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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02333760
Recruitment Status : Recruiting
First Posted : January 7, 2015
Last Update Posted : August 21, 2019
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment Phase
Wiskott-Aldrich Syndrome Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
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 Start Date : September 2014
Estimated Primary Completion Date : December 2027
Estimated Study Completion Date : December 2027

Intervention Details:
  • 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

Primary Outcome Measures :
  1. Incidence and type of SAEs [ Time Frame: 36 months ]
    Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality

  2. Lentiviral integration sites [ Time Frame: 36 months ]
    Presence of lentiviral integration sites in different cells sub-populations

  3. Vector copy numbers [ Time Frame: 36 months ]
    Quantification of vector copy numbers on sorted cells population by q-PCR

  4. Replication competent lentivirus (RCL) [ Time Frame: 36 months ]
    Presence of RCL

  5. Change in medical conditions [ Time Frame: 36 months ]
    Weight and complete clinical exam

  6. Key medical events related to WAS [ Time Frame: 36 months ]
    Eczema status, infections, bleeding symptoms, autoimmune manifestation

  7. Hematological reconstitution [ Time Frame: 36 months ]
    CBC including platelets count and size

  8. 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

Secondary Outcome Measures :
  1. Need for associated treatments [ Time Frame: 36 months ]
    Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions

  2. Representation of TCR families [ Time Frame: 36 months ]
    Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel

  3. Bone marrow content [ Time Frame: 36 months ]
    Numbers and type of cells in bone marrow

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02333760

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Hopital Necker - Enfants Malades Recruiting
Paris, France, 75743
Contact: Marina Cavazzana, MD, PHD    +33 (0)1 44 49 50 68   
Principal Investigator: Marina Cavazzana, MD, PHD         
United Kingdom
UCL Institute of Child Health Recruiting
London, United Kingdom, WC1N 1EH
Contact: Adrian Thrasher, MD, PHD    +44(0) 207 905 2660   
Principal Investigator: Adrian Thrasher, MD, PHD         
Sponsors and Collaborators
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Responsible Party: Genethon Identifier: NCT02333760    
Other Study ID Numbers: GNT-WAS-03
First Posted: January 7, 2015    Key Record Dates
Last Update Posted: August 21, 2019
Last Verified: August 2019
Additional relevant MeSH terms:
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Wiskott-Aldrich Syndrome
Pathologic Processes
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhagic Disorders
Leukocyte Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Immunologic Deficiency Syndromes
Immune System Diseases