4 completed, 4 recruiting, 3 unknown, 2 active not recruiting. 4 Phase 2, 2 Not applicable, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 1, Completed 1, Recruiting 1, Unknown 1 |
| Not applicable | 2 | Recruiting 2 |
| Phase 1 | 2 | Recruiting 1, Unknown 1 |
Follow Fanconi Syndrome: new trials by email Literature Review of Fanconi Syndrome trials Research Report on Fanconi Syndrome
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 |
|---|---|---|---|---|---|---|
| Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease Lactate Meter, Accu Chek - Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I | Recruiting | Connecticut Children's Medical Center | 10 | 2026-08-01 | 2026-06-01 | |
| DFT383 in Pediatric Participants With Nephropathic Cystinosis DFT383 - Nephropathic Cystinosis | Phase 1 / Phase 2 | Recruiting | Novartis Pharmaceuticals | 30 | 2025-06-02 | 2026-10-16 |
| A Study Comparing the Necessity of a Second Transurethral Resection in High-Risk Non-Muscle-Invasive Bladder Cancer Patients With Negative Results From Post-Initial Resection Urine Genome-Wide Low-Depth Sequencing Urine biomarker -Guided without reTURBT - Non Muscle Invasive Bladder Cancer | Not applicable | Recruiting | The First Affiliated Hospital with Nanjing Medical University | 428 | 2025-02-01 | 2025-06-25 |
| The Necessity of a Second Transurethral Resection in High-risk Non-muscle-invasive Bladder Cancer Patients With Negative Urine Biomarker After Initial Transurethral Resection Urine biomarker -Guided without reTURBT - Non Muscle Invasive Bladder Cancer | Not applicable | Recruiting | The First Affiliated Hospital with Nanjing Medical University | 428 | 2023-07-01 | 2024-03-01 |
| Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease Blood sampling - Fanconi Syndrome | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 18 | 2023-06-01 | 2023-06-15 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Gene Therapy for Fanconi Anemia, Complementation Group A RP-L102 - Fanconi Anemia Complementation Group A | Phase 2 | Active not recruiting | Rocket Pharmaceuticals Inc. | 5 | 2020-07-15 | 2025-12-22 |
| Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy Safety and efficacy assessments - Fanconi Anemia Complementation Group A, Fanconi Anemia | Active not recruiting | Rocket Pharmaceuticals Inc. | 9 | 2020-06-01 | 2026-04-13 | |
| Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A RP-L102 - Fanconi Anemia Complementation Group A | Phase 2 | Unknown | Rocket Pharmaceuticals Inc. | 7 | 2019-11-28 | 2024-05-22 |
| A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A RP-L102 - Fanconi Anemia Complementation Group A | Phase 1 | Unknown | Rocket Pharmaceuticals Inc. | 2 | 2019-01-11 | 2020-11-24 |
| Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis) Renal Fanconi Syndrome, Photophobia | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| Cure Cystinosis International Registry Cystinosis, Nephropathic Cystinosis | Unknown | Cystinosis Research Foundation | 750 | 2010-08-01 | 2014-03-12 | |
| Fanconi Syndrome Due to ARVs in HIV-Infected Persons Blood Draws - HIV Infections, Fanconi Syndrome | Completed | Gilead Sciences | 56 | 2007-09-01 | 2014-03-17 | |
| Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias Deferasirox - Beta-thalassemia, Myelodysplastic Syndromes | Phase 2 | Completed | Novartis Pharmaceuticals | 175 | 2003-05-01 | 2017-08-22 |
| Evaluation of Fanconi Syndrome and Cystinosis Cystinosis, Fanconi Syndrome | Completed | National Center for Research Resources (NCRR) | 12 | 1999-10-01 | 2005-06-24 |
Cystinosis (4) · Anemia (3) · Bone Marrow Failure Disorders (3) · Anemia, Diamond-Blackfan (2) · Fanconi Anemia (2) · Non-Muscle Invasive Bladder Neoplasms (2) · Rare Diseases (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency (1) · Acidemia, isovaleric (1) · Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1) · Adrenal Hyperplasia, Congenital (1) · Adrenoleukodystrophy (1) · Andersen Syndrome (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Female Urogenital Diseases (15) · Female Urogenital Diseases and Pregnancy Complications (15) · Male Urogenital Diseases (15) · Renal Tubular Transport, Inborn Errors (15) · Urologic Diseases (15) · Genetic Diseases, Inborn (14) · Kidney Diseases (14) · Urogenital Diseases (14) · Metabolic Diseases (8) · Nutritional and Metabolic Diseases (8) · Bone Marrow Diseases (5) · Hematologic Diseases (5) · Hemic and Lymphatic Diseases (5) · Metabolism, Inborn Errors (5)
Blood Specimen Collection (2) · Deferasirox (1) · Safety (1)
Rocket Pharmaceuticals Inc. (4) · Novartis Pharmaceuticals (2) · The First Affiliated Hospital with Nanjing Medical University (2) · Assistance Publique - Hôpitaux de Paris (1) · CENTOGENE GmbH Rostock (1) · Centre Hospitalier Universitaire de Liege (1) · Connecticut Children's Medical Center (1) · Cystinosis Research Foundation (1) · Gilead Sciences (1) · National Center for Research Resources (NCRR) (1)
United States (7) · China (2) · Spain (2) · Belgium (1) · Canada (1) · Germany (1) · India (1) · Sri Lanka (1) · United Kingdom (1)
Agnieszka Czechowicz, MD (1) · Jerry A Schneider, M.D. (1) · Jerry A. Schneider (1) · John Flaherty, PharmD (1) · Laurent Servais (1) · Malaya Mount, MS, RD, CDN (1) · Margaret MacMillan, MD, MSc (1) · Peter Bauer, Prof. (1) · Rajni Agarwal, MD (1) · Rebecca Riba-Wolman, MD (1)
PMID 20716238, PMID 22205430, PMID 39642902
2018 (1)