Gene therapy for WAS

Phase 1

• Conditions: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages.; MedDRA version: 19.1Level: PTClassification code 10047992Term: Wiskott-Aldrich syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2007-004308-11-GB
• Lead Sponsor: Genethon

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-07-06
• Last Update Posted: 13 February 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: Not specified

Inclusion Criteria

1.
a. Males of all ages
b. Severe WAS (clinical score 3 – 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
c. Molecular confirmation by WAS gene DNA sequencing

2. Lack of HLA-genotypically identical bone marrow OR of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search

3.Parental, guardian, patient signed informed consent/assessment

4.Willing to return for follow-up during the 2 year study

5.Only for patients who have received previous allogenic haematopoietic stem cell transplant:
a.Failed allogenic haematopoietic stem cell transplant
b.Contraindication to repeat allogeneic transplantation for example severe graft versus host disease

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
;
1.
a. Males of all ages
b. Severe WAS (clinical score 3 – 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
c. Molecular confirmation by WAS gene DNA sequencing

2. Lack of HLA-genotypically identical bone marrow OR of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search

3.Parental, guardian, patient signed informed consent/assessment

4.Willing to return for follow-up during the 2 year study

5.Only for patients who have received previous allogenic haematopoietic stem cell transplant:
a.Failed allogenic haematopoietic stem cell transplant
b.Contraindication to repeat allogeneic transplantation for example severe graft versus host disease

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
;
1.
a. Males of all ages
b. Severe WAS (clinical score 3 – 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
c. Molecular confirmation by WAS gene DNA sequencing

2. Lack of HLA-genotypically identical bone marrow OR of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search

3.Parental, guardian, patient signed informed consent/assessment

4.Willing to return for follow-up during the 2 year study

5.Only for patients who have received previous allogenic haematopoietic stem cell transplant:
a.Failed allogenic haematopoietic stem cell transplant
b.Contraindication to repeat allogeneic transplantation for example severe graft versus host disease

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.
a. Patient with HLA-genotypically identical bone marrow
b. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood

2.
a. Contraindication to leukapheresis
i. anaemia (Hb < 8g/dl)
ii. cardiovascular instability
iii. severe coagulopathy
b. Contraindication to bone marrow harvest
c. Contraindication to administration of conditioning medication

3.HIV positive patient
;
1.
a. Patient with HLA-genotypically identical bone marrow
b. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood

2.
a. Contraindication to leukapheresis
i. anaemia (Hb < 8g/dl)
ii. cardiovascular instability
iii. severe coagulopathy
b. Contraindication to bone marrow harvest
c. Contraindication to administration of conditioning medication

3.HIV positive patient
;
1.
a. Patient with HLA-genotypically identical bone marrow
b. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood

2.
a. Contraindication to leukapheresis
i. anaemia (Hb < 8g/dl)
ii. cardiovascular instability
iii. severe coagulopathy
b. Contraindication to bone marrow harvest
c. Contraindication to administration of conditioning medication

3.HIV positive patient

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified