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

Phase 1

Active, not recruiting

• Conditions: Wiskott-Aldrich Syndrome

• Registration Number: NCT02333760
• Lead Sponsor: Genethon

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-09
• Study First Submitted: 2014-10-28
• Study First Submitted QC: 2015-01-05
• Study First Posted: 2015-01-07
• Status Verified: 2021-05
• Last Update Submitted: 2021-05-31
• Last Update Posted: 2021-06-03
• Primary Completion: 2032-10
• Study Completion: 2032-10

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Hematological reconstitution	yearly from 3 years to 10 years	CBC including platelets count and size
Lentiviral integration sites	yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)	Presence of lentiviral integration sites in different cells sub-populations
Vector copy numbers	yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)	Quantification of vector copy numbers on sorted cells population by q-PCR
Incidence and type of SAEs	yearly from 3 years to 15 years	Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
Replication competent lentivirus (RCL)	yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)	Presence of RCL
Change in medical conditions	yearly from 3 years to 10 years	Weight and complete clinical exam
Key medical events related to WAS	yearly from 3 years to 10 years	Eczema status, infections, bleeding symptoms, autoimmune manifestation
Reconstitution of cell mediated and humoral immunity	yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )	Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene

Secondary Outcome Measures

Name	Time	Method
Bone marrow content	yearly from 3 years to 5 years (optional)	Numbers and type of cells in bone marrow
Need for associated treatments	yearly from 3 years to 15 years	Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions
Representation of TCR families	yearly from 3 years to 5 years	Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel

Trial Locations

Locations (2)

UCL Institute of Child Health; 🇬🇧 London, United Kingdom

Hopital Necker - Enfants Malades; 🇫🇷 Paris, France