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

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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

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 : October 2032
Estimated Study Completion Date : October 2032



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: yearly from 3 years to 15 years ]
    Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality

  2. 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

  3. 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

  4. 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

  5. Change in medical conditions [ Time Frame: yearly from 3 years to 10 years ]
    Weight and complete clinical exam

  6. Key medical events related to WAS [ Time Frame: yearly from 3 years to 10 years ]
    Eczema status, infections, bleeding symptoms, autoimmune manifestation

  7. Hematological reconstitution [ Time Frame: yearly from 3 years to 10 years ]
    CBC including platelets count and size

  8. 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


Secondary Outcome Measures :
  1. Need for associated treatments [ Time Frame: yearly from 3 years to 15 years ]
    Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions

  2. 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

  3. Bone marrow content [ Time Frame: yearly from 3 years to 5 years (optional) ]
    Numbers and type of cells in bone marrow



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


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 ClinicalTrials.gov identifier (NCT number): NCT02333760


Locations
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France
Hopital Necker - Enfants Malades
Paris, France, 75743
United Kingdom
UCL Institute of Child Health
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Genethon
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Responsible Party: Genethon
ClinicalTrials.gov Identifier: NCT02333760    
Other Study ID Numbers: GNT-WAS-03
First Posted: January 7, 2015    Key Record Dates
Last Update Posted: June 3, 2021
Last Verified: May 2021
Additional relevant MeSH terms:
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Wiskott-Aldrich Syndrome
Syndrome
Disease
Pathologic Processes
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphopenia
Leukopenia
Leukocyte Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Immunologic Deficiency Syndromes
Immune System Diseases