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    Clinical Trial Results:
    A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial with an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults with Hypoparathyroidism.

    Summary
    EudraCT number
    		 2018-004815-33
    Trial protocol
    		 NO   DE   DK   GB   IT  
    Global end of trial date

    Results information
    Results version number
    		 v1(current)
    This version publication date
    19 Dec 2022
    First version publication date
    19 Dec 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TransCon PTH TCP-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04009291
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Ascendis Pharma A/S
    Sponsor organisation address
    Tuborg Boulevard 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
    06 Mar 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    06 Mar 2020
    Global end of trial reached?
    No
    General information about the trial
    Main objective of the trial
    To assess the effectiveness of daily TransCon PTH on serum and urine calcium levels (FECa) and active vitamin D and calcium doses at 4 weeks of treatment.
    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
    13 Mar 2019
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 4 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 18
    Country: Number of subjects enrolled
    Norway: 1
    Country: Number of subjects enrolled
    Denmark: 12
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Italy: 6
    Country: Number of subjects enrolled
    Canada: 20
    Worldwide total number of subjects
    59
    EEA total number of subjects
    21
    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)
    54
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Overall, 59 subjects were enrolled and dosed. Enrollment of subjects occurred in six countries: Canada, Denmark, Germany, Italy, Norway, and the United States.

    Pre-assignment
    Screening details
    		 A total of 104 subjects were screened and 59 of these met eligibility criteria and were enrolled into the study.

    Period 1
    Period 1 title
    4 Week Blinded Period (overall 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 PTH 15 mcg/day
    Arm description
    		 Once daily subcutaneous administration of 15 mcg of TransCon PTH
    Arm type
    		 Experimental

    Investigational medicinal product name
    TransCon PTH
    Investigational medicinal product code
    ACP-014
    Other name
    Palopegteriparatide
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    TransCon PTH drug product was supplied as a clear solution containing palopegteriparatide with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.

    Arm title
    		 TransCon PTH 18 mcg/day
    Arm description
    		 Once daily subcutaneous administration of 18 mcg of TransCon PTH
    Arm type
    		 Experimental

    Investigational medicinal product name
    TransCon PTH
    Investigational medicinal product code
    ACP-014
    Other name
    Palopegteriparatide
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    TransCon PTH drug product was supplied as a clear solution containing palopegteriparatide with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.

    Arm title
    		 TransCon PTH 21 mcg/day
    Arm description
    		 Once daily subcutaneous administration of 21 mcg of TransCon PTH
    Arm type
    		 Experimental

    Investigational medicinal product name
    TransCon PTH
    Investigational medicinal product code
    ACP-014
    Other name
    Palopegteriparatide
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    TransCon PTH drug product was supplied as a clear solution containing palopegteriparatide with a nominal PTH(1-34) content of 0.3 mg/mL in a pre-filled pen intended for subcutaneous injection.

    Arm title
    		 Placebo
    Arm description
    		 Once daily subcutaneous administration of placebo for TransCon PTH to mimick administration of 15, 18, or 21 mcg of investigational product
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Placebo for TransCon PTH drug product was supplied as a clear solution containing placebo liquid to match the investigational product in a pre-filled pen intended for subcutaneous injection.

    Number of subjects in period 1
    		TransCon PTH 15 mcg/day TransCon PTH 18 mcg/day TransCon PTH 21 mcg/day Placebo
    Started
    14
    15
    15
    15
    Completed
    14
    15
    15
    15

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    TransCon PTH 15 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 15 mcg of TransCon PTH

    Reporting group title
    TransCon PTH 18 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 18 mcg of TransCon PTH

    Reporting group title
    TransCon PTH 21 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 21 mcg of TransCon PTH

    Reporting group title
    Placebo
    Reporting group description
    		 Once daily subcutaneous administration of placebo for TransCon PTH to mimick administration of 15, 18, or 21 mcg of investigational product

    Reporting group values
    		 TransCon PTH 15 mcg/day TransCon PTH 18 mcg/day TransCon PTH 21 mcg/day Placebo Total
    Number of subjects
    		 14 15 15 15 59
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 47.03 ( 13.230 ) 46.58 ( 11.157 ) 53.67 ( 11.287 ) 51.80 ( 12.345 ) -
    Gender categorical
    Units: Subjects
        Female
    		 12 12 12 12 48
        Male
    		 2 3 3 3 11
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0
        Asian
    		 0 0 2 0 2
        Black or African American
    		 0 0 0 0 0
        Pacific Islander
    		 0 0 0 0 0
        White
    		 14 12 13 15 54
        Other
    		 0 3 0 0 3
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 1 0 1
        Not Hispanic or Latino
    		 14 15 14 15 58
        Unknown
    		 0 0 0 0 0
    Height
    Units: cm
        arithmetic mean (standard deviation)
    		 166.92 ( 8.806 ) 166.71 ( 8.385 ) 165.37 ( 10.961 ) 164.07 ( 10.368 ) -
    Weight
    Units: kg
        arithmetic mean (standard deviation)
    		 76.58 ( 22.479 ) 80.04 ( 11.279 ) 72.26 ( 18.621 ) 76.43 ( 14.256 ) -
    Body Mass Index
    Units: kg/m^2
        arithmetic mean (standard deviation)
    		 27.08 ( 5.723 ) 28.76 ( 3.148 ) 26.12 ( 4.647 ) 28.30 ( 3.775 ) -

    End points

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    End points reporting groups
    Reporting group title
    TransCon PTH 15 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 15 mcg of TransCon PTH

    Reporting group title
    TransCon PTH 18 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 18 mcg of TransCon PTH

    Reporting group title
    TransCon PTH 21 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 21 mcg of TransCon PTH

    Reporting group title
    Placebo
    Reporting group description
    		 Once daily subcutaneous administration of placebo for TransCon PTH to mimick administration of 15, 18, or 21 mcg of investigational product

    Primary: Efficacy - Primary Endpoint

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    End point title
    		 Efficacy - Primary Endpoint
    End point description
    The proportion of subjects with albumin-adjusted or ionized serum calcium within the normal range, and spot morning fractional excretion of calcium (spot AM FECa) within normal range (≤2%) or a reduction by at least 50% from baseline, and not taking active vitamin D supplements, and taking ≤1000 mg/day of calcium supplements
    End point type
    Primary
    End point timeframe
    4 weeks
    End point values
    		 TransCon PTH 15 mcg/day TransCon PTH 18 mcg/day TransCon PTH 21 mcg/day Placebo
    Number of subjects analysed
    14
    15
    15
    15
    Units: percent
        number (confidence interval 95%)
    50.0 (23.0 to 77.0)
    40.0 (16.3 to 67.7)
    60.0 (32.3 to 83.7)
    26.7 (7.8 to 55.1)
    Statistical analysis title
    		 Primary efficacy endpoint
    Statistical analysis description
    Fisher's exact test is used to compare differences in the proportion of subjects meeting the composite primary endpoint in the TransCon PTH versus pooled placebo group
    Comparison groups
    TransCon PTH 15 mcg/day v Placebo
    Number of subjects included in analysis
    29
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.2635
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 Primary efficacy endpoint
    Statistical analysis description
    Fisher's exact test is used to compare differences in the proportion of subjects meeting the composite primary endpoint in the TransCon PTH versus pooled placebo group
    Comparison groups
    TransCon PTH 18 mcg/day v Placebo
    Number of subjects included in analysis
    30
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.6999
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 Primary efficacy endpoint
    Statistical analysis description
    Fisher's exact test is used to compare differences in the proportion of subjects meeting the composite primary endpoint in the TransCon PTH versus pooled placebo group
    Comparison groups
    TransCon PTH 21 mcg/day v Placebo
    Number of subjects included in analysis
    30
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1394
    Method
    		 t-test, 2-sided
    Confidence interval

    Secondary: Efficacy - Secondary Endpoint

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    End point title
    		 Efficacy - Secondary Endpoint
    End point description
    The proportion of subjects with albumin-adjusted or ionized serum calcium within the normal range, and spot morning fractional excretion of calcium (spot AM FECa) within normal range (≤2%) or a reduction by at least 50% from baseline, and not taking active vitamin D supplements, and taking ≤500 mg/day of calcium supplements
    End point type
    Secondary
    End point timeframe
    4 weeks
    End point values
    		 TransCon PTH 15 mcg/day TransCon PTH 18 mcg/day TransCon PTH 21 mcg/day Placebo
    Number of subjects analysed
    14
    15
    15
    15
    Units: percent
        number (confidence interval 95%)
    50.0 (23.0 to 77.0)
    26.7 (7.8 to 55.1)
    60 (32.3 to 83.7)
    20.0 (4.3 to 48.1)
    Statistical analysis title
    		 Secondary efficacy endpoint
    Statistical analysis description
    Fisher's exact test is used to compare differences in the proportion of subjects meeting the composite primary endpoint in the TransCon PTH versus pooled placebo group
    Comparison groups
    TransCon PTH 15 mcg/day v Placebo
    Number of subjects included in analysis
    29
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1281
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 Secondary efficacy endpoint
    Statistical analysis description
    Fisher's exact test is used to compare differences in the proportion of subjects meeting the composite primary endpoint in the TransCon PTH versus pooled placebo group
    Comparison groups
    TransCon PTH 18 mcg/day v Placebo
    Number of subjects included in analysis
    30
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 > 0.9999
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 Secondary efficacy endpoint
    Statistical analysis description
    Fisher's exact test is used to compare differences in the proportion of subjects meeting the composite primary endpoint in the TransCon PTH versus pooled placebo group
    Comparison groups
    TransCon PTH 21 mcg/day v Placebo
    Number of subjects included in analysis
    30
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0604
    Method
    		 t-test, 2-sided
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    4 Week Blinded Period
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24
    Reporting groups
    Reporting group title
    TransCon PTH 15 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 15 mcg of TransCon PTH

    Reporting group title
    TransCon PTH 18 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 18 mcg of TransCon PTH

    Reporting group title
    TransCon PTH 21 mcg/day
    Reporting group description
    		 Once daily subcutaneous administration of 21 mcg of TransCon PTH

    Reporting group title
    Placebo
    Reporting group description
    		 Once daily subcutaneous administration of placebo for TransCon PTH to mimick administration of 15, 18, or 21 mcg of investigational product

    Serious adverse events
    		 TransCon PTH 15 mcg/day TransCon PTH 18 mcg/day TransCon PTH 21 mcg/day Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 3.4%
    Non-serious adverse events
    		 TransCon PTH 15 mcg/day TransCon PTH 18 mcg/day TransCon PTH 21 mcg/day Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 14 (42.86%)
    5 / 15 (33.33%)
    7 / 15 (46.67%)
    6 / 15 (40.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 14 (21.43%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
         occurrences all number
    3
    1
    1
    1
    Dizziness
         subjects affected / exposed
    0 / 14 (0.00%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    2
    0
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Injection site pain
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Thirst
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    1 / 15 (6.67%)
         occurrences all number
    2
    1
    1
    1
    Constipation
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Influenza
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Hypercalcemia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 15 (6.67%)
    2 / 15 (13.33%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    2
    0
    Hypocalcemia
         subjects affected / exposed
    0 / 14 (0.00%)
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    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
    08 May 2019
    The TCP-201 protocol was amended to incorporate feedback from multiple Health Authorities and included changes to the trial design, secondary endpoints, and exclusion criteria.

    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

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/34347093
    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
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