Gene Therapy for Chronic Granulomatous Disease in Korea

• Status: Active, not recruiting
• Trial end date: 2022-10-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Helixmith Co., Ltd.; ViroMed Co., Ltd. dba VM BioPharma

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