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Clinical trials for Methylmalonic Acid

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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    9 result(s) found for: Methylmalonic Acid. 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) NL (Ongoing)
    Trial results: (No results available)
    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: 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: 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: 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
    EudraCT Number: 2018-003619-23 Sponsor Protocol Number: 6630-4200-42 Start Date*: 2019-11-29
    Sponsor Name:MEDICE Arzneimittel Pütter GmbH & Co. KG
    Full Title: EFFICACY AND SAFETY OF A PARENTERALLY ADMINISTERED FIXED VITAMIN COMBINATION (VITAMIN B6, B12 AND FOLIC ACID) ON VITAMIN B12 STATUS AND METHYLATION CAPACITY IN COBALAMIN DEFICIENCY COMPARED TO ORAL...
    Medical condition: vitamin B12 deficiency
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-000356-18 Sponsor Protocol Number: 25051974 Start Date*: 2023-12-21
    Sponsor Name:Stichting Apotheek der Haarlemse Ziekenhuizen
    Full Title: Effect of repeated intranasal cobalamin administration on cobalamin deficiency in elderly
    Medical condition: Cobalamin (vitamin B12) deficiency
    Disease:
    Population Age: Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-015844-41 Sponsor Protocol Number: BMN162-502 Start Date*: 2013-07-25
    Sponsor Name:BioMarin International Ltd.
    Full Title: A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years.
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10034873 Phenylketonuria (PKU) LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) IT (Ongoing) ES (Completed)
    Trial results: View results
    EudraCT Number: 2018-002217-35 Sponsor Protocol Number: AG348-C-010 Start Date*: 2018-11-20
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 2, Open-Label, Multicenter Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Subjects With Non-Transfusion-Dependent Thalassemia
    Medical condition: Non-Transfusion-Dependent Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    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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