Lentiviral Gene Therapy for CGD

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-07-01

Study Completion Date

2021-12-31

Brief Summary

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This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.

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Detailed Description

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Chronic granulomatous disease (CGD) is a rare disorder caused by inherited defects in the NADPH oxidase multienzyme complex. It is associated with severe and life-threatening bacterial and fungal infections. Approximately two-thirds of all CGD cases result from mutations within the X-linked gp91phox gene (CYBB), followed by the autosomal recessive forms of CGD, with defects in the gene coding for p47phox (NCF1) accounting for 10-30% of all CGD cases.

The primary objectives are to evaluate the safety of the advanced self-inactivating lentiviral vector TYF-CYBB and TYF-NCF1, the ex-vivo gene transfer clinical protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment, assessment of vector integration sites, and finally the long-term correction of immune dysfunctions.

Conditions

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Chronic Granulomatous Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lentiviral TYF-CGD-modified autologous stem cells

Autologous hematopoietic stem cells transduced with lentiviral TYF vector carrying the functional gene

Group Type EXPERIMENTAL

Infusion of lentiviral TYF-CGD-modified autologous stem cells

Intervention Type GENETIC

Infusion of lentiviral TYF-modified autologous stem cells at 1\~10x10\^6 gene-modified cells per kg body weight

Interventions

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Infusion of lentiviral TYF-CGD-modified autologous stem cells

Infusion of lentiviral TYF-modified autologous stem cells at 1\~10x10\^6 gene-modified cells per kg body weight

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

1. CGD patients \>= 0 years of age
2. Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activities of the NADPH-oxidase
3. Karnofsky-Index \> =70%
4. At least one prior, ongoing or refractory severe infection and/or inflammatory complications requiring hospitalization despite drug intervention
5. Written informed consent for adult patient, and assent for pediatric subjects seven years or older

Exclusion Criteria

1. Contraindication for leukapheresis (anaemia Hb \<8g/dl, cardiovascular instability, severe coagulopathy) or for administration of conditioning medication
2. Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test

Eligible Sex

ALL

Accepts Healthy Volunteers

No