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

NCT02967679 | Completed Phase 2 Results

• 1 other identifier: MD1003CT2015-01 SERENDEM
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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

Keywords

Neuropathy; Demyelinating polyradiculoneuropathy

Outcome Measures

Primary Outcomes (4)

• Motor Nerve Conduction Velocity (m/Sec)

  Absolute change from baseline at Week 48.

  48 weeks

• Distal Latency (Msec)

  Absolute change from baseline at Week 48.

  48 weeks

• F Wave Latency (Msec)

  Absolute change from baseline at Week 48.

  48 weeks

• Length of Motor Nerve Potential

  Absolute change from baseline at W48.

  48 weeks

Secondary Outcomes (12)

• ONLS (Overall Neuropathy Limitations Scale)

  48 weeks

• Change From Baseline at Week 48 for Timed 10-meter Walk Test

  48 weeks

• Absolute Change From Baseline at Week 48 for Medical Research Council (MRC) Subscore (Total Muscle) and Total Score

  48 weeks

• INCAT Sensory Sum Score (ISS)

  48 weeks

• 6-minute Walk Test

  48 weeks

Study Arms (1)

MD1003

EXPERIMENTAL

MD1003 100mg capsules, 1 capsule tid for 48 weeks

Drug: MD1003

Interventions

MD1003 DRUG

Also known as: High Dose Biotin

MD1003

Eligibility Criteria

• Age: 20 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Not easily contactable by the investigator in case of emergency or not capable to call the investigator
• Subjects without effective contraception

Sponsors & Collaborators

• MedDay Pharmaceuticals SA: lead

Study Sites (1)

Hôpital Henri Mondor, Créteil, France

Créteil, 94010, France

Location

Related Publications (1)

• 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.

  PMID: 37899433 DERIVED

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Peripheral Nervous System Diseases; Charcot-Marie-Tooth Disease

Interventions

Biotin

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Coenzymes; Enzymes and Coenzymes

Results Point of Contact

• Title: Dr Frédéric SEDEL, Chief Scientific Officer and Co-founder
• Organization: MedDay Pharmaceuticals

frederic.sedel@medday-pharma.com

+33 1 80 40 14 40

Study Officials

• Alain CREANGE, MD

  Hôpital Henri Mondor, Créteil, France

  PRINCIPAL INVESTIGATOR

• Frederic Sedel, MD

  Medday Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2016
• First Posted: November 18, 2016
• Study Start: December 5, 2016
• Primary Completion: March 18, 2019
• Study Completion: March 18, 2019
• Last Updated: November 2, 2020
• Results First Posted: November 2, 2020

Record last verified: 2020-10

Locations

FR (1)