Lentiviral Gene Therapy for CGD

NCT03645486 | Recruiting

• 1 other identifier: GIMI-IRB-18004
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.

Conditions

Chronic Granulomatous Disease

Keywords

Chronic granulomatous disease; Lentiviral vector

Outcome Measures

Primary Outcomes (2)

• Overall survival

  Patient will be monitored for overall health condition, including immune cell assessments, blood biochemistry and metabolitic activities, metabolic detoxification.

  15 year follow up

• Gene marking in bone marrow cells

  Gene-modified cells in the bone marrow will be measured by vector-specific quantitative PCR of colony-forming cells. Patient overall survival will be followed up for 15 years.

  15 year follow up

Secondary Outcomes (2)

• Change in infection frequency

  1 year after treatment by clinical history, complete physical examination, haematological and microbiological tests

• Recovery of immune function

  1 year follow up

Study Arms (1)

Lentiviral TYF-CGD-modified autologous stem cells

EXPERIMENTAL

Autologous hematopoietic stem cells transduced with lentiviral TYF vector carrying the functional gene

Genetic: Infusion of lentiviral TYF-CGD-modified autologous stem cells

Interventions

Infusion of lentiviral TYF-CGD-modified autologous stem cells GENETIC

Infusion of lentiviral TYF-modified autologous stem cells at 1\~10x10\^6 gene-modified cells per kg body weight

Lentiviral TYF-CGD-modified autologous stem cells

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• CGD patients \>= 0 years of age
• Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activities of the NADPH-oxidase
• Karnofsky-Index \> =70%
• At least one prior, ongoing or refractory severe infection and/or inflammatory complications requiring hospitalization despite drug intervention
• Written informed consent for adult patient, and assent for pediatric subjects seven years or older

You may not qualify if:

• Contraindication for leukapheresis (anaemia Hb \<8g/dl, cardiovascular instability, severe coagulopathy) or for administration of conditioning medication
• Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test

Sponsors & Collaborators

• Shenzhen Geno-Immune Medical Institute: lead

Study Sites (1)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, 518000, China

RECRUITING

MeSH Terms

Conditions

Granulomatous Disease, Chronic

Condition Hierarchy (Ancestors)

Phagocyte Bactericidal Dysfunction; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Lung-Ji Chang, Ph.D

CONTACT

+86 0755-86573763; c@szgimi.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2018
• First Posted: August 24, 2018
• Study Start: July 1, 2025
• Primary Completion (Estimated): June 30, 2028
• Study Completion (Estimated): December 31, 2029
• Last Updated: June 22, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)