4 completed, 2 terminated, 1 active not recruiting, 1 recruiting. 6 Phase 3, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 6 | Completed 2, Active not recruiting 1, Recruiting 1, Terminated 1, Withdrawn 1 |
| Phase 2 | 4 | Completed 3, Terminated 1 |
Follow IB1001: new trials by email Literature Review of IB1001 trials Research Report on IB1001
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 |
|---|---|---|---|---|---|---|
| A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T) N-Acetyl-L-Leucine, Placebo - Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T) | Phase 3 | Active not recruiting | IntraBio Inc | 60 | 2025-03-18 | 2025-07-03 |
| A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C N-Acetyl-L-Leucine, Placebo - Niemann-Pick Disease, Type C | Phase 3 | Recruiting | IntraBio Inc | 53 | 2022-06-30 | 2026-04-03 |
| Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B APVO101 - Hemophilia B | Phase 3 | Completed | Medexus Pharma, Inc. | 21 | 2020-01-16 | 2024-05-16 |
| N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) IB1001 - Ataxia Telangiectasia | Phase 2 | Terminated | IntraBio Inc | 17 | 2020-01-08 | 2026-03-25 |
| N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) IB1001 - Niemann-Pick Disease, Type C | Phase 2 | Completed | IntraBio Inc | 33 | 2019-09-04 | 2023-11-28 |
| N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) IB1001 - GM2 Gangliosidosis, Tay-Sachs Disease | Phase 2 | Completed | IntraBio Inc | 30 | 2019-06-07 | 2024-03-12 |
| Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B IB1001 - Hemophilia B | Phase 3 | Withdrawn | Cangene Corporation | 2014-03-01 | 2016-08-23 | |
| Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B IB1001 - Hemophilia B | Phase 3 | Terminated | Medexus Pharma, Inc. | 9 | 2010-11-01 | 2021-03-15 |
| Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B IB1001, nonacog alfa - Hemophilia B | Phase 2 / Phase 3 | Completed | Medexus Pharma, Inc. | 77 | 2009-01-01 | 2021-04-06 |
Hemophilia B (4) · Ataxia Telangiectasia (2) · Niemann-Pick Disease, Type C (2) · Blood Coagulation Disorders (1) · Blood Coagulation Disorders, Inherited (1) · Coagulation Protein Disorders (1) · Gangliosidoses, GM2 (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Hematologic Diseases (1) · Hemophilia A (1) · Hemorrhagic Disorders (1) · Sandhoff Disease (1) · Tay-Sachs Disease (1)
IntraBio Inc (5) · Medexus Pharma, Inc. (3) · Cangene Corporation (1)
United Kingdom (8) · United States (8) · Germany (5) · Spain (4) · Slovakia (3) · India (2) · Switzerland (2) · Australia (1) · Brazil (1) · Czechia (1) · France (1) · Georgia (1) · Israel (1) · Italy (1) · Moldova (1)
Khaled Mohamed (1)
PMID 33482890, PMID 34349180, PMID 34387740, PMID 40739200, PMID 22764744, PMID 28833808, PMID 30115089, PMID 33738443, PMID 34620022, PMID 36456200, PMID 38294974, PMID 40513057, PMID 42309084, PMID 42467089