Gene Therapy for Chronic Granulomatous Disease in Korea - Full Text View

Sponsored by:	ViroMed Co., Ltd.
Information provided by:	ViroMed Co., Ltd.
ClinicalTrials.gov Identifier:	NCT00778882

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 I Phase II

Genetics Home Reference related topics: L1 syndrome

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Single Group Assignment, Safety/Efficacy Study
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

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:	January 2009
Estimated 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

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.

Investigators

Principal Investigator:

Joong Gon Kim, MD, PhD

Seoul National University Hospital

More Information

No publications provided

Responsible Party:	ViroMed Co., Ltd ( Chang-Wan Joo/Project leader, R&D Team )
Study ID Numbers:	VM106-KR-01
Study First Received:	October 21, 2008
Last Updated:	October 23, 2008
ClinicalTrials.gov Identifier:	NCT00778882 History of Changes
Health Authority:	Korea: Food and Drug Administration

Keywords provided by ViroMed Co., Ltd.:

Chronic Granulomatous Disease
Retroviral Vector
Gene Therapy

Study placed in the following topic categories:

Lymphatic Diseases
Genetic Diseases, Inborn
Hematologic Diseases
Granulomatous Disease, Chronic
Chronic Granulomatous Disease

Genetic Diseases, X-Linked
Leukocyte Disorders
Lymphoproliferative Disorders
Granuloma
Immunologic Deficiency Syndromes

Additional relevant MeSH terms:

Phagocyte Bactericidal Dysfunction
Lymphatic Diseases
Pathologic Processes
Immune System Diseases
Genetic Diseases, Inborn
Hematologic Diseases

Granulomatous Disease, Chronic
Genetic Diseases, X-Linked
Leukocyte Disorders
Lymphoproliferative Disorders
Granuloma
Immunologic Deficiency Syndromes

ClinicalTrials.gov processed this record on May 07, 2009