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NCT Number: NCT01347242

Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.

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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

Great Ormond Street Hospital, London, United Kingdom

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About this study

This clinical trial is an ex vivo gene therapy trial. The investigational product corresponds to autologous CD34+ cells transduced with a lentiviral vector harboring the human WASP gene.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • males of all ages
  • severe WAS (clinical score 3-5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
  • molecular confirmation by WAS gene DNA sequencing
  • lack of HLA-genotypically identical bone marrow or of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search
  • parental, guardian, patient signed informed consent/assent
  • willing to return for follow-up
  • only for patients who have received previous allogenic hematopoietic stem cell transplant:
  • failed allogenic hematopoietic stem cell transplant
  • contraindication to repeat transplantation

Exclusion criteria

  • patient with HLA-genotypically identical bone marrow
  • patient with 10/10 antigen HLA-matched unrelated donor or cord blood
  • contraindication to leukapheresis
  • contraindication to bone marrow harvest
  • contraindication to administration of conditioning medication
  • HIV positive patient

Treatment and study plan

Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene

Genetic

transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene

Primary outcomes

  1. Improvement in the eczema status

    Time frame: 2 years

    Improvement in the eczema status as compared with the baseline status at study entry on clinical evaluation

  2. Reduction in the frequency and severity of infection episodes

    Time frame: 2 years

    Reduction in the frequency and severity of infection episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry

  3. Reduction in the frequency and severity of bruising and bleeding episodes

    Time frame: 2 years

    Reduction in the frequency and severity of bruising and bleeding episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry

  4. Reduction in the frequency and severity of autoimmune disorders

    Time frame: 2 years

    Reduction in the frequency and severity of autoimmune disorders as compared with the baseline status at study entry

  5. Reduction in the number of disease related days of hospitalization

    Time frame: 2 years

    Reduction in the number of disease related days of hospitalization as compared with the patient's historical data collected over the 2 years prior to study entry

Secondary outcomes

  1. Occurrence and type of adverse events

    Time frame: 2 years

    Occurrence and type of adverse events reported during the course of the study

  2. Change in medical conditions

    Time frame: 2 years

    Assessment of weight, vital signs, ECG and laboratory exams during the course of the study

  3. Safety of lentivirus gene transfer into Hematopoietic Stem Cells

    Time frame: 3, 6, 12, 24 months / 6, 12, 18, 24 months

    Detection of replication competent lentivirus (RCL) and lentivirus integration sites analysis

  4. Improvement of microthrombocytopenia

    Time frame: 3, 6, 12, 24 months

    Improvement of microthrombocytopenia as compared with the baseline evaluation at study entry

  5. Decrease in the number and volume of platelets transfusions

    Time frame: 2 years

    Decrease in the number and volume of platelets transfusions as compared with patient's historical data collected over the 2 years prior to study entry

  6. Evidence of sustained engrafment of WASP-expressing transduced cells

    Time frame: 6 weeks, 1, 3, 6, 9, 12, 18 & 24 months

    Quantification of vector copy numbers and detection of vector-derived WASP expression

  7. Reconstitution of humoral and cell mediated immunity

    Time frame: 9, 12, 18 & 24 months

    Reconstitution of humoral and cell mediated immunity as compared with the baseline evaluation at study entry

Sponsors and collaborators

Lead sponsor

Genethon

Other

Collaborators

  • Great Ormond Street Hospital for Children NHS Foundation Trust
  • Institute of Child Health

Registry information

Official study title

Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome

Important dates

Study start
2011
Primary completion
2019
Study completion
2019
First posted
May 4, 2011
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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