Gene Therapy for Chronic Granulomatous Disease in Korea

Helixmith

Status and phase: Withdrawn; Phase 2; Phase 1

Conditions: Chronic Granulomatous Disease

Treatments: Drug: VM106

Study type: Interventional

Funder types: Industry

Identifiers

NCT00778882

VM106-KR-01

Details and patient eligibility

About

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.

Sex: Male

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

VM106

Experimental group

Treatment:

Drug: VM106