MedTrace Logo

Gene Therapy for Fanconi Anemia

NCT01331018 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2024-05-17

No results posted yet for this study

Summary

This clinical trial will access the toxicity and efficacy of infusion of gene modified cells for patients with Fanconi anemia (FA). Infusion of autologous patient blood stem cells that have been corrected in the laboratory by introduction of the normal gene may improve blood counts in patients with FA.

Conditions

  • Fanconi Anemia

Interventions

PROCEDURE

Bone Marrow Aspiration

Undergo bone marrow harvest

BIOLOGICAL

Filgrastim

Given SC

BIOLOGICAL

Genetically Engineered Hematopoietic Stem Progenitor Cells

Undergo infusion of genetically modified hematopoietic progenitor cell therapy

OTHER

Laboratory Biomarker Analysis

Correlative studies

PROCEDURE

Leukapheresis

Undergo leukapheresis

DRUG

Methylprednisolone

Given IV

DRUG

Plerixafor

Given SC

DRUG

Prednisone

Given PO

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Rocket Pharma Limited

    collaborator UNKNOWN

  • Fred Hutchinson Cancer Center

    lead OTHER

Principal Investigators

  • Hans-Peter Kiem · Fred Hutch/University of Washington Cancer Consortium

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-02-22
Primary Completion
2024-02-15
Completion
2024-02-15

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01331018 on ClinicalTrials.gov