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Results for “Isolated Methylmalonic Acidemia”

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Showing 10 of 0 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 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
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)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
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 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 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
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
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
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