246. Methylmalonic acidemia
14 clinical trials,   22 drugs   (DrugBank: 2 drugs),   1 drug target gene,   6 drug target pathways

Searched query = "Methylmalonic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
6 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
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PhaseCountries
1NCT04284917
(ClinicalTrials.gov)
November 28, 201924/2/2020Long-term Efficacy of Carglumic Acid in Organic Acidemia.Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)Drug: Carglumic Acid (Carbaglu®)National Taiwan University HospitalNULLEnrolling by invitation18 YearsN/AAll5N/ATaiwan
2NCT04176523
(ClinicalTrials.gov)
January 15, 201920/11/2019Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachUnderstanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachMethylmalonic Acidemia;Propionic AcidemiaDrug: Carglumic AcidRecordati Rare DiseasesNULLRecruiting6 Months99 YearsAll75France;Italy;Norway;Spain;United Kingdom
3NCT02426775
(ClinicalTrials.gov)
November 201522/4/2015Carglumic Acid in Methylmalonic Acidemia and Propionic AcidemiaRandomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)Propionic Acidemia;Methylmalonic AcidemiaDrug: Carglumic AcidKing Abdullah International Medical Research CenterNULLCompletedN/A15 YearsAll33Phase 3Saudi Arabia
4NCT01599286
(ClinicalTrials.gov)
September 201211/5/2012Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute HyperammonemiaShort-Term Outcome of N-Carbamylglutamate in the Treatment of Acute HyperammonemiaPropionic Acidemia, Type I and/or Type II;Methylmalonic Acidemia;Carbamoyl-Phosphate Synthase I Deficiency Disease;Ornithine Carbamoyltransferase DeficiencyDrug: Carbaglu;Drug: Placebo;Drug: Standard of Care TreatmentMendel TuchmanChildren's National Research Institute;Boston Children's Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Stanford University;Icahn School of Medicine at Mount Sinai;University of Pittsburgh;Children's Hospital ColoradoActive, not recruitingN/A99 YearsAll114Phase 2United States
5NCT01597440
(ClinicalTrials.gov)
September 201210/5/2012Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic AcidemiaLong-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic AcidemiaPropionic Acidemia;Methylmalonic AcidemiaDrug: N-carbamylglutamate;Other: Standard of CareMendel TuchmanChildren's Research Institute;Boston Children’s Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Lucile Packard Children's Hospital;University of Colorado, DenverTerminatedN/A4 WeeksAll1Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT01341379
(ClinicalTrials.gov)
December 201022/4/2011Increasing Ureagenesis in Inborn Errors of Metabolism With N-CarbamylglutamateIncreasing Ureagenesis in Inborn Errors of Metabolism With N-CarbamylglutamateUrea Cycle Disorders, Inborn;Inborn Errors of Metabolism;Propionic Acidemia;Methylmalonic Acidemia;Carbamyl Phosphate Synthetase DeficiencyDrug: N-carbamylglutamateChildren's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Withdrawn3 Years70 YearsBoth0Phase 2United States