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Results for “Methylmalonicacidemia”

30 trials

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Showing 20 of 30 results

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
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 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
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
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
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 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
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
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
Testing effectiveness (Phase 2)Ended earlyNCT04581785
What this trial is testing

Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia

Who this might be right for
Methylmalonic Acidemia
LogicBio Therapeutics, Inc 4
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
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
Large-scale testing (Phase 3)Not Yet RecruitingNCT07163364
What this trial is testing

Evaluate the Effects and Safety of Hydroxocobalamin in Participants With Combined Methylmalonic Academia (cblC Type)

Who this might be right for
Methylmalonic Acidemia (MMA)
CSPC ZhongQi Pharmaceutical Technology Co., Ltd. 20
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 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 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 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
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.
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
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