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

Helixmith Co., Ltd.1 site in 1 country2 target enrollmentJanuary 1, 2007

ConditionsChronic Granulomatous Disease

InterventionsVM106

DrugsVM106

Overview

Phase: Phase 1
Intervention: VM106
Conditions: Chronic Granulomatous Disease
Sponsor: Helixmith Co., Ltd.
Enrollment: 2
Locations: 1
Primary Endpoint: The incidence of adverse events through 1 year
Status: Withdrawn
Last Updated: 7 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

January 1, 2007

End Date

October 1, 2010

Last Updated

7 months ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Helixmith Co., Ltd.

Responsible Party

Sponsor

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

Arms & Interventions

VM106

Intervention: VM106

Outcomes

Primary Outcomes

The incidence of adverse events through 1 year

Time Frame: 1 year

Secondary Outcomes

RCR, insertional mutagenesis, immune response against normal gp91 protein(1 year)
Safety and efficacy of fludarabine/busulfan conditioning(1 year)
Functional reconstitution of respiratory burst(1 year)
Presence of vector-positive cells(1 year)