NCT04286815 is a NA clinical trial of Lentiviral Vector Gene Therapy in Gene Therapy, sponsored by Children's Hospital of Chongqing Medical University.

Who is sponsoring NCT04286815?

NCT04286815 is sponsored by Children's Hospital of Chongqing Medical University.

What drugs are being tested in NCT04286815?

Interventions in NCT04286815: Lentiviral Vector Gene Therapy.

What condition does NCT04286815 study?

NCT04286815 studies Gene Therapy.

Is NCT04286815 still recruiting?

NCT04286815 is currently status unknown. Last updated 27 March 2020.

Last reviewed 2020-03-27 · How we verify

NCT04286815

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

Gene Therapy for X Linked Severe Combined Immunodeficiency

Status unknown NA Last updated 27 March 2020

What this trial tests

NA trial testing Lentiviral Vector Gene Therapy in Gene Therapy in 10 participants. Status unknown.

Timeline

1 May 2020

Primary endpoint
1 May 2023

1 May 2025

Quick facts

Lead sponsor	Children's Hospital of Chongqing Medical University
Phase	NA
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	1 May 2020
Primary completion	1 May 2023
Estimated completion	1 May 2025
Sites	1 location across China

Drugs / interventions tested

Lentiviral Vector Gene Therapy

Conditions studied

Gene Therapy — all drugs for Gene Therapy →

Sponsor

Children's Hospital of Chongqing Medical University

Who can join

Under 18, male only, with Gene Therapy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.

Publications & conference data

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

Large-Scale Production of Lentiviral Vectors: Current Perspectives and Challenges.
Martínez-Molina E, Chocarro-Wrona C, Martínez-Moreno D, Marchal JA, et al · · 2020 · cited 55× · PMID 33153183 · DOI 10.3390/pharmaceutics12111051
Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.
Blanco E, Izotova N, Booth C, Thrasher AJ. · · 2020 · cited 29× · PMID 33329605 · DOI 10.3389/fimmu.2020.608653
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
Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.
Castiello MC, Ferrari S, Villa A. · · 2023 · cited 11× · PMID 36863140 · DOI 10.1016/j.smim.2023.101731
Gene Therapy for Primary Immunodeficiency.
Houghton BC, Booth C. · · 2021 · cited 10× · PMID 33403354 · DOI 10.1097/hs9.0000000000000509
Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side.
Garcia-Perez L, Ordas A, Canté-Barrett K, Meij P, et al · · 2020 · cited 6× · PMID 32545727 · DOI 10.3390/pharmaceutics12060549
Progress in the field of hematopoietic stem cell-based therapies for inborn errors of immunity.
Arnold DE, Pai SY. · · 2023 · cited 3× · PMID 37732933 · DOI 10.1097/mop.0000000000001292
Gene therapy for inborn errors of immunity: past progress, current status and future directions.
Torrance R, Orf K, Fox TA. · · 2025 · PMID 41693912 · DOI 10.20517/rdodj.2025.42

Verify or expand the search:

Other recruiting trials for Gene Therapy

Currently open trials in the same condition.

NCT07024524 — Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss · NA · recruiting
NCT05991336 — Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy · recruiting
NCT04728841 — Gene Therapy for Chinese Hemophilia A · NA · recruiting
NCT03311503 — Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning · Phase 1, PHASE2 · recruiting

Other Children's Hospital of Chongqing Medical University trials

Trials by the same sponsor.

NCT06201013 — Vitamin D Treat-to-Target Strategy for Children With Overactive Bladder-Wet · NA · recruiting
NCT07472829 — Research on Speech Development Trajectories and Predictive Models in Children With Autism Spectrum Disorder · not yet recruiting
NCT05911893 — the Effect of High Caloric Oral Nutritional Supplements on Growth and Development of Malnourished Children · NA · recruiting
NCT06404294 — The External Diaphragm Pacemaker Assisted Extubation in Premature Infants With Invasive Mechanical Ventilation · NA · recruiting
NCT05768399 — The Role of Biomarkers in the Occurrence and Development of Asthma · 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 NCT04286815 (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 Chongqing Medical University
Last refreshed: 27 March 2020

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/NCT04286815.

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