Phase 2
N=15
SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
Chronic Inflammatory Demyelinating Polyneuropathy · Peripheral Neuropathy · Charcot-Marie-Tooth Disease · Charcot-Marie-Tooth Disease Type 1A · Charcot-Marie-Tooth Disease, Type 1B
Bottom Line
View on ClinicalTrials.gov: NCT02967679 ↗Enrolled (actual)
15
Serious AEs
13.3%
Results posted
Nov 2020
Primary outcome: Primary: Motor Nerve Conduction Velocity (m/Sec) — 1.67 m/s — p=0.7615
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- MD1003 (Drug)
- Age
- Adult, Older Adult · 20+ yrs
- Sex
- All
- Sponsor
- MedDay Pharmaceuticals SA
- Primary completion
- Mar 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Motor Nerve Conduction Velocity (m/Sec) |
1.67 | 0.7615 |
| PRIMARY Distal Latency (Msec) |
0.20 | 0.5995 |
| PRIMARY F Wave Latency (Msec) |
4.8 | 0.1641 |
| PRIMARY Length of Motor Nerve Potential |
8.5 | 0.0353 sig |
| SECONDARY ONLS (Overall Neuropathy Limitations Scale) |
-0.5 | 0.2642 |
| SECONDARY Change From Baseline at Week 48 for Timed 10-meter Walk Test |
-0.6 | 0.1777 |
| SECONDARY Absolute Change From Baseline at Week 48 for Medical Research Council (MRC) Subscore (Total Muscle) and Total Score |
4.5 | 0.0371 sig |
| SECONDARY INCAT Sensory Sum Score (ISS) |
-2.2 | 0.0142 sig |
| SECONDARY 6-minute Walk Test |
61.1 | — |
| SECONDARY Posturography Score |
0.111 | 0.1055 |
| SECONDARY Excitability Testing: Supernormality (%) |
9.84 | 0.2130 |
| SECONDARY Strength-duration Time Constant (ms) |
0.068 | 0.0393 sig |
| SECONDARY Rheobase (mA) |
-5.905 | 0.0004 sig |
| SECONDARY Refractoriness (%) |
-4.28 | 0.3303 |
| SECONDARY Minimum Absolute Refractory Period (ms). |
0.000 | 0.9229 |
| SECONDARY Maximum Absolute Refractory Period (ms). |
-0.277 | — |
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
Data sourced from ClinicalTrials.gov (NCT02967679). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.