NCT04390971 is a NA clinical trial of ET-01 in Transfusion Dependent Beta-Thalassaemia, sponsored by Institute of Hematology & Blood Diseases Hospital, China.

Who is sponsoring NCT04390971?

NCT04390971 is sponsored by Institute of Hematology & Blood Diseases Hospital, China.

What drugs are being tested in NCT04390971?

Interventions in NCT04390971: ET-01.

What condition does NCT04390971 study?

NCT04390971 studies Transfusion Dependent Beta-Thalassaemia.

Is NCT04390971 still recruiting?

NCT04390971 is currently completed. Last updated 11 February 2026.

Last reviewed 2026-02-11 · How we verify

NCT04390971

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

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

Completed NA Last updated 11 February 2026

What this trial tests

NA trial testing ET-01 in Transfusion Dependent Beta-Thalassaemia in 3 participants. Completed in 5 March 2023.

Timeline

10 February 2023

Primary endpoint
5 March 2023

5 March 2023

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	3
Start date	10 February 2023
Primary completion	5 March 2023
Estimated completion	5 March 2023
Sites	3 locations across China

Drugs / interventions tested

ET-01 — full drug profile →

Conditions studied

Transfusion Dependent Beta-Thalassaemia — all drugs for Transfusion Dependent Beta-Thalassaemia →

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Who can join

Adults 6 to 35, any sex, with Transfusion Dependent Beta-Thalassaemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

Publications & conference data

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

CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments.
Laurent M, Geoffroy M, Pavani G, Guiraud S. · · 2024 · cited 45× · PMID 38786024 · DOI 10.3390/cells13100800
Gene editing and its applications in biomedicine.
Li G, Li X, Zhuang S, Wang L, et al · · 2022 · cited 44× · PMID 35235150 · DOI 10.1007/s11427-021-2057-0
Current trends of clinical trials involving CRISPR/Cas systems.
Zhang S, Wang Y, Mao D, Wang Y, et al · · 2023 · cited 35× · PMID 38020120 · DOI 10.3389/fmed.2023.1292452
CRISPR-Cas System: The Current and Emerging Translational Landscape.
Bhokisham N, Laudermilch E, Traeger LL, Bonilla TD, et al · · 2023 · cited 25× · PMID 37190012 · DOI 10.3390/cells12081103
Gene Therapy and Gene Editing for β-Thalassemia.
Christakopoulos GE, Telange R, Yen J, Weiss MJ. · · 2023 · cited 24× · PMID 36907613 · DOI 10.1016/j.hoc.2022.12.012
Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects.
Cetin B, Erendor F, Eksi YE, Sanlioglu AD, et al · · 2025 · cited 15× · PMID 40160040 · DOI 10.1017/erm.2025.10
Genetic Manipulation Strategies for β-Thalassemia: A Review.
Zakaria NA, Bahar R, Abdullah WZ, Mohamed Yusoff AA, et al · · 2022 · cited 7× · PMID 35783328 · DOI 10.3389/fped.2022.901605
Stem cell therapies in the clinic.
Acharya S, Shaha S, Bibbey MG, Mukherji M, et al · · 2025 · cited 3× · PMID 40385529 · DOI 10.1002/btm2.70000

Verify or expand the search:

Other trials of ET-01

Trials testing the same drug.

NCT05752123 — A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaem · NA · terminated
NCT04925206 — A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia · Phase 1 · terminated

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 NCT04390971 (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: 11 February 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/NCT04390971.

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