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    Clinical Trial Results:
    ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period.

    Summary
    EudraCT number
    2019-002754-22
    Trial protocol
    IE   GB   DE   AT   DK   PT  
    Global end of trial date

    Results information
    Results version number
    v1(current)
    This version publication date
    11 Apr 2023
    First version publication date
    11 Apr 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TCC-201
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04085523
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Ascendis Pharma Growth Disorders A/S
    Sponsor organisation address
    Tuborg Blvd 12, Hellerup, Denmark, DK 2900
    Public contact
    Clinical Trial Information Desk, Ascendis Pharma A/S, 0045 70222244, clinhelpdesk@ascendispharma.com
    Scientific contact
    Clinical Trial Information Desk, Ascendis Pharma A/S, 0045 70222244, clinhelpdesk@ascendispharma.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
    Interim
    Date of interim/final analysis
    20 Mar 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    27 Sep 2022
    Global end of trial reached?
    No
    General information about the trial
    Main objective of the trial
    In prepubertal children with achondroplasia (ACH) at 52 weeks • To determine the safety of once weekly subcutaneous (SC) doses of TransCon CNP • To evaluate the effect of once weekly SC doses of TransCon CNP on annualized height velocity (AHV)
    Protection of trial subjects
    Written informed consent was obtained from all subjects prior to enrollment into the trial, as dictated by the Declaration of Helsinki.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    01 May 2020
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety, Efficacy
    Long term follow-up duration
    2 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 9
    Country: Number of subjects enrolled
    New Zealand: 2
    Country: Number of subjects enrolled
    United States: 29
    Country: Number of subjects enrolled
    Austria: 3
    Country: Number of subjects enrolled
    Denmark: 6
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Ireland: 6
    Country: Number of subjects enrolled
    Portugal: 1
    Worldwide total number of subjects
    57
    EEA total number of subjects
    17
    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)
    57
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    Overall, 57 subjects were enrolled and dosed. Enrollment of subjects occurred in eight countries: Australia, Austria, Denmark, Germany, Ireland, Portugal, New Zealand, and the United States.

    Pre-assignment
    Screening details
    A total of 60 subjects were screened and 57 of these met eligibility criteria and were enrolled into the study.

    Period 1
    Period 1 title
    52 Week Blinded Treatment Period (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Investigator, Monitor, Data analyst, Carer, Assessor, Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    TransCon CNP (6 mcg/kg/wk)
    Arm description
    Once weekly subcutaneous administration of TransCon CNP 6 mcg CNP/kg/week
    Arm type
    Experimental

    Investigational medicinal product name
    TransCon CNP
    Investigational medicinal product code
    ACP-015
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    TransCon CNP 3.9 mg CNP-38/vial drug product is a lyophilized powder in a single-use glass vial. Prior to use, the lyophilizate is reconstituted with sterile Water for Injection from a prefilled syringe.

    Arm title
    TransCon CNP (20 mcg/kg/wk)
    Arm description
    Once weekly subcutaneous administration of TransCon CNP 20 mcg CNP/kg/week
    Arm type
    Experimental

    Investigational medicinal product name
    TransCon CNP
    Investigational medicinal product code
    ACP-015
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    TransCon CNP 3.9 mg CNP-38/vial drug product is a lyophilized powder in a single-use glass vial. Prior to use, the lyophilizate is reconstituted with sterile Water for Injection from a prefilled syringe.

    Arm title
    TransCon CNP (50 mcg/kg/wk)
    Arm description
    Once weekly subcutaneous administration of TransCon CNP 50 mcg CNP/kg/week
    Arm type
    Experimental

    Investigational medicinal product name
    TransCon CNP
    Investigational medicinal product code
    ACP-015
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    TransCon CNP 3.9 mg CNP-38/vial drug product is a lyophilized powder in a single-use glass vial. Prior to use, the lyophilizate is reconstituted with sterile Water for Injection from a prefilled syringe.

    Arm title
    TransCon CNP (100 mcg/kg/wk)
    Arm description
    Once weekly subcutaneous administration of TransCon CNP 100 mcg CNP/kg/week
    Arm type
    Experimental

    Investigational medicinal product name
    TransCon CNP
    Investigational medicinal product code
    ACP-015
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    TransCon CNP 3.9 mg CNP-38/vial drug product is a lyophilized powder in a single-use glass vial. Prior to use, the lyophilizate is reconstituted with sterile Water for Injection from a prefilled syringe.

    Arm title
    Pooled Placebo
    Arm description
    Once weekly subcutaneous administration of placebo for TransCon CNP to mimick the dose (6, 20, 50, or 100 mcg/kg/week) of investigational product
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo for TransCon CNP is a lyophilized powder in a single-use glass vial. Prior to use, the lyophilizate is reconstituted with sterile Water for Injection from a prefilled syringe.

    Number of subjects in period 1
    TransCon CNP (6 mcg/kg/wk) TransCon CNP (20 mcg/kg/wk) TransCon CNP (50 mcg/kg/wk) TransCon CNP (100 mcg/kg/wk) Pooled Placebo
    Started
    10
    11
    10
    11
    15
    Completed
    10
    11
    10
    11
    15

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    TransCon CNP (6 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 6 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (20 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 20 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (50 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 50 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (100 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 100 mcg CNP/kg/week

    Reporting group title
    Pooled Placebo
    Reporting group description
    Once weekly subcutaneous administration of placebo for TransCon CNP to mimick the dose (6, 20, 50, or 100 mcg/kg/week) of investigational product

    Reporting group values
    TransCon CNP (6 mcg/kg/wk) TransCon CNP (20 mcg/kg/wk) TransCon CNP (50 mcg/kg/wk) TransCon CNP (100 mcg/kg/wk) Pooled Placebo Total
    Number of subjects
    10 11 10 11 15 57
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    6.52 ± 2.593 6.29 ± 2.896 5.20 ± 2.991 5.79 ± 2.613 5.89 ± 3.109 -
    Gender categorical
    Units: Subjects
        Female
    7 3 3 6 5 24
        Male
    3 8 7 5 10 33
    Race
    Units: Subjects
        American Indian or Alaskan Native
    0 0 0 0 0 0
        Asian
    2 1 1 0 2 6
        Black or African American
    0 0 0 1 1 2
        Native Hawaiian or Other Pacific Islander
    0 0 1 0 0 1
        White
    8 10 8 10 12 48
        Other
    0 0 0 0 0 0
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    1 0 2 2 1 6
        Not Hispanic or Latino
    9 11 6 9 14 49
        Unknown/Not Reported
    0 0 2 0 0 2
    Region
    Units: Subjects
        North America
    4 4 4 8 9 29
        Europe
    2 5 4 3 3 17
        Oceania
    4 2 2 0 3 11
    Height
    Units: cm
        arithmetic mean (standard deviation)
    90.63 ± 8.973 92.29 ± 12.103 86.61 ± 12.967 89.23 ± 12.822 90.85 ± 14.920 -
    Height SDS
    Units: Standard deviation score (SDS)
        arithmetic mean (standard deviation)
    -5.45 ± 1.046 -4.87 ± 0.673 -4.85 ± 0.801 -4.92 ± 0.829 -4.85 ± 0.958 -
    Weight
    Units: kg
        arithmetic mean (standard deviation)
    17.49 ± 3.677 19.67 ± 6.602 15.67 ± 4.399 17.03 ± 4.699 17.99 ± 5.542 -
    Body Mass Index
    Units: kg^m2
        arithmetic mean (standard deviation)
    21.10 ± 1.664 22.52 ± 2.599 20.61 ± 1.496 21.11 ± 1.612 21.39 ± 1.853 -
    Baseline AHV
    Baseline annualized height velocity (AHV)
    Units: cm/year
        arithmetic mean (standard deviation)
    5.04 ± 2.157 5.29 ± 1.619 5.76 ± 3.147 4.73 ± 1.133 6.17 ± 1.394 -

    End points

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    End points reporting groups
    Reporting group title
    TransCon CNP (6 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 6 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (20 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 20 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (50 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 50 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (100 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 100 mcg CNP/kg/week

    Reporting group title
    Pooled Placebo
    Reporting group description
    Once weekly subcutaneous administration of placebo for TransCon CNP to mimick the dose (6, 20, 50, or 100 mcg/kg/week) of investigational product

    Primary: Annualized Height Velocity

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    End point title
    Annualized Height Velocity
    End point description
    The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (TransCon CNP dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set.
    End point type
    Primary
    End point timeframe
    52 weeks
    End point values
    TransCon CNP (6 mcg/kg/wk) TransCon CNP (20 mcg/kg/wk) TransCon CNP (50 mcg/kg/wk) TransCon CNP (100 mcg/kg/wk) Pooled Placebo
    Number of subjects analysed
    10
    11
    10
    11
    15
    Units: cm/year
        least squares mean (confidence interval 95%)
    4.09 (3.34 to 4.84)
    4.52 (3.82 to 5.22)
    5.16 (4.43 to 5.90)
    5.42 (4.74 to 6.11)
    4.35 (3.75 to 4.94)
    Statistical analysis title
    Primary efficacy endpoint
    Statistical analysis description
    ANCOVA model using treatment (dose groups and pooled placebo) and sex as fixed effects, and baseline age and baseline height SDS as the covariates.
    Comparison groups
    TransCon CNP (6 mcg/kg/wk) v Pooled Placebo
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.6004
    Method
    ANCOVA
    Confidence interval
    Statistical analysis title
    Primary efficacy endpoint
    Statistical analysis description
    ANCOVA model using treatment (dose groups and pooled placebo) and sex as fixed effects, and baseline age and baseline height SDS as the covariates.
    Comparison groups
    TransCon CNP (20 mcg/kg/wk) v Pooled Placebo
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.7022
    Method
    ANCOVA
    Confidence interval
    Statistical analysis title
    Primary efficacy endpoint
    Statistical analysis description
    ANCOVA model using treatment (dose groups and pooled placebo) and sex as fixed effects, and baseline age and baseline height SDS as the covariates.
    Comparison groups
    TransCon CNP (50 mcg/kg/wk) v Pooled Placebo
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0849
    Method
    ANCOVA
    Confidence interval
    Statistical analysis title
    Primary efficacy endpoint
    Statistical analysis description
    ANCOVA model using treatment (dose groups and pooled placebo) and sex as fixed effects, and baseline age and baseline height SDS as the covariates.
    Comparison groups
    TransCon CNP (100 mcg/kg/wk) v Pooled Placebo
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0218
    Method
    ANCOVA
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    52 Week Blinded Period
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    TransCon CNP (6 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 6 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (20 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 20 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (50 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 50 mcg CNP/kg/week

    Reporting group title
    TransCon CNP (100 mcg/kg/wk)
    Reporting group description
    Once weekly subcutaneous administration of TransCon CNP 100 mcg CNP/kg/week

    Reporting group title
    Pooled Placebo
    Reporting group description
    Once weekly subcutaneous administration of placebo for TransCon CNP to mimick the dose (6, 20, 50, or 100 mcg/kg/week) of investigational product

    Serious adverse events
    TransCon CNP (6 mcg/kg/wk) TransCon CNP (20 mcg/kg/wk) TransCon CNP (50 mcg/kg/wk) TransCon CNP (100 mcg/kg/wk) Pooled Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    Nervous system disorders
    Febrile convulsion
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Viral infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    TransCon CNP (6 mcg/kg/wk) TransCon CNP (20 mcg/kg/wk) TransCon CNP (50 mcg/kg/wk) TransCon CNP (100 mcg/kg/wk) Pooled Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 10 (90.00%)
    11 / 11 (100.00%)
    10 / 10 (100.00%)
    10 / 11 (90.91%)
    14 / 15 (93.33%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    3 / 10 (30.00%)
    2 / 11 (18.18%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    4
    2
    8
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 10 (10.00%)
    4 / 11 (36.36%)
    2 / 10 (20.00%)
    2 / 11 (18.18%)
    5 / 15 (33.33%)
         occurrences all number
    2
    7
    2
    4
    8
    Injection site reaction
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    1
    2
    1
    Fatigue
         subjects affected / exposed
    1 / 10 (10.00%)
    2 / 11 (18.18%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    2
    0
    0
    1
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    1
    0
    1
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 10 (10.00%)
    2 / 11 (18.18%)
    2 / 10 (20.00%)
    3 / 11 (27.27%)
    3 / 15 (20.00%)
         occurrences all number
    1
    4
    3
    3
    5
    Diarrhoea
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    2 / 10 (20.00%)
    1 / 11 (9.09%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    3
    2
    2
    Abdominal pain upper
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    1
    1
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 10 (40.00%)
    3 / 11 (27.27%)
    4 / 10 (40.00%)
    2 / 11 (18.18%)
    3 / 15 (20.00%)
         occurrences all number
    7
    7
    6
    4
    5
    Rhinorrhoea
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 11 (18.18%)
    2 / 10 (20.00%)
    2 / 11 (18.18%)
    1 / 15 (6.67%)
         occurrences all number
    0
    2
    4
    2
    1
    Nasal congestion
         subjects affected / exposed
    1 / 10 (10.00%)
    3 / 11 (27.27%)
    2 / 10 (20.00%)
    0 / 11 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    4
    3
    0
    5
    Epistaxis
         subjects affected / exposed
    1 / 10 (10.00%)
    2 / 11 (18.18%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    2
    1
    0
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    2 / 10 (20.00%)
    1 / 11 (9.09%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    3
    1
    2
    Snoring
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    1
    0
    3
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    3 / 10 (30.00%)
    3 / 11 (27.27%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    7
    3
    1
    Arthralgia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    3 / 10 (30.00%)
    1 / 11 (9.09%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    4
    1
    18
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    3 / 10 (30.00%)
    1 / 11 (9.09%)
    5 / 10 (50.00%)
    0 / 11 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    3
    1
    8
    0
    2
    Nasopharyngitis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    5 / 10 (50.00%)
    2 / 11 (18.18%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    6
    2
    3
    Gastroenteritis
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 11 (18.18%)
    3 / 10 (30.00%)
    1 / 11 (9.09%)
    2 / 15 (13.33%)
         occurrences all number
    0
    2
    3
    1
    2
    COVID-19
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    3 / 11 (27.27%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    1
    3
    1
    Otitis media
         subjects affected / exposed
    1 / 10 (10.00%)
    2 / 11 (18.18%)
    2 / 10 (20.00%)
    0 / 11 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    2
    2
    0
    3
    Viral infection
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    2
    4
    0
    3
    Respiratory tract infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    3 / 10 (30.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    5
    0
    0
    Metabolism and nutrition disorders
    Vitamin D deficiency
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    01 Sep 2020
    Protocol Version 2.0 summary of changes: - increase enrollment to include approximately 14 participants in Cohorts 2-5 - add allowance for select visits to be conducted off-site - remove BMI as a secondary efficacy endpoint - modify the placebo comparator and dosing procedure for Cohort 1 - add availability of home health nurse to give weekly injections - update contact information for SAE reporting - update the definition of AE
    08 Jan 2021
    Protocol Version 3.0 added a 2 year Open-Label Extension period to assess long term safety and efficacy following the Randomized Period.
    12 Aug 2021
    Protocol Version 4.0 added implementation of unblinding per cohort after completion of the Randomized Treatment Period.
    28 Dec 2022
    Protocol Version 5.0 summary of changes: - Added information of a new separate long-term open-label extension study for participants completing treatment in the TCC-201 protocol - Updated SAE reporting

    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 reported
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