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Register:	EUCTR
Last refreshed on:	2 October 2017
Main ID:	EUCTR2009-011152-22-FR
Date of registration:	09/12/2010
Prospective Registration:	Yes
Primary sponsor:	GENETHON
Public title:	Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS
Scientific title:	Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome - Gene therapy for WAS
Date of first enrolment:
Target sample size:	10
Recruitment status:	Not Recruiting
URL:	https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-011152-22
Study type:	Interventional clinical trial of medicinal product
Study design:	Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
Phase:	Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment
France

Contacts

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Key inclusion & exclusion criteria

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. Unless desease severity indicates that one cannot wait for 3 months (score 5; refractory thrombocytopenia with platelets <5000 with bleeding or severe autoimmunity),
a. Lack of HLA-genotypically identical bone marrow after 3 month search
b. Lack of a 10/10 or 9/10 antigen HLA-matched unrelated donor after 3 month search
c. Lack of a HLA-matched 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 and lifelong for off study review

5. Only for patients who have received previous allogenic haematopoietic stem cell transplant:
a. Failed allogenic haematopoietic stem cell transplant
b. Contraindication to repeat allogenic transplantation

Are the trial subjects under 18? yes
Number of subjects for this age range:
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 or 9/10 antigen HLA-matched unrelated donor or with HLA-matched cord blood

2. a. Contraindication to leukapheresis
i. Anaemia (Hb < 8g/dl)
ii.Severe vascularitis
iii.Refractory thrompopenia
b. Contraindication to bone marrow harvest
c. Contraindication to administration of conditioning medication

3. HIV seropositive patient

Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome. An open labelled, non-randomised, phase I/II, cohort study involving a single infusion of autologous CD34+ cells transduced with the lentiviral vector w1.6_hWASP_WPRE (VSVg) in up to 5 patients with WAS.

Intervention(s)

Product Name: Autologous CD34+ cells transduced with the Lentiviral vector containing the human Wiskott Aldrich Sy
Product Code: GTG003.08
Pharmaceutical Form: Solution for injection

Primary Outcome(s)

Primary end point(s): 1. Safety of conditioning regimen (hematopoietic recovery within 6 weeks a assessed by absolute neutrophil count (ANC) above 0.5 x 109 /l)

2. Safety of the transduction procedure (as assessed by availability of greater than 1 x 106CD34+ cells per kg; retrospective undetectable RCL; and cell viability equal to or greater than 70%, in accordance with the GMO release criteria).

3. Engraftment of genetically corrected haematopoietic progenitors and/or differentiated cells in peripheral blood and/or in bone marrow (as assessed by evidence of vector sequences or transgene expression in the cells)

4. Reconstitution of cell mediated and humoral immunity (as assessed by evidence of changes in T cell function and circulating immunoglobulin levels).

5. Correction of microthrombocytopenia (as assessed by increased blood platelet counts, expected to rise above 50,000/mm3 and platelets size restoration)

Main Objective: To safely administer a lentiviral gene therapy vector encoding the human WAS cDNA in patients with WAS
To provide sustained engraftment of WASP-expressing transduced cells, reconstitution of humoral and cell mediated immunity, and correction of microthrombocytopenia.

Secondary Objective: To improve the overall health of the patient, including reduction in frequency of infections, resolution of autoimmunity, and improvement in eczema, reduction in bruising and bleeding episodes.
To evaluate the longitudinal clinical effect in terms of improved immunity.

Secondary Outcome(s)

Secondary ID(s)

GTG003.08

Source(s) of Monetary Support

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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