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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
    01 Oct 2024

    Results information
    Results version number
    		 v2(current)
    This version publication date
    24 May 2025
    First version publication date
    11 Apr 2023
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    update to include OLE safety data

    Trial information

    		 Close Top of page
    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
    Final
    Date of interim/final analysis
    19 Feb 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Oct 2024
    Was the trial ended prematurely?
    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
    Portugal: 1
    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
    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. All 57 subjects completed the 52-week double-blind period and entered the 104-week open-label extension (OLE), where they received TransCon CNP. All subjects received up to a maximum dose level of 100-mcg dose of TransCon CNP during OLE.

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

    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
    Period 2
    Period 2 title
    Open-Label Period (Weeks 52 to 156)
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Open-Label Extension Period: TransCon CNP
    Arm description
    		 Subjects who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection.
    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.

    Number of subjects in period 2
    		Open-Label Extension Period: TransCon CNP
    Started
    57
    Completed
    55
    Not completed
    2
         Consent withdrawn by subject
    2

    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.
    Reporting group title
    Open-Label Extension Period: TransCon CNP
    Reporting group description
    		 Subjects who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection.

    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
    From Week 0 to Week 52 for double-blind treatment period and up to Week 156 for OLE Period
    Adverse event reporting additional description
    Analysis was performed on safety analysis set that included all randomised subjects who received at least one dose of trial drug. Subjects were analysed according to trial treatment as treated. Adverse Events were reported as per MedDRA version 24.1 for double-blind treatment period and MedDRA version 26.0 for OLE 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.

    Reporting group title
    Open-Label Extension Period: TransCon CNP
    Reporting group description
    		 Subjects who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection.

    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 Open-Label Extension Period: TransCon CNP
    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%)
    2 / 57 (3.51%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    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%)
    0 / 57 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Obstructive sleep apnoea syndrome
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    1 / 57 (1.75%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Tonsillar hypertrophy
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    1 / 57 (1.75%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    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%)
    0 / 57 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Influenza
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    1 / 57 (1.75%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    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 Open-Label Extension Period: TransCon CNP
    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%)
    57 / 57 (100.00%)
    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%)
    23 / 57 (40.35%)
         occurrences all number
    2
    7
    2
    4
    8
    56
    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%)
    5 / 57 (8.77%)
         occurrences all number
    1
    1
    1
    2
    1
    24
    Fatigue
         subjects affected / exposed
    1 / 10 (10.00%)
    2 / 11 (18.18%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
    0 / 57 (0.00%)
         occurrences all number
    1
    2
    0
    0
    1
    0
    Influenza like illness
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    0
    0
    0
    0
    0
    3
    Injection site pain
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    6 / 57 (10.53%)
         occurrences all number
    1
    0
    0
    0
    0
    26
    Injection site swelling
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    4 / 57 (7.02%)
         occurrences all number
    0
    0
    1
    0
    0
    6
    Immune system disorders
    Seasonal allergy
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    0 / 15 (0.00%)
    6 / 57 (10.53%)
         occurrences all number
    0
    1
    0
    1
    0
    8
    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%)
    21 / 57 (36.84%)
         occurrences all number
    7
    7
    6
    4
    5
    51
    Rhinorrhoea
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 11 (18.18%)
    2 / 10 (20.00%)
    2 / 11 (18.18%)
    1 / 15 (6.67%)
    11 / 57 (19.30%)
         occurrences all number
    0
    2
    4
    2
    1
    18
    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%)
    13 / 57 (22.81%)
         occurrences all number
    1
    4
    3
    0
    5
    31
    Epistaxis
         subjects affected / exposed
    1 / 10 (10.00%)
    2 / 11 (18.18%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    1
    2
    1
    0
    0
    3
    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%)
    10 / 57 (17.54%)
         occurrences all number
    0
    0
    3
    1
    2
    14
    Snoring
         subjects affected / exposed
    1 / 10 (10.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    3 / 15 (20.00%)
    7 / 57 (12.28%)
         occurrences all number
    1
    1
    1
    0
    3
    9
    Obstructive sleep apnoea syndrome
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    6 / 57 (10.53%)
         occurrences all number
    0
    0
    0
    0
    0
    6
    Sleep apnoea syndrome
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    4 / 57 (7.02%)
         occurrences all number
    0
    1
    0
    0
    0
    5
    Tonsillar hypertrophy
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    0
    0
    0
    0
    0
    3
    Investigations
    Vitamin D decreased
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    0 / 15 (0.00%)
    5 / 57 (8.77%)
         occurrences all number
    0
    0
    0
    1
    0
    5
    Injury, poisoning and procedural complications
    Fall
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
    5 / 57 (8.77%)
         occurrences all number
    1
    0
    0
    0
    2
    6
    Skin abrasion
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    0
    0
    0
    0
    0
    3
    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%)
    17 / 57 (29.82%)
         occurrences all number
    0
    1
    4
    2
    8
    40
    Blood and lymphatic system disorders
    Lymphadenopathy
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    4 / 57 (7.02%)
         occurrences all number
    0
    0
    0
    0
    0
    4
    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%)
    10 / 57 (17.54%)
         occurrences all number
    1
    1
    1
    0
    1
    22
    Hypoacusis
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    7 / 57 (12.28%)
         occurrences all number
    0
    0
    1
    0
    0
    10
    Middle ear effusion
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    5 / 57 (8.77%)
         occurrences all number
    0
    0
    1
    0
    0
    6
    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%)
    16 / 57 (28.07%)
         occurrences all number
    1
    4
    3
    3
    5
    25
    Diarrhoea
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    2 / 10 (20.00%)
    1 / 11 (9.09%)
    2 / 15 (13.33%)
    6 / 57 (10.53%)
         occurrences all number
    1
    0
    3
    2
    2
    8
    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%)
    4 / 57 (7.02%)
         occurrences all number
    0
    1
    1
    1
    2
    7
    Abdominal pain
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    0
    1
    0
    0
    0
    4
    Nausea
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    0 / 15 (0.00%)
    3 / 57 (5.26%)
         occurrences all number
    0
    0
    0
    1
    0
    3
    Skin and subcutaneous tissue disorders
    Rash
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    1 / 15 (6.67%)
    4 / 57 (7.02%)
         occurrences all number
    0
    0
    0
    1
    1
    7
    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%)
    7 / 57 (12.28%)
         occurrences all number
    1
    1
    7
    3
    1
    8
    Arthralgia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    3 / 10 (30.00%)
    1 / 11 (9.09%)
    1 / 15 (6.67%)
    9 / 57 (15.79%)
         occurrences all number
    1
    0
    4
    1
    18
    14
    Back pain
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    9 / 57 (15.79%)
         occurrences all number
    0
    0
    0
    0
    0
    10
    Knee deformity
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
    3 / 57 (5.26%)
         occurrences all number
    0
    0
    0
    0
    1
    3
    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%)
    14 / 57 (24.56%)
         occurrences all number
    3
    1
    8
    0
    2
    22
    Nasopharyngitis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    5 / 10 (50.00%)
    2 / 11 (18.18%)
    1 / 15 (6.67%)
    20 / 57 (35.09%)
         occurrences all number
    2
    0
    6
    2
    3
    35
    Gastroenteritis
         subjects affected / exposed
    0 / 10 (0.00%)
    2 / 11 (18.18%)
    3 / 10 (30.00%)
    1 / 11 (9.09%)
    2 / 15 (13.33%)
    10 / 57 (17.54%)
         occurrences all number
    0
    2
    3
    1
    2
    13
    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%)
    20 / 57 (35.09%)
         occurrences all number
    0
    1
    1
    3
    1
    22
    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%)
    14 / 57 (24.56%)
         occurrences all number
    1
    2
    2
    0
    3
    18
    Viral infection
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    2 / 15 (13.33%)
    8 / 57 (14.04%)
         occurrences all number
    0
    2
    4
    0
    3
    17
    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%)
    0 / 57 (0.00%)
         occurrences all number
    0
    0
    5
    0
    0
    0
    Conjunctivitis
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    4 / 57 (7.02%)
         occurrences all number
    0
    0
    1
    0
    0
    5
    Ear infection
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
    14 / 57 (24.56%)
         occurrences all number
    0
    0
    1
    0
    1
    27
    Gastroenteritis viral
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    2 / 11 (18.18%)
    0 / 15 (0.00%)
    7 / 57 (12.28%)
         occurrences all number
    0
    0
    0
    2
    0
    11
    Influenza
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    10 / 57 (17.54%)
         occurrences all number
    1
    0
    0
    0
    0
    10
    Otitis media acute
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    1 / 15 (6.67%)
    7 / 57 (12.28%)
         occurrences all number
    0
    0
    1
    0
    3
    15
    Pharyngitis
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    5 / 57 (8.77%)
         occurrences all number
    0
    0
    0
    0
    0
    5
    Pharyngitis streptococcal
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    5 / 57 (8.77%)
         occurrences all number
    0
    0
    0
    0
    0
    6
    Rhinitis
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 11 (9.09%)
    1 / 10 (10.00%)
    0 / 11 (0.00%)
    0 / 15 (0.00%)
    6 / 57 (10.53%)
         occurrences all number
    0
    1
    1
    0
    0
    6
    Sinusitis
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    2 / 11 (18.18%)
    0 / 15 (0.00%)
    4 / 57 (7.02%)
         occurrences all number
    0
    0
    0
    3
    0
    4
    Viral upper respiratory tract infection
    alternative dictionary used: MedDRA 26.0
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    0 / 10 (0.00%)
    0 / 11 (0.00%)
    2 / 15 (13.33%)
    6 / 57 (10.53%)
         occurrences all number
    0
    0
    0
    0
    2
    8
    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%)
    5 / 57 (8.77%)
         occurrences all number
    1
    1
    0
    0
    1
    6

    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 subjects 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 subjects 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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    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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