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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02333760
Recruitment Status : Active, not recruiting
First Posted : January 7, 2015
Last Update Posted : June 3, 2021
Sponsor:
Information provided by (Responsible Party):
Genethon

Tracking Information
First Submitted Date  ICMJE October 28, 2014
First Posted Date  ICMJE January 7, 2015
Last Update Posted Date June 3, 2021
Study Start Date  ICMJE September 2014
Estimated Primary Completion Date October 2032   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 31, 2021)
  • Incidence and type of SAEs [ Time Frame: yearly from 3 years to 15 years ]
    Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
  • Lentiviral integration sites [ 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) ]
    Presence of lentiviral integration sites in different cells sub-populations
  • Vector copy numbers [ 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) ]
    Quantification of vector copy numbers on sorted cells population by q-PCR
  • Replication competent lentivirus (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) ]
    Presence of RCL
  • Change in medical conditions [ Time Frame: yearly from 3 years to 10 years ]
    Weight and complete clinical exam
  • Key medical events related to WAS [ Time Frame: yearly from 3 years to 10 years ]
    Eczema status, infections, bleeding symptoms, autoimmune manifestation
  • Hematological reconstitution [ Time Frame: yearly from 3 years to 10 years ]
    CBC including platelets count and size
  • Reconstitution of cell mediated and humoral immunity [ Time Frame: 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
Original Primary Outcome Measures  ICMJE
 (submitted: January 5, 2015)
  • Incidence and type of SAEs [ Time Frame: 36 months ]
    Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
  • Lentiviral integration sites [ Time Frame: 36 months ]
    Presence of lentiviral integration sites in different cells sub-populations
  • Vector copy numbers [ Time Frame: 36 months ]
    Quantification of vector copy numbers on sorted cells population by q-PCR
  • Replication competent lentivirus (RCL) [ Time Frame: 36 months ]
    Presence of RCL
  • Change in medical conditions [ Time Frame: 36 months ]
    Weight and complete clinical exam
  • Key medical events related to WAS [ Time Frame: 36 months ]
    Eczema status, infections, bleeding symptoms, autoimmune manifestation
  • Hematological reconstitution [ Time Frame: 36 months ]
    CBC including platelets count and size
  • Reconstitution of cell mediated and humoral immunity [ Time Frame: 36 months ]
    Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 31, 2021)
  • Need for associated treatments [ Time Frame: yearly from 3 years to 15 years ]
    Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions
  • Representation of TCR families [ Time Frame: yearly from 3 years to 5 years ]
    Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel
  • Bone marrow content [ Time Frame: yearly from 3 years to 5 years (optional) ]
    Numbers and type of cells in bone marrow
Original Secondary Outcome Measures  ICMJE
 (submitted: January 5, 2015)
  • Need for associated treatments [ Time Frame: 36 months ]
    Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions
  • Representation of TCR families [ Time Frame: 36 months ]
    Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel
  • Bone marrow content [ Time Frame: 36 months ]
    Numbers and type of cells in bone marrow
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
Official Title  ICMJE 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).
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Wiskott-Aldrich Syndrome
Intervention  ICMJE 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
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: January 5, 2015)
10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2032
Estimated Primary Completion Date October 2032   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02333760
Other Study ID Numbers  ICMJE GNT-WAS-03
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Genethon
Study Sponsor  ICMJE Genethon
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Genethon
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP