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Gene Therapy for Chronic Granulomatous Disease in Korea

Primary Purpose

Chronic Granulomatous Disease

Status
Unknown status
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
VM106
Sponsored by
Helixmith Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Granulomatous Disease focused on measuring Chronic Granulomatous Disease, Retroviral Vector, Gene Therapy

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Seoul National University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VM106

Arm Description

Outcomes

Primary Outcome Measures

The incidence of adverse events through 1 year

Secondary Outcome Measures

RCR, insertional mutagenesis, immune response against normal gp91 protein
Safety and efficacy of fludarabine/busulfan conditioning
Functional reconstitution of respiratory burst
Presence of vector-positive cells

Full Information

First Posted
October 21, 2008
Last Updated
October 16, 2019
Sponsor
Helixmith Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT00778882
Brief Title
Gene Therapy for Chronic Granulomatous Disease in Korea
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 Type
Interventional

2. Study Status

Record Verification Date
October 2019
Overall Recruitment Status
Unknown status
Study Start Date
January 2007 (undefined)
Primary Completion Date
October 2008 (Actual)
Study Completion Date
October 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Helixmith Co., Ltd.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Granulomatous Disease
Keywords
Chronic Granulomatous Disease, Retroviral Vector, Gene Therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VM106
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
VM106
Intervention Description
Autologous hematopoietic stem cells with MT-gp91 retroviral vector
Primary Outcome Measure Information:
Title
The incidence of adverse events through 1 year
Time Frame
1 year
Secondary Outcome Measure Information:
Title
RCR, insertional mutagenesis, immune response against normal gp91 protein
Time Frame
1 year
Title
Safety and efficacy of fludarabine/busulfan conditioning
Time Frame
1 year
Title
Functional reconstitution of respiratory burst
Time Frame
1 year
Title
Presence of vector-positive cells
Time Frame
1 year

10. Eligibility

Sex
Male
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Joong Gon Kim, MD, PhD
Organizational Affiliation
Seoul National University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
110-744
Country
Korea, Republic of

12. IPD Sharing Statement

Learn more about this trial

Gene Therapy for Chronic Granulomatous Disease in Korea

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