SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
Condition: 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
MeSH terms: Tooth Diseases; Peripheral Nervous System Diseases; Polyneuropathies; Charcot-Marie-Tooth Disease; Nerve Compression Syndromes; Hereditary Sensory and Motor Neuropathy; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
Intervention: MD1003
Intervention MeSH terms: Biotin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 15.0
Sex: All
Minimum age: 20 Years
Maximum age: 85 Years
Healthy volunteers: No
Keywords: Neuropathy; Demyelinating polyradiculoneuropathy

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.
    

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