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Clinical trials for Bardet Biedl Syndrome

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   43881   clinical trials with a EudraCT protocol, of which   7295   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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    7 result(s) found for: Bardet Biedl Syndrome. Displaying page 1 of 1.
    EudraCT Number: 2021-004167-27 Sponsor Protocol Number: RM-493-033 Start Date*: 2022-03-02
    Sponsor Name:Rhythm Pharmaceuticals Limited
    Full Title: A Phase 3 Multi-Center, One-Year, Open-Label study of Setmelanotide in Pediatric Patients Aged 2 to <6 years of age with Rare Genetic Causes of Obesity
    Medical condition: POMC deficiency obesity due to mutations in the POMC gene PCSK1 deficiency due to mutations in the PCSK1 gene LEPR deficiency obesity due to mutations in the LEPR gene Bardet-Biedl syndrome
    Disease: Version SOC Term Classification Code Term Level
    23.0 10010331 - Congenital, familial and genetic disorders 10084105 Leptin receptor deficiency PT
    23.0 10010331 - Congenital, familial and genetic disorders 10083937 Pro-opiomelanocortin deficiency PT
    20.1 10010331 - Congenital, familial and genetic disorders 10048680 Bardet-Biedl syndrome LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: NL (Prematurely Ended) ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-004597-65 Sponsor Protocol Number: RM-493-037 Start Date*: 2022-10-20
    Sponsor Name:Rhythm Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) with a Crossover to Open-Label Once Weekly Setmelanotide in Patients with Specific Gene Defects in ...
    Medical condition: Treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway. Diseases that are the result of genetic defects affecting the MC4R pathway, con...
    Disease: Version SOC Term Classification Code Term Level
    23.0 10010331 - Congenital, familial and genetic disorders 10084105 Leptin receptor deficiency PT
    23.0 10010331 - Congenital, familial and genetic disorders 10083937 Pro-opiomelanocortin deficiency PT
    20.1 10010331 - Congenital, familial and genetic disorders 10048680 Bardet-Biedl syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed) DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-004058-11 Sponsor Protocol Number: RM-493-023 Start Date*: Information not available in EudraCT
    Sponsor Name:Rhythm Pharmaceuticals, Inc.
    Full Title: A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström syndrome (AS) Patients with Moderate to Severe Obesity
    Medical condition: Obesity and hyperphagia in patients with Bardet-Biedl Syndrome or Alström syndrome
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) ES (Completed) GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2014-002392-28 Sponsor Protocol Number: RM-493-Supplementation-Therapy Start Date*: 2014-11-28
    Sponsor Name:Charité Univeritaetsmedizin Berlin
    Full Title: Setmelanotide (RM-493) Treatment Trial in Patients with rare genetic disorders of obesity
    Medical condition: Body weight is regulated within the hypothalamus. In rare cases mutations in genes, which are embedded in the signaling cascades of the hypothalamus lead to early onset severe obesity. POMC is one ...
    Disease:
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2014-000385-22 Sponsor Protocol Number: RF-2010-2318561 Start Date*: 2014-10-07
    Sponsor Name:Ospedale San Raffaele di Milano
    Full Title: Nerve Growth Factor eye drops as a novel treatment for vision loss in patients with Retinitis Pigmentosa: from preclinical to clinical Phase II trial.
    Medical condition: Retinitis pigmentosa with cystoid macular edema
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004853 10054467 Macular edema LLT
    17.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-000387-14 Sponsor Protocol Number: RM-493-014 Start Date*: 2017-05-24
    Sponsor Name:Rhythm Pharmaceuticals, Inc.
    Full Title: Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity
    Medical condition: - POMC/PCSK1/LEPR heterozygous - POMC/PCSK1/LEPR compound heterozygous or homozygous deficiency obesity - POMC/PCSK1/LEPR composite heterozygous deficiency obesity - Smith-Magenis Syndrome - SH2B1...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing) DE (Completed) FR (Ongoing) NL (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2017-005006-35 Sponsor Protocol Number: RM-493-022 Start Date*: 2018-05-30
    Sponsor Name:Rhythm Pharmaceuticals, Inc.
    Full Title: Long Term Extension Trial of setmelanotide (RM-493) for patients who have completed a trial of Setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 recepto...
    Medical condition: Patients with obesity caused by genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway. Human Genetics studies have identified several diseases that are the result of genet...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing) FR (Ongoing) NL (Ongoing) GB (GB - no longer in EU/EEA) BE (Ongoing) ES (Ongoing) GR (Ongoing)
    Trial results: (No results available)
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    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
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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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