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    Clinical Trial Results:
    SERENDEM study: MD1003 in patients suffering from demyelinating neuropathies, an open label pilot study

    Summary
    EudraCT number
    		 2015-001150-15
    Trial protocol
    		 FR  
    Global end of trial date
    18 Mar 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Aug 2020
    First version publication date
    15 Aug 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    "MD1003CT2015-01-SERENDEM
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    MEDDAY SA
    Sponsor organisation address
    24 rue de la Pépinière, Paris, France,
    Public contact
    Frédéric Sedel, MEDDAY PHARMACEUTICALS, frederic.sedel@medday-pharma.com
    Scientific contact
    Frédéric Sedel, MEDDAY PHARMACEUTICALS, frederic.sedel@medday-pharma.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Dec 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    18 Mar 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Mar 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective is to detect a signal of efficacy of high dose biotin in demyelinating polyneuropathies
    Protection of trial subjects
    This protocol complied with the principal laid down by the 18th World Medical Assembly (Helsinki, 1964 an following amendments) and all applicable amendments laid down by the World Medical Assemblies, as well as the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. The trial complied with the laws and regulations of the country in which the study was performed, and any applicable guidelines.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    05 Dec 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 15
    Worldwide total number of subjects
    15
    EEA total number of subjects
    15
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    10
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 15
    Number of subjects completed
    		 15

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Single arm
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    MD1003
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    MD1003 capsules consisting of 100 mg biotin and excipients (lactose, magnesium stearate, croscarmellose sodium, silica) taken orally three times a day (one in the morning, one at noon, and one in the evening)

    Number of subjects in period 1
    		Single arm
    Started
    15
    Completed
    14
    Not completed
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 15 15
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        median (full range (min-max))
    		 62.1 (30.8 to 83.5) -
    Gender categorical
    Units: Subjects
        Female
    		 8 8
        Male
    		 7 7
    Subject analysis sets

    Subject analysis set title
    FAS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All patients who received at least one dose of study medication and with at least one assessment at baseline and during the study will be included in Full Analysis Set.

    Subject analysis sets values
    		 FAS
    Number of subjects
    15
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        median (full range (min-max))
    62.1 (30.8 to 83.5)
    Gender categorical
    Units: Subjects
        Female
    8
        Male
    7

    End points

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    End points reporting groups
    Reporting group title
    Single arm
    Reporting group description
    		 -

    Subject analysis set title
    FAS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All patients who received at least one dose of study medication and with at least one assessment at baseline and during the study will be included in Full Analysis Set.

    Primary: Number of patients with at least 10% improvement for at least 2 out of the 4 criteria of demyelination, in at least 3 out of 8 nerves

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    End point title
    		 Number of patients with at least 10% improvement for at least 2 out of the 4 criteria of demyelination, in at least 3 out of 8 nerves [1]
    End point description
    End point type
    Primary
    End point timeframe
    Baseline to end of study (week 48)
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The primary endpoint, for the primary analysis, was summarized as the number and percentage of patients with a clinical significant relative change (at least 10%) for at least two out of the four criteria in at least three nerves out of the eight investigated by disease group and overall on the FAS population.
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
    1
    1
    No statistical analyses for this end point

    Secondary: ONLS absolute change

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    End point title
    		 ONLS absolute change
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
        arithmetic mean (full range (min-max))
    -0.5 (-3 to 2)
    -0.5 (-3 to 2)
    No statistical analyses for this end point

    Secondary: Timed 10-meter walk test absolute change

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    End point title
    		 Timed 10-meter walk test absolute change
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
        arithmetic mean (full range (min-max))
    -0.6 (-2 to 2)
    -0.6 (-2 to 2)
    No statistical analyses for this end point

    Secondary: MRC total score absolute change

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    End point title
    		 MRC total score absolute change
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to Week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
        arithmetic mean (full range (min-max))
    4.5 (-8 to 27)
    4.5 (-8 to 27)
    No statistical analyses for this end point

    Secondary: INCAT sensory sum score absolute change

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    End point title
    		 INCAT sensory sum score absolute change
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
        arithmetic mean (full range (min-max))
    -2.2 (-8 to 4)
    -2.2 (-8 to 4)
    No statistical analyses for this end point

    Secondary: Distance 6-min walk test absolute change

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    End point title
    		 Distance 6-min walk test absolute change
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
        arithmetic mean (full range (min-max))
    61.1 (-15 to 151)
    61.1 (-15 to 151)
    No statistical analyses for this end point

    Secondary: Posturography: speed absolute change in spontaneous speed condition

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    End point title
    		 Posturography: speed absolute change in spontaneous speed condition
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    13
    13
    Units: none
        arithmetic mean (full range (min-max))
    0.111 (-0.2 to 0.45)
    0.111 (-0.2 to 0.45)
    No statistical analyses for this end point

    Secondary: Excitability testing: strengh-duration time constant absolute change

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    End point title
    		 Excitability testing: strengh-duration time constant absolute change
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
        arithmetic mean (full range (min-max))
    0.068 (-0.11 to 0.28)
    0.068 (-0.11 to 0.28)
    No statistical analyses for this end point

    Secondary: Excitability testing: rheobase absolute change

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    End point title
    		 Excitability testing: rheobase absolute change
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to week 48
    End point values
    		 Single arm FAS
    Number of subjects analysed
    15
    15
    Units: none
        arithmetic mean (full range (min-max))
    -5.905 (-18.29 to 0.67)
    -5.905 (-18.29 to 0.67)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Screening to 30 days after last study drug intake
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Overall - safety set
    Reporting group description
    		 -

    Serious adverse events
    		 Overall - safety set
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 15 (13.33%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Clear cell renal cell carcinoma
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Autoimmune encephalopathy
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Overall - safety set
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    14 / 15 (93.33%)
    Investigations
    Laboratory test interference
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    2
    Haemoglobin decreased
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Ankle fracture
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Burn oesophageal
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Foot fracture
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Surgical and medical procedures
    Breast reconstruction
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Nervous system disorders
    Insomnia
         subjects affected / exposed
    3 / 15 (20.00%)
         occurrences all number
    3
    BALANCE DISORDER
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Memory impairment
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    MUSCLE CONTRACTIONS INVOLUNTARY
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Neuralgia
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    2
    Restless legs syndrome
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Sciatica
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    3 / 15 (20.00%)
         occurrences all number
    3
    Oedema peripheral
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Gastric disorder
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Sleep apnoea syndrome
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    2
    Alopecia
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Rash papular
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    3 / 15 (20.00%)
         occurrences all number
    4
    Muscle spasms
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Musculoskeletal stiffness
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Pain in extremity
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    10 Feb 2017
    Protocol was amended to allow for the inclusion of patients with CMT1b in addition to patients with CMT1a.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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