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Gene Therapy for Chronic Granulomatous Disease in Korea

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ClinicalTrials.gov Identifier: NCT00778882
Recruitment Status : Active, not recruiting
First Posted : October 24, 2008
Last Update Posted : February 11, 2016
Sponsor:
Information provided by (Responsible Party):
ViroMed Co., Ltd.

Brief Summary:
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 or disease Intervention/treatment Phase
Chronic Granulomatous Disease Drug: VM106 Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : January 2007
Actual Primary Completion Date : October 2008
Estimated Study Completion Date : October 2022


Arm Intervention/treatment
Experimental: VM106 Drug: VM106
Autologous hematopoietic stem cells with MT-gp91 retroviral vector




Primary Outcome Measures :
  1. The incidence of adverse events through 1 year [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. RCR, insertional mutagenesis, immune response against normal gp91 protein [ Time Frame: 1 year ]
  2. Safety and efficacy of fludarabine/busulfan conditioning [ Time Frame: 1 year ]
  3. Functional reconstitution of respiratory burst [ Time Frame: 1 year ]
  4. Presence of vector-positive cells [ Time Frame: 1 year ]


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00778882


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

Responsible Party: ViroMed Co., Ltd.
ClinicalTrials.gov Identifier: NCT00778882     History of Changes
Other Study ID Numbers: VM106-KR-01
First Posted: October 24, 2008    Key Record Dates
Last Update Posted: February 11, 2016
Last Verified: February 2016

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

Additional relevant MeSH terms:
Granulomatous Disease, Chronic
Granuloma
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