This trial is active, not recruiting.

Condition chronic granulomatous disease
Treatment vm106
Phase phase 1/phase 2
Sponsor ViroMed Co., Ltd. dba VM BioPharma
Start date January 2007
End date October 2008
Trial size 2 participants
Trial identifier NCT00778882, VM106-KR-01


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Autologous hematopoietic stem cells with MT-gp91 retroviral vector

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
time frame: 1 year
Safety and efficacy of fludarabine/busulfan conditioning
time frame: 1 year
Functional reconstitution of respiratory burst
time frame: 1 year
Presence of vector-positive cells
time frame: 1 year

Eligibility Criteria

Male participants of any age.

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

Additional Information

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
Principal investigator Joong Gon Kim, MD, PhD
Trial information was received from ClinicalTrials.gov and was last updated in February 2016.
Information provided to ClinicalTrials.gov by ViroMed Co., Ltd. dba VM BioPharma.