Trial | NCT00778882  Report issue

Title

Gene Therapy for Chronic Granulomatous Disease in Korea
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

  • Phase

    Phase 1/Phase 2

  • Study Type

    Interventional

  • Status

    Unknown status

  • Intervention/Treatment

    vm106 ...

  • Study Participants

    2
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 Started
Jan 31
2007
Primary Completion
Oct 31
2008
Study Completion
Oct 31
2022
Anticipated
Last Update
Oct 18
2019

Drug VM106

Autologous hematopoietic stem cells with MT-gp91 retroviral vector

VM106 Experimental

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
No Results Posted