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

• Status: Completed
• Trial end date: 2019-03-18
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: MedDay Pharmaceuticals SA
• Treatments: Biotin

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