Back to resultsLong Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (WASFUP)
Primary Purpose
Wiskott-Aldrich Syndrome
Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Autologous CD34+ cells transduced with WASP lentiviral vector
Sponsored by
About this trial
This is an interventional other trial for Wiskott-Aldrich Syndrome
Eligibility Criteria
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.
Sites / Locations
- Hopital Necker - Enfants Malades
- UCL Institute of Child Health
Outcomes
Primary Outcome Measures
Incidence and type of SAEs
Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
Lentiviral integration sites
Presence of lentiviral integration sites in different cells sub-populations
Vector copy numbers
Quantification of vector copy numbers on sorted cells population by q-PCR
Replication competent lentivirus (RCL)
Presence of RCL
Change in medical conditions
Weight and complete clinical exam
Key medical events related to WAS
Eczema status, infections, bleeding symptoms, autoimmune manifestation
Hematological reconstitution
CBC including platelets count and size
Reconstitution of cell mediated and humoral immunity
Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene
Secondary Outcome Measures
Need for associated treatments
Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions
Representation of TCR families
Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel
Bone marrow content
Numbers and type of cells in bone marrow
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02333760
Brief Title
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
Acronym
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).
Study Type
Interventional
2. Study Status
Record Verification Date
May 2021
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 2014 (undefined)
Primary Completion Date
October 2032 (Anticipated)
Study Completion Date
October 2032 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genethon
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wiskott-Aldrich Syndrome
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Intervention Type
Genetic
Intervention Name(s)
Autologous CD34+ cells transduced with WASP lentiviral vector
Intervention Description
Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene
Primary Outcome Measure Information:
Title
Incidence and type of SAEs
Description
Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
Time Frame
yearly from 3 years to 15 years
Title
Lentiviral integration sites
Description
Presence of lentiviral integration sites in different cells sub-populations
Time Frame
yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Title
Vector copy numbers
Description
Quantification of vector copy numbers on sorted cells population by q-PCR
Time Frame
yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Title
Replication competent lentivirus (RCL)
Description
Presence of RCL
Time Frame
yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Title
Change in medical conditions
Description
Weight and complete clinical exam
Time Frame
yearly from 3 years to 10 years
Title
Key medical events related to WAS
Description
Eczema status, infections, bleeding symptoms, autoimmune manifestation
Time Frame
yearly from 3 years to 10 years
Title
Hematological reconstitution
Description
CBC including platelets count and size
Time Frame
yearly from 3 years to 10 years
Title
Reconstitution of cell mediated and humoral immunity
Description
Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene
Time Frame
yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Secondary Outcome Measure Information:
Title
Need for associated treatments
Description
Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions
Time Frame
yearly from 3 years to 15 years
Title
Representation of TCR families
Description
Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel
Time Frame
yearly from 3 years to 5 years
Title
Bone marrow content
Description
Numbers and type of cells in bone marrow
Time Frame
yearly from 3 years to 5 years (optional)
10. Eligibility
Sex
Male
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Hopital Necker - Enfants Malades
City
Paris
ZIP/Postal Code
75743
Country
France
Facility Name
UCL Institute of Child Health
City
London
ZIP/Postal Code
WC1N 1EH
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
35075289
Citation
Magnani A, Semeraro M, Adam F, Booth C, Dupre L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24. Erratum In: Nat Med. 2022 Oct;28(10):2217.
Results Reference
derived
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Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
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