245. Propionic acidemia Clinical trials / Disease details


Clinical trials : 13 Drugs : 17 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathways : 6

  
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05040178
(ClinicalTrials.gov)
March 202216/6/2021A Surveillance Protocol of Carbaglu® for the Treatment of HyperammonemiaA Surveillance Protocol of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient PopulationsHyperammonemiaDrug: Carglumic AcidRecordati Rare DiseasesNULLNot yet recruitingN/AN/AAll10United States
2NCT05130437
(ClinicalTrials.gov)
November 9, 20213/11/2021A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 StudyPropionic AcidemiaBiological: mRNA-3927ModernaTX, Inc.NULLRecruiting1 YearN/AAll36Phase 1/Phase 2United States;Canada;United Kingdom
3NCT04159103
(ClinicalTrials.gov)
April 15, 20217/11/2019Open-Label Study of mRNA-3927 in Participants 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 AcidemiaPropionic AcidemiaBiological: mRNA-3927ModernaTX, Inc.NULLRecruiting1 YearN/AAll36Phase 1/Phase 2United States;Canada;United Kingdom
4NCT04836494
(ClinicalTrials.gov)
March 25, 202129/3/2021A 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 AcidemiaA First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic AcidemiaHealthy Volunteers;Propionic Acidemia;Methylmalonic Acidemia;Organic AcidemiaDrug: BBP-671;Drug: PlaceboCoA Therapeutics, Inc., a BridgeBio companyNULLRecruiting18 Years55 YearsAll128Phase 1United States
5NCT04732429
(ClinicalTrials.gov)
March 15, 202121/1/2021Study of HST5040 in Subjects With Propionic or Methylmalonic AcidemiaA Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension StudyMethylmalonic Acidemia;Propionic AcidemiaDrug: HST5040;Drug: PlaceboHemoShear TherapeuticsNULLRecruiting2 YearsN/AAll12Phase 2United States
6EUCTR2019-003529-36-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
7NCT04284917
(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
8NCT04176523
(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
9NCT02426775
(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
10NCT01599286
(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
11NCT01597440
(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
12NCT01341379
(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
13NCT00645879
(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