NCT05454774 is a EARLY_PHASE1 clinical trial of Single dose intravenous injection of BBM 002 in Hemophilia A, sponsored by Institute of Hematology & Blood Diseases Hospital, China.

Who is sponsoring NCT05454774?

NCT05454774 is sponsored by Institute of Hematology & Blood Diseases Hospital, China.

What drugs are being tested in NCT05454774?

Interventions in NCT05454774: Single dose intravenous injection of BBM 002.

What condition does NCT05454774 study?

NCT05454774 studies Hemophilia A.

Is NCT05454774 still recruiting?

NCT05454774 is currently recruiting now. Last updated 21 February 2025.

Last reviewed 2025-02-21 · How we verify

NCT05454774

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

A Study of FVIII Gene Therapy for Hemophilia A

Recruiting now EARLY_PHASE1 Last updated 21 February 2025

What this trial tests

EARLY_PHASE1 trial testing Single dose intravenous injection of BBM 002 in Hemophilia A in 8 participants. Currently enrolling.

Timeline

19 July 2022

Primary endpoint
15 December 2024

15 September 2027

Quick facts

Lead sponsor	Institute of Hematology & Blood Diseases Hospital, China
Phase	EARLY_PHASE1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	8
Start date	19 July 2022
Primary completion	15 December 2024
Estimated completion	15 September 2027
Sites	1 location across China

Drugs / interventions tested

Single dose intravenous injection of BBM 002

Conditions studied

Hemophilia A — all drugs for Hemophilia A →

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Who can join

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

Sponsor's own description

This is a single-arm, open-label, clinical study to evaluate the safety, tolerability of BBM 002 injection in Hemophilia A subjects with residual factor VIII (FVIII) levels ≤2 International unit per deciliter (IU/dl) . BBM 002 injection is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene and raises circulating levels of endogenous FVIII.

Publications & conference data

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

Liver directed adeno-associated viral vectors to treat metabolic disease.
Chuecos MA, Lagor WR. · · 2024 · cited 23× · PMID 37254440 · DOI 10.1002/jimd.12637
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
Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives.
Chernyi N, Gavrilova D, Saruhanyan M, Oloruntimehin ES, et al · · 2024 · cited 13× · PMID 39062568 · DOI 10.3390/biom14070854
Therapeutic advances in hemophilia: from molecular innovation to patient-centered global care.
Dushimova Z, Pashimov M, Kaibullayeva J, Danyarova L, et al · · 2025 · PMID 41080961 · DOI 10.3389/fmed.2025.1618464

Verify or expand the search:

Other recruiting trials for Hemophilia A

Currently open trials in the same condition.

NCT07416526 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A · Phase 3 · recruiting
NCT07416604 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A · Phase 3 · recruiting
NCT07523399 — Joint Health, Balance and Quality of Life in Adults With Hemophilia A · recruiting
NCT06833983 — To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A · Phase 3 · recruiting
NCT06579144 — Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A · Phase 1 · recruiting

Other Institute of Hematology & Blood Diseases Hospital, China trials

Trials by the same sponsor.

NCT06991920 — Immune-targeted Combination With Chemotherapy for Acute Leukemia of Ambiguous Lineage · NA · not yet recruiting
NCT07407010 — BCMA/CD3 Bispecific Antibody as Bridging Therapy Before CAR-T Cell Infusion in RRMM · Phase 1 · not yet recruiting
NCT07407140 — VAG Versus Standard Chemotherapy With FLT3 Inhibitor in Adult Patients With FLT3-Mutated AML · Phase 3 · not yet recruiting
NCT07454226 — ABL/JAK Inhibitors With Chemotherapy and Venetoclax for Ph-like ALL · NA · not yet recruiting
NCT07490288 — Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML · NA · not yet 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 NCT05454774 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 9 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/NCT05454774.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing