type: DRUG

Interventions

MD1003

Intervention Type: DRUG

Other Intervention Names

High Dose Biotin

Eligibility Criteria

Inclusion Criteria

• Male and female aged between 20 and 85 years.
• Patients fulfilling one of the following diagnosis:
• Five patients with chronic inflammatory demyelinating polyneuropathy on both clinical and neurophysiological grounds.
• Five patients with proven genetic diagnosis of CMT1a or CMT1b
• Five patients with anti-MAG polyneuropathy.
• Electrophysiological parameters worsening for the past 3 years
• Available EMG record, performed during the past 6 months to assess variability of NCV parameters
• Signed and dated written informed consent to participate in the study in accordance with local regulations
• Likely to be able to participate in all scheduled evaluation and complete all required study procedures,
• In the opinion of the investigator, the patient will be compliant and have a high probability of completing the study.
• Both male and female subjects who are not either surgically sterile (tubal ligation/obstruction or removal of ovaries or uterus) or post-menopausal (no spontaneous menstrual periods for at least one year confirmed by a negative hormone panel) must commit to using TWO highly effective method of birth control for the duration of the study and for two months after the treatment termination.

Exclusion Criteria

• Any general chronic handicapping disease other than peripheral neuropathy
• Impossibility to perform the 10 meters walking test
• Impossibility to assess electrophysiological parameters
• Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or cancer,
• Patients with hypersensitivity to MD1003 excipients (lactose)
• Laboratory tests out of normal range according to the reference laboratory values. Deviations may be accepted if considered by the investigator as not clinically significant with regards to the study continuation,
• Patients with history or presence of alcohol abuse or drug addiction,
• Patients likely to be non-compliant to the study procedures or for whom a long-term follow-up seems to be difficult to achieve.
• Any new medication for neuropathy initiated less than 3 months prior to inclusion. For CIDP patients, relapse in the past 3 months before inclusion.
• Not easily contactable by the investigator in case of emergency or not capable to call the investigator
• Subjects without effective contraception

• Minimum Eligible Age: 20 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

MedDay Pharmaceuticals SA

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alain CREANGE, MD

Role: PRINCIPAL_INVESTIGATOR

Hôpital Henri Mondor, Créteil, France

Frederic Sedel, MD

Role: STUDY_DIRECTOR

Medday Pharmaceuticals

Locations

Hôpital Henri Mondor, Créteil, France

Créteil, , France

Countries

France

References

Creange A, Hutin E, Sedel F, Le Vigouroux L, Lefaucheur JP. High-dose pharmaceutical-grade biotin in patients with demyelinating neuropathies: a phase 2b open label, uncontrolled, pilot study. BMC Neurol. 2023 Oct 30;23(1):389. doi: 10.1186/s12883-023-03440-y.

Reference Type: DERIVED

PMID: 37899433 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

MD1003CT2015-01 SERENDEM

Identifier Type: -

Identifier Source: org_study_id