Gene Therapy for Chronic Granulomatous Disease in Korea

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
ViroMed Co., Ltd. dba VM BioPharma
ClinicalTrials.gov Identifier:
NCT00778882
First received: October 21, 2008
Last updated: January 15, 2014
Last verified: January 2014
  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:


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)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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. dba VM BioPharma
Investigators
Principal Investigator: Joong Gon Kim, MD, PhD Seoul National University Hospital
  More Information

No publications provided

Responsible Party: ViroMed Co., Ltd. dba VM BioPharma
ClinicalTrials.gov Identifier: NCT00778882     History of Changes
Other Study ID Numbers: VM106-KR-01
Study First Received: October 21, 2008
Last Updated: January 15, 2014
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:
Granuloma
Granulomatous Disease, Chronic
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases

ClinicalTrials.gov processed this record on October 01, 2014