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Active, Not Recruiting

NCT Number: NCT02333760

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Wiskott-Aldrich Syndrome Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cytopenia Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Immune System Diseases Immunologic Deficiency Syndromes Leukocyte Disorders Leukopenia Lymphopenia Primary Immunodeficiency Diseases

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Hopital Necker - Enfants Malades, Paris, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Autologous CD34+ cells transduced with WASP lentiviral vector

Genetic

Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Genethon

Other

Registry information

Official study 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).

Acronym: WASFUP

Important dates

Study start
2014
Primary completion
2032
Study completion
2032
First posted
Jan 7, 2015
Registry last updated
Jun 3, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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