Lentiviral FVIII Gene Therapy

NCT03217032 | Recruiting Phase 1

• 1 other identifier: GIMI-IRB-17007
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.

Conditions

Hemophilia A

Keywords

Hemophilia A; factor VIII; Gene therapy; lentiviral vector

Outcome Measures

Primary Outcomes (1)

• Number of participants experiencing drug-related adverse events

  As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FVIII inhibitor

  a year

Secondary Outcomes (1)

• Changes from baseline in circulating FVIII activity (IU/dL or % normal)

  a year

Study Arms (1)

YUVA-GT-F801

EXPERIMENTAL

Gene transfer to treat Hemophilia A

Biological: YUVA-GT-F801

Interventions

YUVA-GT-F801 BIOLOGICAL

Lentiviral factor VIII gene modified autologous hematopoeitic stem cells and mesenchymal stem cells

YUVA-GT-F801

Eligibility Criteria

• Age: 2 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Able to provide informed consent and comply with requirements of the study.
• Males ≥2 years with confirmed diagnosis of hemophilia A (endogenous factor VIII ≤2 IU/dL or ≤2% of normal).
• A minimum average of 4 bleeding events per year requiring episodic treatment of factor VIII infusions or prophylactic factor VIII infusions.
• No measurable factor VIII inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor VIII protein.
• Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences.

You may not qualify if:

• Significant liver dysfunction as defined by abnormal alanine transaminase, bilirubin and alkaline phosphatase.
• History of inhibitor against factor VIII.
• Evidence of active hepatitis B or C and currently on antiviral therapy.
• Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (subjects who are HIV+ and stable with CD4 count \>200/mm3 and undetectable viral load are eligible to enroll).
• Any evidence of active infection or any immunosuppressive disorder.
• Participated in a gene transfer trial within the last 6 months or in a clinical trial with an investigational drug within the last 12 weeks.
• Unable or unwilling to comply with study assessments.

Sponsors & Collaborators

• Shenzhen Geno-Immune Medical Institute: lead

Study Sites (1)

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, 518000, China

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Lung-Ji Chang

  Shenzhen Geno-Immune Medical Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lung-Ji Chang, PhD

CONTACT

+86 0755-86573763; c@szgimi.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: President

Study Record Dates

• First Submitted: July 3, 2017
• First Posted: July 13, 2017
• Study Start: June 1, 2025
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): June 1, 2028
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)