Clinical trial NCT02967679: SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
Official title: SERENDEM Study: MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
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
Lead sponsor: MedDay Pharmaceuticals SA
Sponsor class: INDUSTRY
First posted: 2016-11-18
Enrollment: 15
Sex: ALL
Minimum age (years): 20
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Motor Nerve Conduction Velocity (m/Sec)
timeFrame: 48 weeks
measure: Distal Latency (Msec)
timeFrame: 48 weeks
measure: F Wave Latency (Msec)
timeFrame: 48 weeks
measure: Length of Motor Nerve Potential
timeFrame: 48 weeks
Secondary outcomes:
measure: ONLS (Overall Neuropathy Limitations Scale)
timeFrame: 48 weeks
measure: Change From Baseline at Week 48 for Timed 10-meter Walk Test
timeFrame: 48 weeks
measure: Absolute Change From Baseline at Week 48 for Medical Research Council (MRC) Subscore (Total Muscle) and Total Score
timeFrame: 48 weeks
measure: INCAT Sensory Sum Score (ISS)
timeFrame: 48 weeks
measure: 6-minute Walk Test
timeFrame: 48 weeks
measure: Posturography Score
timeFrame: 48 weeks
measure: Excitability Testing: Supernormality (%)
timeFrame: 48 weeks
measure: Strength-duration Time Constant (ms)
timeFrame: 48 weeks
measure: Rheobase (mA)
timeFrame: 48 weeks
measure: Refractoriness (%)
timeFrame: 48 weeks
measure: Minimum Absolute Refractory Period (ms).
timeFrame: 48 weeks
measure: Maximum Absolute Refractory Period (ms).
timeFrame: Week 48
Other outcomes:
Locations:
city: Creteil
country: France
facility: Hôpital Henri Mondor, Créteil, France
Study officials:
affiliation: Hôpital Henri Mondor, Créteil, France
name: Alain CREANGE, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Medday Pharmaceuticals
name: Frederic Sedel, MD
role: STUDY_DIRECTOR
References: