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Clinical trials for Skeletal dysplasia

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   44334   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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    11 result(s) found for: Skeletal dysplasia. Displaying page 1 of 1.
    EudraCT Number: 2006-003549-18 Sponsor Protocol Number: 004/001 Start Date*: Information not available in EudraCT
    Sponsor Name:Cliniques Universitaires SAINT-LUC
    Full Title: REDUCTION OF BONE MORBIDITY USING AN ORAL BISPHOSPHONATE IN FIBROUS DYSPLASIA OF BONE (PROFIDYS Trial)
    Medical condition: Fibrous dysplasia •Study I: patients with fibrous dysplasia of bone, with bone pain intensity above 3 on a visual analogical scale from 0 to 10. •Study II: patients with fibrous dysplasia of bone...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10016664 Fibrous dysplasia of bone PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-024282-41 Sponsor Protocol Number: 2010.648 Start Date*: 2012-12-05
    Sponsor Name:Hospices Civils de Lyon
    Full Title: TRAITEMENT DE LA DYSPLASIE FIBREUSE DES OS CHEZ LES PATIENTS NON REPONDEURS AUX BISPHOSPHONATES PAR LE TOCILIZUMAB. L’ESSAI TOCIDYS
    Medical condition: dysplasie fibreuse des os
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10016664 Fibrous dysplasia of bone PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-002611-29 Sponsor Protocol Number: 82050 Start Date*: 2023-06-12
    Sponsor Name:Leiden University Medical Center
    Full Title: DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD): a randomized double-blind placebo-controlled trial
    Medical condition: Fibrous Dysplasia/ McCune Albright Syndrome
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-002633-38 Sponsor Protocol Number: 08781 Start Date*: 2018-11-19
    Sponsor Name:The Newcastle upon Tyne Hospitals NHS Foundation Trust
    Full Title: An open label phase I/IIa trial repurposing carbamazepine (CBZ) for the treatment of skeletal dysplasia in children.
    Medical condition: Metaphyseal chondrodysplasia type Schmid (MCDS)
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Completed)
    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: 2020-001109-22 Sponsor Protocol Number: PBGM01-001 Start Date*: Information not available in EudraCT
    Sponsor Name:Passage Bio, Inc.
    Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered into the Cisterna Magna of Pediatric Su...
    Medical condition: GM1 gangliosidosis (GM1) is an autosomal recessive disorder that results from mutations in the human galactosidase beta 1 gene (GLB1), which encodes beta-galactosidase (β-gal).
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2013-005373-43 Sponsor Protocol Number: LUM001-304 Start Date*: 2014-08-05
    Sponsor Name:Lumena Pharmaceuticals Inc
    Full Title: Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients wit...
    Medical condition: Alagille Syndrome
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10053870 Alagille syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed) PL (Completed) BE (Completed) GB (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2016-003181-12 Sponsor Protocol Number: HOTPOT1 Start Date*: 2016-10-07
    Sponsor Name:Royal College of Surgeons in Ireland
    Full Title: Can sonographic assessment of pulmonary vascular reactivity following maternal hyperoxygenation therapy predict neonatal outcome in fetuses at risk of pulmonary hypertension?
    Medical condition: Persistent pulmonary hypertension of the newborn (PPHN) is a relatively common condition occurring in 7/1000 births and can result in significant cardiovascular instability in the newborn. It occur...
    Disease:
    Population Age: Adults Gender: Female
    Trial protocol: IE (Completed)
    Trial results: View results
    EudraCT Number: 2016-002328-10 Sponsor Protocol Number: TIGEM1-MPSVI Start Date*: 2018-10-29
    Sponsor Name:FONDAZIONE TELETHON
    Full Title: A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects with Mucopolysaccharidosis type VI (MPS VI) Using Adeno-Associated Viral Vector 8 to Deliver the human ARSB gene to Liver.
    Medical condition: The clinical trial will be conducted on patients with Mucopolysaccharidosis Type VI. MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal d...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Ongoing) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2010-021048-16 Sponsor Protocol Number: MOR-100 Start Date*: 2010-10-07
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2010-020199-45 Sponsor Protocol Number: MOR-005 Start Date*: 2011-06-08
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Medical condition: Mucopolysaccharidosis Type IVA
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10028095 Mucopolysaccharidosis IV PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) IT (Completed) PT (Completed) DK (Completed) ES (Completed)
    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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