Gene Therapy for Chronic Granulomatous Disease in Korea

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
ViroMed Co., Ltd. dba VM BioPharma Identifier:
First received: October 21, 2008
Last updated: February 10, 2016
Last verified: February 2016
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: VM106
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 Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease Patients

Resource links provided by NLM:

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

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)
Arms Assigned Interventions
Experimental: VM106 Drug: VM106
Autologous hematopoietic stem cells with MT-gp91 retroviral vector


Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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
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Please refer to this study by its identifier: NCT00778882

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

Responsible Party: ViroMed Co., Ltd. dba VM BioPharma Identifier: NCT00778882     History of Changes
Other Study ID Numbers: VM106-KR-01 
Study First Received: October 21, 2008
Last Updated: February 10, 2016
Health Authority: Korea: Food and Drug Administration

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

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Immune System Diseases
Immunologic Deficiency Syndromes
Leukocyte Disorders
Lymphatic Diseases
Lymphoproliferative Disorders
Pathologic Processes
Phagocyte Bactericidal Dysfunction processed this record on May 26, 2016