337. Homocystinuria Clinical trials / Disease details
Clinical trials : 16 / Drugs : 19 - (DrugBank : 7) / Drug target genes : 2 - Drug target pathways : 23
| 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 | NCT05154890 (ClinicalTrials.gov) | May 13, 2021 | 20/8/2021 | A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency | A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177 | Homocystinuria Due to Cystathionine Beta-Synthase Deficiency | Drug: Pegtarviliase | Aeglea Biotherapeutics | NULL | Recruiting | 12 Years | N/A | All | 36 | Phase 1/Phase 2 | United States;Australia;United Kingdom |
| 2 | EUCTR2019-004791-19-GB (EUCTR) | 11/05/2020 | 14/01/2020 | A Phase 1/2 Study of ACN00177 in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency | A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177 - SA notification 100D New site and PI | Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency MedDRA version: 20.0;Level: PT;Classification code 10020365;Term: Homocystinuria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: ACN00177 | Aeglea Biotherapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 1 | United Kingdom |