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Clinical trials for Methylmalonic acidemia

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   44364   clinical trials with a EudraCT protocol, of which   7388   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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    5 result(s) found for: Methylmalonic acidemia. Displaying page 1 of 1.
    EudraCT Number: 2020-004980-24 Sponsor Protocol Number: mRNA-3705-P101 Start Date*: 2023-08-21
    Sponsor Name:ModernaTX, Inc.
    Full Title: A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidem...
    Medical condition: Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) FR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2023-000785-33 Sponsor Protocol Number: HST20-CL01 Start Date*: 2023-08-18
    Sponsor Name:HemoShear Therapeutics
    Full Title: A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects with Propionic or Methylmalonic Acidemia Followed by a 6-Month, Randomized, Double-blind, Placebo-controlled, 2-Period Crossover S...
    Medical condition: Methylmalonic Acidemia (MMA) Propionic Acidemia (PA)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-002120-33 Sponsor Protocol Number: HLSCS01-11 Start Date*: 2013-12-06
    Sponsor Name:AZIENDA SANITARIA OSPEDALIERA O.I.R.M. - S. ANNA
    Full Title: Human Liver Stem Cells (HLSCs) in patients suffering from liver-based inborn metabolic diseases causing life-threatening neonatal onset of hyperammonemic encephalopathy
    Medical condition: neonatal hyperammonemic encephalopathy associated with inborn liver metabolic diseases
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10059521 Methylmalonic aciduria PT
    14.1 10010331 - Congenital, familial and genetic disorders 10058299 Argininosuccinate lyase deficiency PT
    14.1 10010331 - Congenital, familial and genetic disorders 10052450 Ornithine transcarbamoylase deficiency PT
    14.1 10010331 - Congenital, familial and genetic disorders 10058297 Carbamoyl phosphate synthetase deficiency PT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-001061-32 Sponsor Protocol Number: mRNA-3704-P101 Start Date*: 2019-10-25
    Sponsor Name:ModernaTX, Inc.
    Full Title: A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients with Isolated Methylmalonic Acidemia Due to Methylmalo...
    Medical condition: Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-016654-41 Sponsor Protocol Number: PTC124-GD-012-MMA Start Date*: 2010-03-02
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia
    Medical condition: Nonsense Mutation Methylmalonic Acidemia
    Disease: Version SOC Term Classification Code Term Level
    12.1 10064180 Blood methylmalonic acid increased LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) GB (Prematurely Ended) IT (Prematurely Ended) BE (Prematurely Ended)
    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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