NCT06364774 is a Phase 1, PHASE2 clinical trial of ALS20 in Beta-Thalassemia, sponsored by Children's Hospital of Philadelphia.

Who is sponsoring NCT06364774?

NCT06364774 is sponsored by Children's Hospital of Philadelphia.

What drugs are being tested in NCT06364774?

Interventions in NCT06364774: ALS20.

What condition does NCT06364774 study?

NCT06364774 studies Beta-Thalassemia.

Is NCT06364774 still recruiting?

NCT06364774 is currently recruiting now. Last updated 15 April 2026.

Last reviewed 2026-04-15 · How we verify

NCT06364774

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

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Recruiting now Phase 1, PHASE2 Last updated 15 April 2026

What this trial tests

Phase 1, PHASE2 trial testing ALS20 in Beta-Thalassemia in 12 participants. Currently enrolling.

Timeline

14 April 2025

Primary endpoint
31 December 2027

31 December 2027

Quick facts

Lead sponsor	Children's Hospital of Philadelphia
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	14 April 2025
Primary completion	31 December 2027
Estimated completion	31 December 2027
Sites	1 location across United States

Drugs / interventions tested

ALS20 — full drug profile →

Conditions studied

Beta-Thalassemia — all drugs for Beta-Thalassemia →

Sponsor

Children's Hospital of Philadelphia

Who can join

Adults 18 to 40, any sex, with Beta-Thalassemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 ("study drug"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.

Publications & conference data

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

Clinical hematopoietic stem cell-based gene therapy.
John T, Czechowicz A. · · 2025 · cited 6× · PMID 40285354 · DOI 10.1016/j.ymthe.2025.04.029
Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.
Ball J, Bradley A, Le A, Tisdale JF, et al · · 2025 · cited 5× · PMID 40083162 · DOI 10.1016/j.ymthe.2025.03.016
Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.
Winkler R, Herath I, Kaczmarek R, Xiao W, et al · · 2026 · PMID 41773269 · DOI 10.2147/jbm.s576017
Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.
Aprile A, Lidonnici MR, Ferrari G. · · 2025 · PMID 41253147 · DOI 10.1016/j.xcrm.2025.102419

Verify or expand the search:

Other recruiting trials for Beta-Thalassemia

Currently open trials in the same condition.

NCT06568926 — Adherence of Beta Thalssemia Patients to Oral Chelation Therapy · NA · recruiting
NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · recruiting
NCT06308159 — An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia · Phase 1, PHASE2 · recruiting
NCT06065189 — Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major · EARLY_PHASE1 · active not recruiting
NCT05477563 — Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia an · Phase 3 · recruiting

Other Children's Hospital of Philadelphia trials

Trials by the same sponsor.

NCT07023380 — EoE Food Test Pilot Study · NA · not yet recruiting
NCT06693752 — CEUS Evaluation of Hydrocephalus in Neonates and Infants · Phase 2 · recruiting
NCT07425691 — SPACE for Youth With Chronic Pain · recruiting
NCT07444528 — Upper Airway Ultrasound to Predict Difficult Laryngoscopy in Neonates and Infants · enrolling by invitation
NCT07321327 — Implementation Strategies for Caregiver and Teacher Use of Behavioral Interventions: Aim 2 · NA · enrolling by invitation

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 NCT06364774 (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 Children's Hospital of Philadelphia
Last refreshed: 15 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/NCT06364774.

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