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Results for “Propionic Acidemia”

26 trials

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Showing 20 of 26 results

Not applicableStudy completedNCT05330039
What this trial is testing

Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)

Who this might be right for
Inborn Errors of MetabolismPropionic AciduriaMethylmalonic Aciduria+1 more
Emma Marie Caroline Slack 22
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
Not applicableStudy completedNCT03484767
What this trial is testing

"The MaP Study": Mapping the Patient Journey in MMA and PA

Who this might be right for
Methylmalonic AcidemiaPropionic Acidemia
ModernaTX, Inc. 97
Testing effectiveness (Phase 2)Looking for participantsNCT04159103
What this trial is testing

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

Who this might be right for
Propionic Acidemia
ModernaTX, Inc. 77
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)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
Testing effectiveness (Phase 2)Looking for participantsNCT05130437
What this trial is testing

Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

Who this might be right for
Propionic Acidemia
ModernaTX, Inc. 50
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
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
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 applicableLooking for participantsNCT05769621
What this trial is testing

A Retrospective Study to Characterize Participants With Propionic Acidemia

Who this might be right for
Propionic Acidemia
ModernaTX, Inc. 60
Not applicableUnknownNCT05910151
What this trial is testing

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Who this might be right for
Propionic/Methylmalonic AcidemiasMaple Syrup Urine DiseaseCitrullinemia+26 more
West Kazakhstan Medical University 2,250
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 applicableActive Not RecruitingNCT03655223
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
Early research (Phase 1)Study completedNCT00645879
What this trial is testing

Anaplerotic Therapy in Propionic Acidemia

Who this might be right for
Propionic Acidemia
Nicola Longo 3
Not applicableLooking for participantsNCT02890342
What this trial is testing

Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia

Who this might be right for
Metabolic DiseasePropionic AcidemiaOrganic Acidemia
National Human Genome Research Institute (NHGRI) 1,045
Not applicableEnded earlyNCT05438485
What this trial is testing

Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia

Who this might be right for
Methylmalonic AcidemiaPropionic Acidemia
HemoShear Therapeutics 11
Not applicableStudy completedNCT03159026
What this trial is testing

Review of Charts From Amish/Mennonite Variant PA Patients

Who this might be right for
Propionic Acidemia
University of Pittsburgh 38
Not applicableStudy completedNCT03539809
What this trial is testing

Branched Chain Amino Acids Ratio in Medical Foods

Who this might be right for
Children
University of British Columbia 8
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
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