Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • About
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Methylmalonicacidemia”

29 trials

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 20 of 29 results

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
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)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
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)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)Active Not RecruitingNCT04899310
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. 74
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 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
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)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 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
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
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
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
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)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 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
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 participantsNCT05040178
What this trial is testing

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

Who this might be right for
HyperammonemiaMethylmalonic AcidemiaPropionic Acidemia
RECORDATI GROUP 20
Load More Results