307. カナバン病 Canavan disease Clinical trials / Disease details
臨床試験数 : 5 / 薬物数 : 9 - (DrugBank : 3) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04998396 (ClinicalTrials.gov) | September 8, 2021 | 5/8/2021 | A Study of AAV9 Gene Therapy in Participants With Canavan Disease | A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene | Canavan Disease;Demyelinating Diseases;Genetic Diseases, Inborn;Metabolism, Inborn Errors;Metabolic Disease;Neurological Diseases or Conditions;Leukodystrophy;Leukoencephalopathies;Neurodevelopmental Disorders;Neurodegenerative Diseases | Biological: AAV9 BBP-812 | Aspa Therapeutics | NULL | Recruiting | N/A | 30 Months | All | 18 | Phase 1/Phase 2 | United States |
| 2 | NCT04833907 (ClinicalTrials.gov) | April 1, 2021 | 24/3/2021 | rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease | Phase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of AVASPA Gene Therapy for Treatment of Children With Typical Canavan Disease | Canavan Disease | Drug: rAAV-Olig001-ASPA;Drug: Levetiracetam;Drug: Prednisolone | Myrtelle Inc. | NULL | Recruiting | 3 Months | 60 Months | All | 24 | Phase 1/Phase 2 | United States |
| 3 | NCT00657748 (ClinicalTrials.gov) | September 2009 | 9/4/2008 | Lithium and Acetate for Canavan Disease | Evaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patients With Canavan Disease | Canavan Disease;Infantile;Deficiency Disease;Aspartoacylase;Leukodystrophy, Spongiform | Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug) | Assistance Publique - Hôpitaux de Paris | European Leukodystrophy Association;European Leukodystrophy Association | Withdrawn | 1 Year | 15 Years | Both | 0 | Phase 2 | France |
| 4 | NCT00724802 (ClinicalTrials.gov) | March 2008 | 18/6/2008 | Oral Glyceryl Triacetate (GTA) in Newborns With Canavan | Oral Glyceryl Triacetate (GTA) in Newborns With Canavan | Canavan Disease | Dietary Supplement: GTA (Glyceryl triacetate);Drug: GTA glyceryl triacetate | Shaare Zedek Medical Center | NULL | Recruiting | N/A | 18 Months | Both | 1 | N/A | Israel |
| 5 | NCT00278707 (ClinicalTrials.gov) | January 2006 | 15/1/2006 | GTA-Glyceryltriacetate for Canavan Disease | Phase 1 Treatment With GTA in Two Infant With Canavan Disease | Infantile Canavan Disease;Deficiency Disease, Aspartoacylase | Drug: GTA: Glyceryltriacetate | Sheba Medical Center | NULL | Active, not recruiting | N/A | 15 Months | Both | 5 | Phase 1 | Israel |