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Results for “Organic Acidemia”

18 trials

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Showing 18 of 18 results

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 applicableEnrolling By InvitationNCT06953505
What this trial is testing

At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism

Who this might be right for
Urea Cycle DisordersOrganic AcidemiasFatty Acid Oxidation Disorder+1 more
Sequitur Health Corp. 30
Not applicableStudy completedNCT03917212
What this trial is testing

Energy Metabolism in Branched-chain Organic Acidemias

Who this might be right for
Energy MetabolismMetabolic SyndromeInsulin Resistance
German Diabetes Center 57
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 applicableLooking for participantsNCT04602325
What this trial is testing

Systemic Biomarkers of Brain Injury From Hyperammonemia

Who this might be right for
Urea Cycle DisorderOrganic AcidemiaMaple Syrup Urine Disease+3 more
Children's National Research Institute 24
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
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
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
Not applicableStudy completedNCT04904705
What this trial is testing

The Effect of the Cognitive Orientation to Daily Occupational Performance Approach for Children With Organic Acidemia

Who this might be right for
Organic AcidemiaCognitive Orientation
Hacettepe University 34
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 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 participantsNCT04176523
What this trial is testing

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

Who this might be right for
Methylmalonic AcidemiaPropionic Acidemia
RECORDATI GROUP 95
Testing effectiveness (Phase 2)WithdrawnNCT05778877
What this trial is testing

Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA

Who this might be right for
Methylmalonic Acidemia (MMA)
Selecta Biosciences, Inc.
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
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 completedNCT02322177
What this trial is testing

Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol

Who this might be right for
Inborn Errors of MetabolismPregnancyAcidemias
National Human Genome Research Institute (NHGRI) 2
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