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    Clinical Trial Results:
    A Phase 2 Open Label Extension Study to Assess the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Children with Achondroplasia

    Summary
    EudraCT number
    		 2021-003149-39
    Trial protocol
    		 ES   IT   PT   BE   DK  
    Global end of trial date
    30 Mar 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Oct 2023
    First version publication date
    15 Oct 2023
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    C4181008
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05116046
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Pfizer Inc.
    Sponsor organisation address
    235E 42nd Street, New York, United States, NY 10017
    Public contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 8007181021, ClinicalTrials.gov_Inquires@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 8007181021, ClinicalTrials.gov_Inquires@pfizer.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    06 Apr 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Mar 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Evaluate the long-term safety and tolerability of recifercept doses and dosing regimens in subjects aged greater than or equal to (>=) 15 months to less than (<) 12 years with achondroplasia. To assess long-term efficacy of recifercept to increase height growth in children with achondroplasia.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Council for Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trials subjects were followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    24 Dec 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 2
    Country: Number of subjects enrolled
    Belgium: 3
    Country: Number of subjects enrolled
    Denmark: 6
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    Italy: 6
    Country: Number of subjects enrolled
    Portugal: 2
    Country: Number of subjects enrolled
    United States: 7
    Worldwide total number of subjects
    35
    EEA total number of subjects
    26
    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)
    33
    Adolescents (12-17 years)
    2
    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
    		 Eligible subjects aged more than or equal to (>=)15 months to less than (<) 12 years (inclusive) diagnosed with achondroplasia from study C4181005 (NCT04638153) were enrolled.

    Pre-assignment
    Screening details
    		 A total of 35 subjects were enrolled and assigned to study treatment.

    Period 1
    Period 1 title
    Overall period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly
    Arm description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recifercept
    Investigational medicinal product code
    PF-07256472
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    1 mg/kg once weekly

    Arm title
    		 Recifercept 2 mg/kg twice weekly
    Arm description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recifercept
    Investigational medicinal product code
    PF-07256472
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    2 mg/kg once weekly

    Arm title
    		 Recifercept 1.5 mg/kg once daily
    Arm description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Recifercept
    Investigational medicinal product code
    PF-07256472
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    1.5 mg/kg once weekly

    Number of subjects in period 1
    		Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Started
    16
    17
    2
    Completed
    0
    0
    0
    Not completed
    16
    17
    2
         Study terminated by sponsor
    14
    16
    1
         Withdrawal by parent/guardian
    2
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Recifercept 1 milligram per kilogram (mg/kg) once weekly
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.

    Reporting group title
    Recifercept 2 mg/kg twice weekly
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.

    Reporting group title
    Recifercept 1.5 mg/kg once daily
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

    Reporting group values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily Total
    Number of subjects
    		 16 17 2 35
    Age Categorical
    Units: Subjects
        Children (2-11 years)
    		 15 16 2 33
        Adolescents (12-17 years)
    		 1 1 0 2
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 7.5 ± 2.66 6.6 ± 2.50 9.5 ± 0.71 -
    Gender Categorical
    Units: Subjects
        Female
    		 9 9 2 20
        Male
    		 7 8 0 15
    Race
    Units: Subjects
        White
    		 14 17 2 33
        Black or African American
    		 0 0 0 0
        Asian
    		 1 0 0 1
        American Indian or Alaska Native
    		 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Other
    		 0 0 0 0
        Unknown
    		 0 0 0 0
        Multiracial
    		 1 0 0 1
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 2 0 1 3
        Not Hispanic or Latino
    		 14 17 1 32

    End points

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    End points reporting groups
    Reporting group title
    Recifercept 1 milligram per kilogram (mg/kg) once weekly
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.

    Reporting group title
    Recifercept 2 mg/kg twice weekly
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.

    Reporting group title
    Recifercept 1.5 mg/kg once daily
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

    Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs

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    End point title
    		 Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs [1]
    End point description
    An AE is any untoward medical occurrence in a subject or clinical study subject, temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was an AE resulting in any of the following outcomes or considered medically significant: death; initial or prolonged inpatient hospitalisation; life-threatening experience; persistent or significant disability/incapacity; congenital anomaly or birth defect; suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. Severe AEs were AEs that were medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling, limiting self-care activities of daily living. Full Analysis Set consisted of all subjects who received at least one dose of recifercept. Subjects were analysed according to the dose they actually received.
    End point type
    Primary
    End point timeframe
    From first dose of recifercept until 28 days after last dose of study treatment (maximum up to 24 months)
    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: Only descriptive analysis was planned for this endpoint.
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    16
    17
    2
    Units: Subjects
        AEs
    9
    11
    0
        SAEs
    0
    0
    0
    No statistical analyses for this end point

    Primary: Change From Baseline in Height at Month 24

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    End point title
    		 Change From Baseline in Height at Month 24 [2]
    End point description
    Height was measured using anthropometric measurements. FAS included all subjects who received at least one dose of recifercept. Subjects were planned to be analysed according to the dose they actually received.
    End point type
    Primary
    End point timeframe
    Baseline and month 24
    Notes
    [2] - 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: Only descriptive analysis was planned for this endpoint.
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [3]
    0 [4]
    0 [5]
    Units: Centimeter (cm)
        least squares mean (confidence interval 95%)
    ( to )
    ( to )
    ( to )
    Notes
    [3] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [4] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [5] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in arm Span to Height/Length Difference at Months 3, 6, 9

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    End point title
    		 Change From Baseline in arm Span to Height/Length Difference at Months 3, 6, 9
    End point description
    Height was calculated with anthropometric measurements. FAS included all subjects who received at least one dose of recifercept. Subjects were planned to be analysed according to the dose they actually received.
    End point type
    Secondary
    End point timeframe
    Baseline and months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [6]
    0 [7]
    0 [8]
    Units: Centimeters
        least squares mean (confidence interval 95%)
    ( to )
    ( to )
    ( to )
    Notes
    [6] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [7] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [8] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9
    End point description
    Height was calculated based upon the anthropometric measurements. FAS included all subjects who received at least one dose of recifercept. Subjects were planned to be analysed according to the dose they actually received.
    End point type
    Secondary
    End point timeframe
    Baseline and Months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [9]
    0 [10]
    0 [11]
    Units: Ratio
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [9] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [10] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [11] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Clearance (CL/F) of Recifercept

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    End point title
    		 Clearance (CL/F) of Recifercept
    End point description
    Clearance of a drug was a measure of the rate at which a drug is metabolised or eliminated by normal biological processes. Pharmacokinetic (PK) concentration set included all subjects who received at least 1 dose of recifercept and had at least 1 evaluable concentration result.
    End point type
    Secondary
    End point timeframe
    Day 91, 181, 271, 361 and 451.
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [12]
    0 [13]
    0 [14]
    Units: milliliters per minute (mL/min)
        geometric mean (geometric coefficient of variation)
    ±
    ±
    ±
    Notes
    [12] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [13] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [14] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9
    End point description
    Knee height was defined as the distance from the sole of the foot to the most anterior surface of the femoral condyles of the thigh (medial being more anterior), with the ankle and knee each flexed to a 90-degree angle. Lower segment of the leg included tibia and foot height. FAS included all subjects who received at least one dose of recifercept. Subjects were planned to be analysed according to the dose they actually received.
    End point type
    Secondary
    End point timeframe
    Baseline and months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [15]
    0 [16]
    0 [17]
    Units: Ratio
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [15] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [16] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [17] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9
    End point description
    Occipito-frontal circumference was measured by anthropometric measurements. It was measured over the most prominent part on the back of the head (occiput) and just above the eyebrows (supraorbital ridges). FAS included all subjects who received at least one dose of recifercept. Subjects were planned to be analysed according to the dose they actually received.
    End point type
    Secondary
    End point timeframe
    Baseline and months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [18]
    0 [19]
    0 [20]
    Units: Centimeters
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [18] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [19] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [20] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9
    End point description
    Occipito-frontal circumference was measured by anthropometric measurements. FAS included all subjects who were planned to receive at least one dose of recifercept.
    End point type
    Secondary
    End point timeframe
    Baseline, months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [21]
    0 [22]
    0 [23]
    Units: Ratio
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [21] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [22] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [23] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Body Mass Index (BMI) at Months 3, 6, 9
    End point description
    FAS included all subjects who were planned to receive at least one dose of recifercept.
    End point type
    Secondary
    End point timeframe
    At Months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [24]
    0 [25]
    0 [26]
    Units: Kilograms per meter square
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [24] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [25] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [26] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9
    End point description
    The Z-score described how many standard deviations a given measurement lies above or below a size or age-specific population mean. A Z-score above the population mean will have a positive value, whereas a Z-score below the population mean will have a negative value. The greater the deviation of the Z-score from zero (in a positive or negative direction), the greater the magnitude of deviation from the mean.
    End point type
    Secondary
    End point timeframe
    Baseline, months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [27]
    0 [28]
    0 [29]
    Units: Units on a scale
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [27] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [28] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [29] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9
    End point description
    Fixed Flexion Angles was measured by anthropometric measurements. FAS included all subjects who were planned to receive at least one dose of recifercept.
    End point type
    Secondary
    End point timeframe
    Baseline, months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [30]
    0 [31]
    0 [32]
    Units: Degrees
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [30] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [31] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [32] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Number of Subjects With Clinically Meaningful Findings in Laboratory Test Parameters Through The Study

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    End point title
    		 Number of Subjects With Clinically Meaningful Findings in Laboratory Test Parameters Through The Study
    End point description
    Laboratory parameters such as lymphocytes, neutrophils, eosinophils, monocytes and potassium were assessed. Clinically significant abnormal laboratory findings were determined by the investigator's decision. FAS included all subjects who were planned to receive at least one dose of recifercept.
    End point type
    Secondary
    End point timeframe
    From baseline up to follow-up
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    15
    16
    2
    Units: Subjects
        Hematology: Lymphocytes
    0
    1
    0
        Hematology:Neutrophils
    0
    1
    0
        Hematology: Eosinophils
    1
    3
    0
        Hematology: Monocytes
    2
    0
    0
        Chemistry: Potassium
    2
    1
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Waist to Chest Circumference Ratio at Months 3, 6, 9

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    End point title
    		 Change From Baseline in Waist to Chest Circumference Ratio at Months 3, 6, 9
    End point description
    FAS included all subjects who were planned to receive at least one dose of recifercept.
    End point type
    Secondary
    End point timeframe
    Baseline, Months 3, 6, 9
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    0 [33]
    0 [34]
    0 [35]
    Units: Ratio
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Notes
    [33] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [34] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    [35] - Data was not collected as study was terminated based on sponsor discretion (not related to safety).
    No statistical analyses for this end point

    Secondary: Number of Subjects With Positive Anti-Drug Antibodies (ADA)

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    End point title
    		 Number of Subjects With Positive Anti-Drug Antibodies (ADA)
    End point description
    End point type
    Secondary
    End point timeframe
    From Day 91 up to Month 24
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    16
    17
    2
    Units: Subjects
    12
    15
    2
    No statistical analyses for this end point

    Secondary: Number of Subjects With Clinically Significant Findings in Vital Signs Through The Study

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    End point title
    		 Number of Subjects With Clinically Significant Findings in Vital Signs Through The Study
    End point description
    Absolute values and changes from baseline in supine systolic and diastolic blood pressure, oral temperature, and pulse rate were planned to be summarised by treatment in accordance with the sponsor reporting standards.
    End point type
    Secondary
    End point timeframe
    From baseline up to follow-up
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    16
    17
    2
    Units: Subjects
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Subjects With Clinically Significant Findings in Physical Examination Through The Study

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    End point title
    		 Number of Subjects With Clinically Significant Findings in Physical Examination Through The Study
    End point description
    A complete physical examination included cardiovascular, respiratory, gastrointestinal systems, and skin. Height and weight will also be measured and recorded as part of the anthropometric measurements collected.
    End point type
    Secondary
    End point timeframe
    From baseline up to follow-up
    End point values
    		 Recifercept 1 milligram per kilogram (mg/kg) once weekly Recifercept 2 mg/kg twice weekly Recifercept 1.5 mg/kg once daily
    Number of subjects analysed
    16
    17
    2
    Units: Subjects
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From start of the study treatment up to follow-up (approximately 24 months)
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    Recifercept 1 mg/kg once weekly
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1 mg/kg once weekly via the subcutaneous route for up to 24 months.

    Reporting group title
    Recifercept 1.5 mg/kg once daily
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 1.5 mg/kg once daily via the subcutaneous route for up to 24 months.

    Reporting group title
    Recifercept 2 mg/kg twice weekly
    Reporting group description
    		 Subjects with achondroplasia who completed study C4181005 (NCT04638153) continued to receive recifercept 2 mg/kg twice weekly via the subcutaneous route for up to 24 months.

    Serious adverse events
    		 Recifercept 1 mg/kg once weekly Recifercept 1.5 mg/kg once daily Recifercept 2 mg/kg twice weekly
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Recifercept 1 mg/kg once weekly Recifercept 1.5 mg/kg once daily Recifercept 2 mg/kg twice weekly
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 16 (56.25%)
    0 / 2 (0.00%)
    11 / 17 (64.71%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    2
    General disorders and administration site conditions
    Injection site rash
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Pyrexia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Fatigue
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Injection site haematoma
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Reproductive system and breast disorders
    Breast pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 16 (18.75%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    0
    Catarrh
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Investigations
    Blood phosphorous increased
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Blood urea increase
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Platelet count increased
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    2
    Injury, poisoning and procedural complications
    Clavicle fracture
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Vaccination complication
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Nervous system disorders
    Nystagmus
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Headache
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    2
    Blood and lymphatic system disorders
    Lymphopenia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Tympanic membrane perforation
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Eustachian tube dysfunction
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Toothache
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Odynophagia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Dermatitis
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Myalgia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    2
    Infections and infestations
    COVID-19
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    0
    Otitis externa
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    2
    Oral herpes
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Nasopharyngiti
         subjects affected / exposed
    3 / 16 (18.75%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    3
    0
    2
    Influenza
         subjects affected / exposed
    2 / 16 (12.50%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    2
    0
    1
    Gastroenteritis viral
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    0
    Ear infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Conjunctivitis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Otitis media
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Viral infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 2 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Skin candida
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Rhinitis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    4 / 17 (23.53%)
         occurrences all number
    1
    0
    4
    Otitis media acute
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 2 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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