Trial Outcomes & Findings for SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study (NCT NCT02967679)
NCT ID: NCT02967679
Last Updated: 2020-11-02
Results Overview
Absolute change from baseline at Week 48.
COMPLETED
PHASE2
15 participants
48 weeks
2020-11-02
Participant Flow
Participant milestones
| Measure |
MD1003
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
14
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
MD1003
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Age, Continuous
|
61.36 years
STANDARD_DEVIATION 14.07 • n=15 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=15 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=15 Participants
|
|
Region of Enrollment
France
|
15 participants
n=15 Participants
|
|
Disease type
CIDP patients
|
5 participants
n=15 Participants
|
|
Disease type
CMT1a or CMT1b patients
|
5 participants
n=15 Participants
|
|
Disease type
anti-MAG polyneuropathy
|
5 participants
n=15 Participants
|
PRIMARY outcome
Timeframe: 48 weeksAbsolute change from baseline at Week 48.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Motor Nerve Conduction Velocity (m/Sec)
|
1.67 m/s
Standard Deviation 11.45
|
PRIMARY outcome
Timeframe: 48 weeksAbsolute change from baseline at Week 48.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Distal Latency (Msec)
|
0.20 ms
Standard Deviation 8.89
|
PRIMARY outcome
Timeframe: 48 weeksAbsolute change from baseline at Week 48.
Outcome measures
| Measure |
MD1003
n=9 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
F Wave Latency (Msec)
|
4.8 ms
Standard Deviation 8.40
|
PRIMARY outcome
Timeframe: 48 weeksAbsolute change from baseline at W48.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Length of Motor Nerve Potential
|
8.5 ms
Standard Deviation 16.50
|
SECONDARY outcome
Timeframe: 48 weeksThe ONLS focuses on upper and lower limb functions, and consists of a checklist for interviewing patients. It is scored from 0 to 5 on the upper limb section and from 0 to 7 on the lower limb section. A score of 0 indicates no limitations (the ceiling of the scale) and a score of 5 or 7 indicates no purposeful movement. Absolute change from baseline at week 48.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
ONLS (Overall Neuropathy Limitations Scale)
|
-0.5 score on a scale
Standard Deviation 1.4
|
SECONDARY outcome
Timeframe: 48 weeksAbsolute change from baseline at week 48. The patient is instructed to walk at normal pace for 10 meters. Start and stop of performance time coincides with the toes of the leading foot crossing the 2-m mark and the 8-m mark, respectively. From these data, the speed may be calculated by dividing the middle 6 m by the time (in seconds).
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Change From Baseline at Week 48 for Timed 10-meter Walk Test
|
-0.6 second
Standard Deviation 1.2
|
SECONDARY outcome
Timeframe: 48 weeksMRC score assessed in 19 muscles. The muscle scale grades muscle power on a scale of 0 to 5 in relation to the maximum expected for that muscle. The patient's effort is graded on a scale of 0-5: Grade 5: Muscle contracts normally against full resistance. Grade 4: Muscle strength is reduced but muscle contraction can still move joint against resistance. Grade 3: Muscle strength is further reduced such that the joint can be moved only against gravity with the examiner's resistance completely removed. As an example, the elbow can be moved from full extension to full flexion starting with the arm hanging down at the side. Grade 2: Muscle can move only if the resistance of gravity is removed. As an example, the elbow can be fully flexed only if the arm is maintained in a horizontal plane. Grade 1: Only a trace or flicker of movement is seen or felt in the muscle or fasciculations are observed in the muscle. Grade 0: No movement is observed.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Absolute Change From Baseline at Week 48 for Medical Research Council (MRC) Subscore (Total Muscle) and Total Score
|
4.5 score on a scale
Standard Deviation 8.2
|
SECONDARY outcome
Timeframe: 48 weeksThis sensory scale comprises pin prick and vibration sense plus a two point discrimination value in the arms and legs, and ranges from 0 ("normal sensation") to 20 ("most severe sensory deficit"). Absolute change from baseline at week 48.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
INCAT Sensory Sum Score (ISS)
|
-2.2 score on a scale
Standard Deviation 2.9
|
SECONDARY outcome
Timeframe: 48 weeksThe 6MWT is a practical simple test that requires a 30 m (100-ft) hallway. This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes. Absolute change from baseline at week 48.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
6-minute Walk Test
|
61.1 m
Standard Deviation 48.9
|
SECONDARY outcome
Timeframe: 48 weeksComputerized dynamic posturography (CDP) is a non-invasive specialized clinical assessment technique used to quantify the central nervous system adaptive mechanisms (sensory, motor and central) involved in the control of posture and balance, both in normal and abnormal conditions. Absolute change of speed in spontaneous speed condition from baseline at week 48.
Outcome measures
| Measure |
MD1003
n=13 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Posturography Score
|
0.111 m/s
Standard Deviation 0.201
|
SECONDARY outcome
Timeframe: 48 weeksNerve excitability testing is a non-invasive approach in investigating the pathophysiology of peripheral nerve disorders, which determines the electrical properties of the nerve membrane at the site of stimulation. Absolute change from baseline at week 48. After a nerve fiber is depolarized, a sequence of excitability changes, called the 'recovery cycle', occurs before the membrane potential returns to its resting stage. This cycle includes phases in which the nerve excitability is decreased ('refractory period' or increased ('supernormal period'). During the 10-30 ms following the end of the refractory period, the axon increases its excitability and the nerve fiber is more easily excited (the supernormal period). Depolarization of the node of Ranvier excites the adjacent internodes, which then charge with electric current as capacitors. Supernormality depends on many factors and its interpretation is therefore not univoqual. Data were provided for information only.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Excitability Testing: Supernormality (%)
|
9.84 percentage of supernormality
Standard Deviation 28.16
|
SECONDARY outcome
Timeframe: 48 weeksThis secondary outcome measure is an electrophysiological testing endpoint. Absolute change from baseline at week 48. Strength-duration Time Constant (ms) is a measurement of excitability, defined as the duration of the stimulus that has twice the strength of the rheobase current (see below). The lower the rheobase is, the higher is the Strenght duration time constant. Accordingly, higer values of SDTC are associated with better outcome.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Strength-duration Time Constant (ms)
|
0.068 ms
Standard Deviation 0.112
|
SECONDARY outcome
Timeframe: 48 weeksThis secondary outcome measure is an electrophysiological testing endpoint. Absolute change from baseline at week 48. Rheobase is the minimal strength of an electrical stimulus of infinitely long duration that is able to cause excitation. Low values are associated with better outcome (the nerve becomes more excitable).
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Rheobase (mA)
|
-5.905 mA
Standard Deviation 6.283
|
SECONDARY outcome
Timeframe: 48 weeksThis secondary outcome measure is an electrophysiological testing endpoint. Absolute change from baseline to week 48. After a nerve fiber is depolarized, a sequence of excitability changes, called the 'recovery cycle', occurs before the membrane potential returns to its resting stage. This cycle includes phases in which the nerve excitability is decreased ('refractory period' or increased ('supernormal period'). Refractoriness depends on many factors and its interpretation is therefore not univoqual. Data were provided for information only.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Refractoriness (%)
|
-4.28 percentage of refractoriness
Standard Deviation 32.21
|
SECONDARY outcome
Timeframe: 48 weeksThis secondary outcome measure is an electrophysiological testing endpoint. Absolute change from baseline at week 48. After a nerve fiber is depolarized, a sequence of excitability changes, called the 'recovery cycle', occurs before the membrane potential returns to its resting stage. This cycle includes phases in which the nerve excitability is decreased ('refractory period' or increased ('supernormal period'). Refractoriness depends on many factors and its interpretation is therefore not univoqual. Data were provided for information only.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Minimum Absolute Refractory Period (ms).
|
0.000 ms
Standard Deviation 0.092
|
SECONDARY outcome
Timeframe: Week 48This secondary outcome measure is an electrophysiological testing endpoint. Absolute change from baseline at week 48. After a nerve fiber is depolarized, a sequence of excitability changes, called the 'recovery cycle', occurs before the membrane potential returns to its resting stage. This cycle includes phases in which the nerve excitability is decreased ('refractory period' or increased ('supernormal period'). Refractoriness depends on many factors and its interpretation is therefore not univoqual. Data were provided for information only.
Outcome measures
| Measure |
MD1003
n=15 Participants
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Maximum Absolute Refractory Period (ms).
|
-0.277 ms
Standard Deviation 0.871
|
Adverse Events
MD1003
Serious adverse events
| Measure |
MD1003
n=15 participants at risk
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Clear cell renal cell carcinoma
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Autoimmune encephalopathy
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
Other adverse events
| Measure |
MD1003
n=15 participants at risk
MD1003 100mg capsules, 1 capsule tid for 48 weeks
|
|---|---|
|
Nervous system disorders
Insomnia
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
Gastrointestinal disorders
Gastric disorder
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
balance disorder
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Memory impairment
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Muscle contractions involuntary
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Neuralgia
|
6.7%
1/15 • Number of events 2 • 52 weeks
|
|
Nervous system disorders
Restless legs syndrome
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Nervous system disorders
Sciatica
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
arthralgia
|
20.0%
3/15 • Number of events 4 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
General disorders
Fatigue
|
20.0%
3/15 • Number of events 3 • 52 weeks
|
|
General disorders
Oedema peripheral
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Diarrhoea
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Nausea
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Burn oesophageal
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Injury, poisoning and procedural complications
Foot fracture
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Investigations
Laboratory test interference
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Investigations
Haemoglobin decreased
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
13.3%
2/15 • Number of events 2 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Infections and infestations
Urinary tract infection
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Psychiatric disorders
Depression
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Surgical and medical procedures
Breast reconstruction
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
|
Vascular disorders
Hypertension
|
6.7%
1/15 • Number of events 1 • 52 weeks
|
Additional Information
Dr Frédéric SEDEL, Chief Scientific Officer and Co-founder
MedDay Pharmaceuticals
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place