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NCT04135300
Gene Therapy for Chinese Hemophilia B
NA trial testing Single dose intravenous injection of BBM-H901 in Hemophilia B in 10 participants. Completed in 7 January 2022.
7 January 2022
Quick facts
| Lead sponsor | Institute of Hematology & Blood Diseases Hospital, China |
|---|---|
| Phase | NA |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 10 |
| Start date | 16 October 2019 |
| Primary completion | 7 January 2022 |
| Estimated completion | 7 January 2022 |
| Sites | 1 location across China |
Drugs / interventions tested
- Single dose intravenous injection of BBM-H901
Conditions studied
- Hemophilia B — all drugs for Hemophilia B →
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Who can join
18 and older, male only, with Hemophilia B. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
GT2019001 is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Gene therapy for hemophilia.
Nathwani AC. · · 2022 · cited 74× · PMID 36485127 · DOI 10.1182/hematology.2022000388 -
Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial.
Xue F, Li H, Wu X, Liu W, et al · · 2022 · cited 67× · PMID 35598604 · DOI 10.1016/s2352-3026(22)00113-2 -
Recent Advances in the Treatment of Hemophilia: A Review.
Marchesini E, Morfini M, Valentino L. · · 2021 · cited 53× · PMID 34163136 · DOI 10.2147/btt.s252580 -
A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders.
Vrellaku B, Sethw Hassan I, Howitt R, Webster CP, et al · · 2024 · cited 39× · PMID 39044426 · DOI 10.1016/j.ymthe.2024.07.016 -
Liver directed adeno-associated viral vectors to treat metabolic disease.
Chuecos MA, Lagor WR. · · 2024 · cited 23× · PMID 37254440 · DOI 10.1002/jimd.12637 -
Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective.
Tomeo F, Mariz S, Brunetta AL, Stoyanova-Beninska V, et al · · 2021 · cited 23× · PMID 33772837 · DOI 10.1111/bcp.14838 -
Gene Therapy Approaches for the Treatment of Hemophilia B.
Soroka AB, Feoktistova SG, Mityaeva ON, Volchkov PY. · · 2023 · cited 21× · PMID 37445943 · DOI 10.3390/ijms241310766 -
An overview of development in gene therapeutics in China.
Wang D, Wang K, Cai Y. · · 2020 · cited 20× · PMID 32528163 · DOI 10.1038/s41434-020-0163-7
Verify or expand the search:
- PubMed search for NCT04135300
- Europe PMC full search
- ASCO Meeting Library
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Related trials
Other trials of Single dose intravenous injection of BBM-H901
Trials testing the same drug.
- NCT05203679 — Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug · Phase 2, PHASE3 · active not recruiting
Other recruiting trials for Hemophilia B
Currently open trials in the same condition.
- NCT07080905 — Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or · Phase 3 · recruiting
- NCT06727669 — Longitudinal Cohort of Thrombosis and Hemostasis Diseases · recruiting
- NCT06565481 — Measurement Properties in People with Hemophilia · NA · recruiting
- NCT06379789 — A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) · Phase 1, PHASE2 · recruiting
- NCT06399289 — Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Pre · Phase 3 · active not recruiting
Other Institute of Hematology & Blood Diseases Hospital, China trials
Trials by the same sponsor.
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- NCT07454226 — ABL/JAK Inhibitors With Chemotherapy and Venetoclax for Ph-like ALL · NA · not yet recruiting
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04135300 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Institute of Hematology & Blood Diseases Hospital, China
- Last refreshed: 21 February 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04135300.
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