Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

Wiskott-Aldrich Syndrome Clinical Trial

— WASFUP  

Official title:

Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08).

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02333760
• Other study ID #: GNT-WAS-03
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: September 2014
• Est. completion date: October 2032

Study information

• Verified date: May 2021
• Source: Genethon
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 10
• Est. completion date: October 2032
• Est. primary completion date: October 2032
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08).
• Parents, guardians or patient signed informed consent, guardians or patient signed informed consent

Exclusion Criteria:

• Parents, guardians, patients unwilling to return for the follow up study period.

Related Conditions & MeSH terms

• Syndrome
• Wiskott-Aldrich Syndrome

Intervention

Genetic:
• Autologous CD34+ cells transduced with WASP lentiviral vector
Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene

Locations

Country	Name	City	State
France	Hopital Necker - Enfants Malades	Paris
United Kingdom	UCL Institute of Child Health	London

Sponsors (1)

Lead Sponsor	Collaborator
Genethon

Countries where clinical trial is conducted

France,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and type of SAEs	Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality	yearly from 3 years to 15 years
Primary	Lentiviral integration sites	Presence of lentiviral integration sites in different cells sub-populations	yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Primary	Vector copy numbers	Quantification of vector copy numbers on sorted cells population by q-PCR	yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Primary	Replication competent lentivirus (RCL)	Presence of RCL	yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Primary	Change in medical conditions	Weight and complete clinical exam	yearly from 3 years to 10 years
Primary	Key medical events related to WAS	Eczema status, infections, bleeding symptoms, autoimmune manifestation	yearly from 3 years to 10 years
Primary	Hematological reconstitution	CBC including platelets count and size	yearly from 3 years to 10 years
Primary	Reconstitution of cell mediated and humoral immunity	Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene	yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Secondary	Need for associated treatments	Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions	yearly from 3 years to 15 years
Secondary	Representation of TCR families	Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel	yearly from 3 years to 5 years
Secondary	Bone marrow content	Numbers and type of cells in bone marrow	yearly from 3 years to 5 years (optional)