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Clinical trials for achondroplasia

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44020   clinical trials with a EudraCT protocol, of which   7318   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    14 result(s) found for: achondroplasia. Displaying page 1 of 1.
    EudraCT Number: 2020-001189-13 Sponsor Protocol Number: C4181005 Start Date*: 2021-02-26
    Sponsor Name:Pfizer Inc.
    Full Title: A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
    Medical condition: Achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: PT (Prematurely Ended) DK (Prematurely Ended) BE (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-003149-39 Sponsor Protocol Number: C4181008 Start Date*: 2021-12-14
    Sponsor Name:Pfizer Inc.
    Full Title: A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
    Medical condition: Achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) IT (Prematurely Ended) PT (Prematurely Ended) BE (Completed) DK (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-002754-22 Sponsor Protocol Number: TCC-201 Start Date*: 2019-11-01
    Sponsor Name:Ascendis Pharma Growth Disorders A/S
    Full Title: ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP admin...
    Medical condition: Achondroplasia (ACH) in prepubertal children
    Disease: Version SOC Term Classification Code Term Level
    25.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IE (Ongoing) GB (GB - no longer in EU/EEA) DE (Ongoing) AT (Ongoing) DK (Ongoing) PT (Ongoing)
    Trial results: View results
    EudraCT Number: 2022-002954-25 Sponsor Protocol Number: ASND0036 Start Date*: 2023-03-21
    Sponsor Name:Ascendis Pharma Growth Disorders A/S
    Full Title: ApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in C...
    Medical condition: Achondroplasia (ACH) in prepubertal children
    Disease: Version SOC Term Classification Code Term Level
    25.0 10010331 - Congenital, familial and genetic disorders 10000452 Achondroplasia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IE (Ongoing) DK (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-000701-21 Sponsor Protocol Number: 111-901 Start Date*: 2017-06-19
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia
    Medical condition: Achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2019-002954-21 Sponsor Protocol Number: QBGJ398-201 Start Date*: 2020-03-31
    Sponsor Name:QED Therapeutics, Inc.
    Full Title: Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2
    Medical condition: Achondroplasia in Children
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2013-004137-32 Sponsor Protocol Number: 111-202 Start Date*: 2014-04-03
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia
    Medical condition: achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10000452 Achondroplasia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2021-001855-15 Sponsor Protocol Number: QBGJ398-203 Start Date*: 2022-02-02
    Sponsor Name:QED Therapeutics, Inc.
    Full Title: Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL OLE
    Medical condition: Achondroplasia in Children
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-004004-30 Sponsor Protocol Number: 111-205 Start Date*: Information not available in EudraCT
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia
    Medical condition: achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-002404-28 Sponsor Protocol Number: 111-302 Start Date*: 2018-10-12
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
    Medical condition: Achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing) DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-003836-11 Sponsor Protocol Number: 111-301 Start Date*: 2017-01-20
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia.
    Medical condition: achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2018-004364-66 Sponsor Protocol Number: BMR111-208 Start Date*: 2019-10-22
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
    Medical condition: Achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2020-001055-40 Sponsor Protocol Number: 111-209 Start Date*: 2020-07-29
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasia at risk of requiring cervicom...
    Medical condition: achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2016-003826-18 Sponsor Protocol Number: 111-206 Start Date*: 2018-09-12
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia, Age 0 to < 60 Months
    Medical condition: Achondroplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000452 Achondroplasia LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
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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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