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Gene Therapy for Chronic Granulomatous Disease in Korea

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
VM Biopharma
Information provided by (Responsible Party):
ViroMed Co., Ltd.
ClinicalTrials.gov Identifier:
NCT00778882
First received: October 21, 2008
Last updated: June 26, 2012
Last verified: June 2012
History of Changes
  Purpose

The purpose of this study is to evaluate the safety and efficacy of administration of autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector for patients with X-linked chronic granulomatous disease.


Condition Intervention Phase
Chronic Granulomatous Disease
 Drug: Autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Single Administration of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease Patients

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by ViroMed Co., Ltd.:

Primary Outcome Measures:
  • The incidence of adverse events through 1 year [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • RCR, insertional mutagenesis, immune response against normal gp91 protein [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
  • Safety and efficacy of fludarabine/busulfan conditioning [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
  • Functional reconstitution of respiratory burst [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Presence of vector-positive cells [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Enrollment: 2
Study Start Date: January 2007
Estimated Study Completion Date: October 2022
Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector
    Drug: Autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector
  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • gp91 defective male patients with chronic granulomatous disease: confirmed by DHR
  • Weigh greater than or equal to 15 kg
  • History of severe infections: more than 2 times
  • Performance status: ECOG 0-2

  • Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

    • Heart: a shortening fraction > 28%; QTc interval < 0.44
    • Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal; AST < 3 x upper limit of normal
    • Kidney: creatine < 2 x normal
    • Blood: WBC > 2,500/uL; platelet > 100,000/uL; hematocrit > 26%
  • Written informed consent obtained from patient (or guardian if patients age < 19)

Exclusion Criteria:

  • Presence of a HLA-matched sibling for stem cell donation
  • Evidence or history of malignant tumor
  • Presence of a severe infection
  • Presence of an active tuberculosis
  • Uncorrectable electrolyte, Ca, P
  • Unable to comply with the protocol or to cooperate fully with the Investigator or site personnel
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00778882

Locations
Korea, Republic of
Seoul National University Hospital
Seoul, Korea, Republic of, 110-744
Sponsors and Collaborators
ViroMed Co., Ltd.
VM Biopharma
Investigators
Principal Investigator: Joong Gon Kim, MD, PhD Seoul National University Hospital
  More Information

No publications provided

Responsible Party: ViroMed Co., Ltd.
ClinicalTrials.gov Identifier: NCT00778882     History of Changes
Other Study ID Numbers: VM106-KR-01
Study First Received: October 21, 2008
Last Updated: June 26, 2012
Health Authority: Korea: Food and Drug Administration

Keywords provided by ViroMed Co., Ltd.:
Chronic Granulomatous Disease
Retroviral Vector
Gene Therapy

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Granuloma
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
 Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on June 13, 2013