Pyruvate Metabolism, Inborn Errors
Showing 26 - 50 of 6,660
Induced Pluripotent Stem Cells for Development of Novel Drug
Recruiting
- Inborn Errors of Metabolism
- Skin biopsy
- Withdraw of blood
-
Lahore, PakistanChildren Hospital and Institute of Child Health
May 14, 2021
Carbohydrate Intolerance Trial in Springfield (Low-FODMAP diet group (LFD), Low Sugar diet group (LAS))
Recruiting
- Carbohydrate Intolerance
- Low-FODMAP diet group (LFD)
- Low Sugar diet group (LAS)
-
Springfield, OregonOregon Health and Science University
Sep 21, 2023
COVID-19 and Hereditary Metabolic Diseases
Recruiting
- Covid19
- Metabolism, Inborn Errors
-
Lille, FranceHop Claude Huriez Chu Lille
Feb 28, 2022
Medium-chain Acyl-CoA Dehydrogenase Deficiency Trial in Pittsburgh (Triheptanoin)
Recruiting
- Medium-chain Acyl-CoA Dehydrogenase Deficiency
-
Pittsburgh, PennsylvaniaUPMC Children's Hospital of Pittsburgh
Sep 27, 2023
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) Trial (Triheptanoin)
Available
- Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
- (no location specified)
Nov 22, 2022
A Non-Interventional Post-Authorization Study of Carbaglu for
Enrolling by invitation
- Hyperammonemia
- +2 more
- carglumic acid
-
Indianapolis, IndianaRiley Children's Hospital
Apr 24, 2023
Medium-chain Acyl-CoA Dehydrogenase Deficiency Trial in Pittsburgh (Sodium phenylbutyrate)
Recruiting
- Medium-chain Acyl-CoA Dehydrogenase Deficiency
- Sodium phenylbutyrate
-
Pittsburgh, PennsylvaniaUPMC Children's Hospital of Pittsburgh
Oct 4, 2023
Mitochondrial Diseases, Pearson Syndrome Trial in Ramat Gan (MNV-201)
Recruiting
- Mitochondrial Diseases
- Pearson Syndrome
- MNV-201
-
Ramat Gan, IsraelSheba Medical Center
Aug 24, 2023
Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem
Active, not recruiting
- Hematologic Malignancies
- +7 more
-
San Francisco, CaliforniaUniversity of California, San Francisco
Feb 28, 2022
Urea Cycle Disorder, Inborn Errors of Metabolism Trial in Philadelphia (UCD Anamix Infant)
Completed
- Urea Cycle Disorder
- Inborn Errors of Metabolism
- UCD Anamix Infant
-
Philadelphia, PennsylvaniaChildren's hospital of Philadelphia
Dec 8, 2021
Methylmalonic Acidemia (MMA) Trial run by the Selecta Biosciences, Inc. (SEL-302)
Recruiting
- Methylmalonic Acidemia (MMA)
-
Bethesda, MarylandNational Human Genome Research Institute, National Institutes of
Mar 9, 2023
Pyruvate Kinase Deficiency, Congenital Non-Spherocytic Hemolytic Anemia Trial in Worldwide
Completed
- Pyruvate Kinase Deficiency
- Congenital Non-Spherocytic Hemolytic Anemia
-
Phoenix, Arizona
- +29 more
May 20, 2020
Cardiac Manifestation and Inherited Metabolic Diseases
Recruiting
- Metabolism, Inborn Errors
-
Lausanne, Vaud, SwitzerlandLausanne University Hospital
Aug 7, 2021
Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes Trial in Philadelphia (Transplant of stem cells with
Recruiting
- Leukemia
- +4 more
- Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
-
Philadelphia, PennsylvaniaChildren's Hospital of Philadelphia
Nov 2, 2021
Methylmalonic Acidemia, Propionic Acidemia Trial in San Francisco, Remote (Registry)
Recruiting
- Methylmalonic Acidemia
- Propionic Acidemia
- Registry
-
San Francisco, California
- +2 more
Jun 27, 2022
Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell
Completed
- Hematologic Malignancies
- +7 more
-
San Francisco, CaliforniaUniversity of California, San Francisco
Sep 27, 2021
Fludarabine Drug Exposure in Pediatric Bone Marrow
Completed
- Hematologic Malignancies
- +7 more
-
San Francisco, CaliforniaUniversity of California, San Francisco
Sep 27, 2021
GLUT1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter type1 (GLUT-1) Deficiency Trial
Recruiting
- GLUT1 Deficiency Syndrome
- +3 more
- No intervention
-
Dallas, TexasUT Southwestern Medical Center
Feb 1, 2022
Clofarabine and Fludarabine Drug Exposure in Pediatric Bone
Completed
- Hematologic Malignancies
- +7 more
- Clofarabine
- Fludarabine Injection
-
San Francisco, CaliforniaUniversity of California, San Francisco
Sep 27, 2021
Methylmalonic Acidemia Trial in United States (hLB-001)
Recruiting
- Methylmalonic Acidemia
- hLB-001
-
Atlanta, Georgia
- +3 more
Aug 16, 2022
Measurements in Inborn Errors of Metabolism
Completed
- Inborn Errors of Metabolism
- +7 more
- Survey
-
Zurich, SwitzerlandUniversity Children's Hospital Zurich
Nov 3, 2020
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies Trial in Durham (CliniMACS CD34 Reagent
Available
- Hematologic Malignancies
- +2 more
- CliniMACS CD34 Reagent System
-
Durham, North CarolinaDuke University Medical Center
Nov 12, 2021
Congenital Sucrase-Isomaltase Deficiency, CSID, Sucrase Isomaltase Deficiency Trial in Buffalo, Oklahoma City (Sucraid
Recruiting
- Congenital Sucrase-Isomaltase Deficiency
- +2 more
- Sucraid (sacrosidase) Oral Solution 8500 IU/mL
-
Buffalo, New York
- +1 more
Aug 16, 2022
Carbaglu® for Treatment of MMA and PA in Adults and Pediatrics
Recruiting
- Hyperammonemia
- +2 more
- Carglumic Acid
-
Washington, District of Columbia
- +1 more
Jun 30, 2022