NCT00778882WithdrawnPhase 1
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
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Withdrawn
- Sponsor
- Helixmith Co., Ltd.
- Enrollment
- 2
- Locations
- 1
- Primary Endpoint
- The incidence of adverse events through 1 year
Study 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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Sex
- Male
- Accepts Healthy Volunteers
- No
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
Experimental
Intervention: VM106 (Drug)
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)
Investigators
Study Sites (1)
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