NCT03351868

Brief Summary

This is a Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Dec 2017

Longer than P75 for not_applicable

Geographic Reach
1 country

3 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 20, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 24, 2017

Completed
7 days until next milestone

Study Start

First participant enrolled

December 1, 2017

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2020

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2021

Completed
Last Updated

September 19, 2019

Status Verified

September 1, 2019

Enrollment Period

3.1 years

First QC Date

November 20, 2017

Last Update Submit

September 18, 2019

Conditions

Fanconi Anemia

Keywords

Fanconi anemiaLentiviral vectorFANCAGene

Outcome Measures

Primary Outcomes (1)

  • Safety in patients using CTCAE version 4.0 standard to evaluate the level of adverse events

    Physiological parameter (measuring cytokine response, fever, symptoms)

    6 months

Secondary Outcomes (2)

  • Treatment responses

    1 year

  • Quality of life

    1 year

Study Arms (1)

Gene-modified autologous stem cells

EXPERIMENTAL

Autologous hematopoeitic stem cells and mesenchymal stem cells transduced with lentiviral vector carrying the FANCA gene ex vivo

Genetic: Gene-modified autologous stem cells

Interventions

Gene-modified autologous stem cellsGENETIC

Infusion for 5x10\^6\~1x10\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances

Gene-modified autologous stem cells

Eligibility Criteria

Age2 Years - 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide.
  • No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.
  • Age: ≥ 4 years.
  • Karnofsky: ≥ 70%.
  • ANC ≥ 5×10\^8/L; PLT ≥ 2×10\^10/L.
  • Hemoglobin ≥ 8g/dL.
  • Proper renal and hepatic functions (ULN denotes "upper limit of normal range") with
  • serum creatinine ≤ 1.5×ULN;
  • serum bilirubin ≤ 3×ULN;
  • AST/ALT ≤ 5×ULN.
  • Pulmonary function is normal; DLCO \> 50%.
  • Written, informed consent obtained prior to any study-specific procedures.

You may not qualify if:

  • Diagnosis of active malignant disease or myelodysplastic syndrome.
  • Diagnosis of myeloid leukemia.
  • Pregnant or lactating females.
  • Existence of an available HLA-identical related donor.
  • Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive.
  • Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Capital Institute of Pediatrics affiliated Children's hospital

Beijing, Beijing Municipality, 100020, China

RECRUITING

Beijing Children's Hospital

Beijing, Beijing Municipality, China

RECRUITING

Shenzhen Geno-immune Medical Institute

Shenzhen, Guangdong, 518000, China

RECRUITING

MeSH Terms

Conditions

Fanconi Anemia

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Lung-Ji Chang, Ph.D

    Shenzhen Geno-Immune Medical Institute

    PRINCIPAL INVESTIGATOR

  • Xiao-Dong Shi, M.D./Ph. D

    Capital Institute of Pediatrics affiliated Children's hospital

    STUDY DIRECTOR

  • Jie Zheng, M.D./Ph. D

    Beijing Children's Hospital

    STUDY DIRECTOR

Central Study Contacts

Lung-Ji Chang, Ph.D

CONTACT

86-13671121909c@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
PRINCIPAL INVESTIGATOR
PI Title
President

Study Record Dates

First Submitted

November 20, 2017

First Posted

November 24, 2017

Study Start

December 1, 2017

Primary Completion

December 31, 2020

Study Completion

December 31, 2021

Last Updated

September 19, 2019

Record last verified: 2019-09

Data Sharing

IPD Sharing
Will not share

Locations

CN(3)