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Results for “Methylmalonic Acidemia (MMA)”

31 trials

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Showing 20 of 31 results

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
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 applicableStudy completedNCT05330039
What this trial is testing

Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)

Who this might be right for
Inborn Errors of MetabolismPropionic AciduriaMethylmalonic Aciduria+1 more
Emma Marie Caroline Slack 22
Early research (Phase 1)Ended earlyNCT04836494
What this trial is testing

A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia

Who this might be right for
Healthy VolunteersPropionic AcidemiaMethylmalonic Acidemia+1 more
CoA Therapeutics, Inc., a BridgeBio company 79
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 applicableLooking for participantsNCT00078078
What this trial is testing

Clinical and Laboratory Study of Methylmalonic Acidemia

Who this might be right for
Organic AcidemiaMethylmalonic AcidemiaInborn Errors of Metabolism
National Human Genome Research Institute (NHGRI) 2,275
Not applicableNot Yet RecruitingNCT06664840
What this trial is testing

MyRareDiet A Novel Diet Tracking Tool

Who this might be right for
Urea Cycle DisorderPropionic AciduriaMaple Syrup Urine Disease+1 more
Oregon Health and Science University 60
Not applicableActive Not RecruitingNCT05506254
What this trial is testing

Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy

Who this might be right for
Methylmalonic Acidemia
Alexion Pharmaceuticals, Inc. 4
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)Study completedNCT01624311
What this trial is testing

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

Who this might be right for
Urea Cycle DisordersOrganic Acidemias
Uta Lichter-Konecki 5
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 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 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 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
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
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.
Testing effectiveness (Phase 2)Looking for participantsNCT05295433
What this trial is testing

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705

Who this might be right for
Methylmalonic Acidemia
ModernaTX, Inc. 56
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
Testing effectiveness (Phase 2)Ended earlyNCT04899310
What this trial is testing

Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

Who this might be right for
Methylmalonic Acidemia
ModernaTX, Inc. 18
Not applicableStudy completedNCT04248062
What this trial is testing

Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism

Who this might be right for
Inborn Errors of MetabolismUrea Cycle DisorderMaple Syrup Urine Disease+5 more
University Children's Hospital, Zurich 69
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