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Results for “Methylmalonic Acidemia”

32 trials

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Showing 20 of 32 results

Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Not applicableLooking for participantsNCT04176523
What this trial is testing

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

Who this might be right for
Methylmalonic AcidemiaPropionic Acidemia
RECORDATI GROUP 95
Not applicableUnknownNCT01289158
What this trial is testing

Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study

Who this might be right for
Malonic AciduriaMethylmalonic Acidemia
McGill University Health Centre/Research Institute of the McGill University Health Centre 6
Testing effectiveness (Phase 2)Study completedNCT01599286
What this trial is testing

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

Who this might be right for
Propionic Acidemia, Type I and/or Type IIMethylmalonic AcidemiaCarbamoyl-Phosphate Synthase I Deficiency Disease+1 more
Mendel Tuchman 35
Not applicableStudy completedNCT03484767
What this trial is testing

"The MaP Study": Mapping the Patient Journey in MMA and PA

Who this might be right for
Methylmalonic AcidemiaPropionic Acidemia
ModernaTX, Inc. 97
Testing effectiveness (Phase 2)WithdrawnNCT05778877
What this trial is testing

Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA

Who this might be right for
Methylmalonic Acidemia (MMA)
Selecta Biosciences, Inc.
Not applicableEnded earlyNCT05438485
What this trial is testing

Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia

Who this might be right for
Methylmalonic AcidemiaPropionic Acidemia
HemoShear Therapeutics 11
Not applicableUnknownNCT05910151
What this trial is testing

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Who this might be right for
Propionic/Methylmalonic AcidemiasMaple Syrup Urine DiseaseCitrullinemia+26 more
West Kazakhstan Medical University 2,250
Testing effectiveness (Phase 2)Ended earlyNCT01597440
What this trial is testing

Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

Who this might be right for
Propionic AcidemiaMethylmalonic Acidemia
Mendel Tuchman 1
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Testing effectiveness (Phase 2)WithdrawnNCT01341379
What this trial is testing

Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate

Who this might be right for
Urea Cycle Disorders, InbornInborn Errors of MetabolismPropionic Acidemia+2 more
Children's Hospital of Philadelphia
Testing effectiveness (Phase 2)Study completedNCT01793090
What this trial is testing

EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment

Who this might be right for
Methylmalonic Aciduria and Homocystinuria,Cblc TypeGenetic DiseaseRetinopathy
Bambino Gesù Hospital and Research Institute 30
Not applicableNot Yet RecruitingNCT07432880
What this trial is testing

A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene

Who this might be right for
Methylmalonic Acidemia (MMA)
Genespire Srl 30
Testing effectiveness (Phase 2)Ended earlyNCT04732429
What this trial is testing

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

Who this might be right for
Methylmalonic AcidemiaPropionic Acidemia
HemoShear Therapeutics 26
Not applicableStudy completedNCT04284917
What this trial is testing

Long-term Efficacy of Carglumic Acid in Organic Acidemia.

Who this might be right for
Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)
National Taiwan University Hospital 3
Large-scale testing (Phase 3)Study completedNCT02426775
What this trial is testing

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

Who this might be right for
Propionic AcidemiaMethylmalonic Acidemia
King Abdullah International Medical Research Center 33
Testing effectiveness (Phase 2)Ended earlyNCT01141075
What this trial is testing

Ataluren for Nonsense Mutation Methylmalonic Acidemia

Who this might be right for
Amino Acid Metabolism, Inborn Errors
PTC Therapeutics 11
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableStudy completedNCT03539809
What this trial is testing

Branched Chain Amino Acids Ratio in Medical Foods

Who this might be right for
Children
University of British Columbia 8
Testing effectiveness (Phase 2)WithdrawnNCT03810690
What this trial is testing

Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

Who this might be right for
Methylmalonic Acidemia (MMA)Metabolism, Inborn Errors
ModernaTX, Inc.
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