6 completed, 1 recruiting, 1 terminated. 8 Phase 2, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Completed 6, Recruiting 1, Terminated 1 |
| Phase 1 | 2 | Completed 2 |
Follow VLCAD deficiency: new trials by email Literature Review of VLCAD deficiency trials Research Report on VLCAD deficiency
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS) MNV-201 - Mitochondrial Diseases, Pearson Syndrome | Phase 2 | Recruiting | Minovia Therapeutics Ltd. | 6 | 2023-07-31 | 2026-10-13 |
| A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome CD34+ cells enriched with MNV-BLD - Mitochondrial Diseases, Pearson Syndrome | Phase 1 / Phase 2 | Completed | Minovia Therapeutics Ltd. | 7 | 2019-02-13 | 2021-08-31 |
| Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies UX007 - Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 94 | 2014-12-09 | 2023-08-01 |
| Phase 2 Study of EPI-743 in Children With Pearson Syndrome EPI-743 - Pearson Syndrome | Phase 2 | Terminated | Edison Pharmaceuticals Inc | 2 | 2014-08-31 | 2020-11-18 |
| A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) UX007 - Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency | Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 29 | 2014-02-06 | 2021-02-11 |
| Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder Triheptanoin - Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency | Phase 2 | Completed | Oregon Health and Science University | 32 | 2011-09-01 | 2017-03-23 |
| Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects Bezafibrate, Placebo - Carnitine Palmitoyltransferase II Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency | Phase 2 | Completed | Copenhagen University Hospital, Rigshospitalet | 12 | 2009-10-01 | 2012-05-30 |
| High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders Diet counseling - Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency | Phase 1 / Phase 2 | Completed | Oregon Health and Science University | 13 | 2006-01-01 | 2020-04-29 |
Mitochondrial Diseases (3) · Carnitine palmitoyl transferase 2 deficiency (2) · Rare Diseases (1) · Trifunctional Protein Deficiency With Myopathy And Neuropathy (1)
Metabolic Diseases (3) · Nutritional and Metabolic Diseases (3) · Disease Attributes (1) · Pathologic Processes (1) · Pathological Conditions, Signs and Symptoms (1)
triheptanoin (3) · Bezafibrate (1) · Diet, Fat-Restricted (1) · Diet, High-Protein (1) · Diet, High-Protein Low-Carbohydrate (1) · alpha-tocotrienol quinone (1)
Minovia Therapeutics Ltd. (2) · Oregon Health and Science University (2) · Ultragenyx Pharmaceutical Inc (2) · Copenhagen University Hospital, Rigshospitalet (1) · Edison Pharmaceuticals Inc (1)
United States (4) · Israel (2) · United Kingdom (2) · Denmark (1)
Matthew Klein, MD, FACS (1) · Melanie B Gillingham, PhD (1)
PMID 28189603, PMID 28871440, PMID 30740733, PMID 33487279, PMID 37276053
2016 (1)