245. プロピオン酸血症
[臨床試験数:9,薬物数:15(DrugBank:3),標的遺伝子数:1,標的パスウェイ数:6

Searched query = "Propionic 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"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
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agemax
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size
PhaseCountries
1EUCTR2019-003529-36-GB
(EUCTR)
18/12/202003/03/2020A clinical study to evaluate the medicinal product in Patients with Propionic AcidemiaA Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia Propionic Acidemia- Propionic acidemia is an autosomal recessive disorder caused by loss-of-function mutations in PCCA or PCCB (and in rare instances, mutations in both PCCA and PCCB), resulting in the lossof enzymatic activity of the mitochondrial complex PCC
MedDRA version: 21.0;Level: LLT;Classification code 10080615;Term: Propionic acidemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: mRNA-3927
INN or Proposed INN: N/A
Other descriptive name: CX-016492
INN or Proposed INN: NA
Other descriptive name: CX-017950
ModernaTX, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 1;Phase 2United States;Canada;United Kingdom
2NCT04159103
(ClinicalTrials.gov)
March 20207/11/2019Open Label Study of mRNA-3927 in Patients With Propionic AcidemiaA Global, Phase 1/2, Open-label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic AcidemiaPropionic AcidemiaBiological: mRNA-3927ModernaTX, Inc.NULLNot yet recruiting1 YearN/AAll46Phase 1;Phase 2NULL
3NCT04284917
(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
4NCT04176523
(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
5NCT02426775
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT01599286
(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
7NCT01597440
(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
8NCT01341379
(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
9NCT00645879
(ClinicalTrials.gov)
July 200825/3/2008Anaplerotic Therapy in Propionic AcidemiaSafety & Efficacy of Investigational Products: Ornithine Alpha-ketoglutarate, Glutamine, or Disodium Citrate on Hyperammonemia in Propionic Acidemia.Propionic AcidemiaDrug: ornithine alpha ketoglutarate;Drug: glutamine;Drug: disodium citrateNicola LongoNational Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Completed5 Years12 YearsBoth3Phase 1United States