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

• ClinicalTrials.gov Identifier: NCT02967679
• Recruitment Status: Unknown
• First Posted: November 18, 2016
• Last Update Posted: November 18, 2016
• Sponsor: MedDay Pharmaceuticals SA
• Information provided by (Responsible Party): MedDay Pharmaceuticals SA

Study Description

Brief Summary:

The primary objective of the study is to assess the effect of MD1003 on motor and sensory conduction, in patients suffering from demyelinating polyneuropathies.

Condition or disease	Intervention/treatment	Phase
Chronic Inflammatory Demyelinating Polyneuropathy; Peripheral Neuropathy; Charcot-Marie-Tooth Disease; Charcot-Marie-Tooth Disease Type 1A; Charcot-Marie-Tooth Disease, Type IA	Drug: MD1003	Phase 1; Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 15 participants
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Study Start Date: September 2016
• Estimated Primary Completion Date: December 2017

Arms and Interventions

Arm	Intervention/treatment
Experimental: MD1003	Drug: MD1003

Outcome Measures

Primary Outcome Measures :

1. Motor nerve conduction velocity (m/sec) [ Time Frame: 48 weeks ]
   A 10% improvement for 2 out of these 4 criteria, in at least 3 out of 8 nerves will be considered as clinically meaningful.
2. Distal latency (msec) [ Time Frame: 48 weeks ]
   A 10% improvement for 2 out of these 4 criteria, in at least 3 out of 8 nerves will be considered as clinically meaningful.
3. F wave latency (msec) [ Time Frame: 48 weeks ]
   A 10% improvement for 2 out of these 4 criteria, in at least 3 out of 8 nerves will be considered as clinically meaningful.
4. Length of motor nerve potential [ Time Frame: 48 weeks ]
   A 10% improvement for 2 out of these 4 criteria, in at least 3 out of 8 nerves will be considered as clinically meaningful.

Secondary Outcome Measures :

1. ONLS (Overall Neuropathy Limitations Scale) [ Time Frame: 48 weeks ]
   Secondary endpoints will be part of exploratory analyses. They will consist in studying mean change or proportions for the following clinical and electrophysiological parameters.
2. Timed 10-meter walk test [ Time Frame: 48 weeks ]
3. Medical Research Council (MRC) sum score [ Time Frame: 48 weeks ]
4. INCAT Sensory Sum Score (ISS) [ Time Frame: 48 weeks ]
5. 6-minute walk test [ Time Frame: 48 weeks ]
6. Posturometry [ Time Frame: 48 weeks ]
7. Supernormality (%) [ Time Frame: 48 weeks ]
8. Strength-duration time constant (ms) [ Time Frame: 48 weeks ]
9. Rheobase (mA) [ Time Frame: 48 weeks ]
10. Refractoriness (%) [ Time Frame: 48 weeks ]
11. Min-max absolute refractory period (ms) [ Time Frame: 48 weeks ]

Eligibility Criteria

• Ages Eligible for Study: 20 Years to 85 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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
• 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

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.

Contacts and Locations

Contacts

Contact: abdelkarim Bendarraz; contact@medday-pharma.com

Locations

France

Hôpital Henri Mondor, Créteil, France; Recruiting

Creteil, France, 94010

Contact: Alain CREANGE, MD +33149812315 alain.creange@aphp.fr

Sponsors and Collaborators

MedDay Pharmaceuticals SA

Investigators

• Principal Investigator: Alain CREANGE, MD; Hôpital Henri Mondor, Créteil, France
• Study Director: Frederic Sedel, MD; Medday Pharmaceuticals

More Information

Additional Information:

Sponsor 

• Responsible Party: MedDay Pharmaceuticals SA
• ClinicalTrials.gov Identifier: NCT02967679 History of Changes
• Other Study ID Numbers: MD1003CT2015-01-SERENDEM
• First Posted: November 18, 2016
• Last Update Posted: November 18, 2016
• Last Verified: November 2016

Keywords provided by MedDay Pharmaceuticals SA:

Neuropathy; Demyelinating polyradiculoneuropathy

Additional relevant 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; Neuromuscular Diseases; Nervous System Diseases; Stomatognathic Diseases; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases