Who is sponsoring NCT05203679?

NCT05203679 is sponsored by Shanghai Xinzhi BioMed Co., Ltd..

What drugs are being tested in NCT05203679?

Interventions in NCT05203679: Single dose intravenous injection of BBM-H901.

What condition does NCT05203679 study?

NCT05203679 studies Hemophilia B.

Is NCT05203679 still recruiting?

NCT05203679 is currently active, enrolled. Last updated 1 April 2026.

Last reviewed 2026-04-01 · How we verify

NCT05203679

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

Active, enrolled Phase 2, PHASE3 Last updated 1 April 2026

What this trial tests

Phase 2, PHASE3 trial testing Single dose intravenous injection of BBM-H901 in Hemophilia B in 32 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

30 December 2021

Primary endpoint
16 April 2024

30 June 2028

Quick facts

Lead sponsor	Shanghai Xinzhi BioMed Co., Ltd.
Phase	Phase 2, PHASE3
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	32
Start date	30 December 2021
Primary completion	16 April 2024
Estimated completion	30 June 2028
Sites	9 locations across China

Drugs / interventions tested

Single dose intravenous injection of BBM-H901

Conditions studied

Hemophilia B — all drugs for Hemophilia B →

Sponsor

Shanghai Xinzhi BioMed Co., Ltd. — full company profile →

Who can join

18 and older, male only, with Hemophilia B. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per deciliter (IU/dl) residual factor IX (FIX) levels. BBM-H901 is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene and raises circulating levels of endogenous FIX.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Current clinical applications of AAV-mediated gene therapy.
Byrne BJ, Flanigan KM, Matesanz SE, Finkel RS, et al · · 2025 · cited 43× · PMID 40329530 · DOI 10.1016/j.ymthe.2025.04.045
Viral and non-viral vectors in gene therapy: current state and clinical perspectives.
Geng G, Xu Y, Hu Z, Wang H, et al · · 2025 · cited 38× · PMID 40602323 · DOI 10.1016/j.ebiom.2025.105834
Liver directed adeno-associated viral vectors to treat metabolic disease.
Chuecos MA, Lagor WR. · · 2024 · cited 23× · PMID 37254440 · DOI 10.1002/jimd.12637
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
Hemophilic Arthropathy: Barriers to Early Diagnosis and Management.
Cuesta-Barriuso R, Donoso-Úbeda E, Meroño-Gallut J, Ucero-Lozano R, et al · · 2022 · cited 17× · PMID 36277171 · DOI 10.2147/jbm.s343924
Hemostats in the clinic.
Joshi M, Zhao Z, Mitragotri S. · · 2024 · cited 4× · PMID 39545083 · DOI 10.1002/btm2.10673
Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials.
Xue F, Ju M, Zhu T, Zhou Z, et al · · 2026 · cited 1× · PMID 41266685 · DOI 10.1038/s41591-025-04012-y
Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression.
Fong S, Swystun LL, Batty P, Lillicrap D. · · 2026 · PMID 41624053 · DOI 10.1177/20406207251406537

Verify or expand the search:

Other trials of Single dose intravenous injection of BBM-H901

Trials testing the same drug.

NCT04135300 — Gene Therapy for Chinese Hemophilia B · NA · completed

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 Shanghai Xinzhi BioMed Co., Ltd. trials

Trials by the same sponsor.

NCT07195825 — A Clinical Study to Evaluate the Safety, and Tolerability of BBM-P002 in the Treatment of Parkinson's Disease · Phase 1 · recruiting
NCT06111638 — Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs · Phase 2, PHASE3 · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT05203679 (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 Shanghai Xinzhi BioMed Co., Ltd.
Last refreshed: 1 April 2026

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/NCT05203679.

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