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Clinical trials for Dwarfism

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   44335   clinical trials with a EudraCT protocol, of which   7366   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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    47 result(s) found for: Dwarfism. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2015-001569-20 Sponsor Protocol Number: 27709 Start Date*: 2015-07-06
    Sponsor Name:Merck Serono Co., Ltd.
    Full Title: A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient Pre-pubertal Children Treated With Saizen®
    Medical condition: Dwarfism, Pituitary
    Disease: Version SOC Term Classification Code Term Level
    18.0 10028395 - Musculoskeletal and connective tissue disorders 10013883 Dwarfism PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2011-003309-12 Sponsor Protocol Number: P100129 Start Date*: 2011-11-10
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10062282 Silver-Russell syndrome PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    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 (Trial now transitioned) DK (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    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: 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: 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 (Completed) GB (GB - no longer in EU/EEA) DE (Completed) AT (Completed) DK (Completed) PT (Completed)
    Trial results: View results
    EudraCT Number: 2004-000642-21 Sponsor Protocol Number: ASB-03-06 Start Date*: Information not available in EudraCT
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI
    Medical condition: Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome)
    Disease: Version SOC Term Classification Code Term Level
    6.0 10056892 PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IE (Completed)
    Trial results: View results
    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: 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: 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: 2005-003512-30 Sponsor Protocol Number: ASB-008 Start Date*: 2007-04-05
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI)
    Medical condition: Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome)
    Disease: Version SOC Term Classification Code Term Level
    6.0 10056892 PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) PT (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 (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: 2019-001881-14 Sponsor Protocol Number: 20072650 Start Date*: 2021-03-19
    Sponsor Name:Department of Endocrinology and Internal Medicine, Aarhus University Hospital
    Full Title: Endocrine, cardiovascular, pharmacologic and physiologic aspects of sex hormone treatment in Turner syndrome - project part 1
    Medical condition: Ovarian dysgenesis and related hypogonadism caused by Turner syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-002954-21 Sponsor Protocol Number: QBGJ398-201 Start Date*: 2020-05-06
    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: FR (Completed) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2011-000460-10 Sponsor Protocol Number: EMR200104_10 Start Date*: 2011-08-03
    Sponsor Name:Merck Serono SA
    Full Title: First year growth response associated genetic markers validation Phase IV open-label study in Growth Hormone Deficient and Turner Syndrome pre-pubertal children : the PREDICT Pharmacogenetics V...
    Medical condition: idiopathic growth hormone deficienty Turner Syndrome
    Disease: Version SOC Term Classification Code Term Level
    13.1 10014698 - Endocrine disorders 10056438 Growth hormone deficiency PT
    13.1 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) CZ (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-004934-40 Sponsor Protocol Number: 20072651 Start Date*: 2021-03-19
    Sponsor Name:Department of Endocrinology and Internal Medicine, Aarhus University Hospital
    Full Title: Endocrine, cardiovascular, pharmacologic and physiologic aspects of sex hormone treatment in Turner syndrome - project part B
    Medical condition: Ovarian dysgenesis and related hypogonadism causes by Turner syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2009-010280-17 Sponsor Protocol Number: TSHYPE-2601-2009 Start Date*: 2009-04-17
    Sponsor Name:Organisation name was not entered
    Full Title: TS HYPE - Turner syndrome and Hypertension; a double-blinded randomised interventional trial.
    Medical condition: Turner syndrome, hypertension and aortopathy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10045181 Turner's syndrome LLT
    9.1 10020772 Hypertension LLT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2010-022370-14 Sponsor Protocol Number: 1015202 Start Date*: Information not available in EudraCT
    Sponsor Name:Centre Hospitalier de Toulouse
    Full Title: Evaluation de la tolérance d'une administration intra-nasale d'ocytocine chez des nourrissons présentant un syndrome de Prader-Willi et de son effet sur la succion et la prise alimentaire.
    Medical condition: Syndrome de Prader Willi
    Disease: Version SOC Term Classification Code Term Level
    12.1 10036476 Prader-Willi syndrome LLT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
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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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