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Gene Therapy for WAS

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ClinicalTrials.gov Identifier: NCT01347346
Recruitment Status : Completed
First Posted : May 4, 2011
Last Update Posted : May 22, 2018
Sponsor:
Collaborator:
Hôpital Necker-Enfants Malades
Information provided by (Responsible Party):
Genethon

May 3, 2011
May 4, 2011
May 22, 2018
May 2011
January 13, 2016   (Final data collection date for primary outcome measure)
  • Improvement in the eczema status [ Time Frame: 2 years ]
    Improvement in eczema status as compared with the baseline status at study entry on clinical evaluation
  • 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
  • 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
  • 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
  • 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
  • number of patients safely receiving the conditioning regimen [ Time Frame: 6 weeks ]
    safety of conditioning regimen is measured by hematopoietic recovery within 6 weeks.
  • number of patients whose stem cells are safely transduced [ Time Frame: 1 week ]

    safety of transduction procedure is determined prior to transplantation and is assessed through:

    • transduced cells number determination
    • cell viability measure
    • RCL detection
  • number of patients with engraftment of genetically corrected hematopoietic progenitors/differentiated cells [ Time Frame: 2 years ]
    engraftment is assessed by evidence of vector sequences or transgene expression in the cells.
  • number of patients with reconstituted cell mediated and humoral immunity [ Time Frame: 2 years ]
    reconstitution of cell mediated and humoral immunity is assessed by evidence of changes in T cell function and circulating immunoglobulin levels
  • number of patients with corrected microthrombocytopenia [ Time Frame: 2 years ]
    correction of microthrombocytopenia is assessed by blood platelet counts, expected to rise above 50,000/mm3
Complete list of historical versions of study NCT01347346 on ClinicalTrials.gov Archive Site
  • Occurrence and type of adverse events [ Time Frame: 2 years ]
    Occurrence and type of adverse events reported during the course of the study
  • Change in medical conditions [ Time Frame: 2 years ]
    Assessment of weight, vital signs, ECG and laboratory exams during the course of the study
  • 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
  • Improvement of microthrombocytopenia [ Time Frame: 3, 6, 12, 24 months ]
    Improvement of microthrombocytopenia as compared with the baseline evaluation at study entry
  • 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
  • Evidence of sustained engraftment 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
  • 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
  • number of patients with reduced frequency of infection [ Time Frame: 2 years ]
    reduction in frequency of infection is evaluated by clinical histroy, complete physical examinations, heamatological and microbiological tests
  • number of patients with resolution/reduction of autoimmunity [ Time Frame: 2 years ]
    resolution/reduction of autoimmunity is considered as a decrease from baseline observations assessed by clinical examination
  • number of patients with improvement in eczema [ Time Frame: 2 years ]
    improvement of eczema is considered as a decrease from baseline observations assessed by clinical examinations
  • number of patients with reduction in bruising and bleeding episodes [ Time Frame: 2 years ]
    reduction in bruising and bleeding episodes is assessed by clinical monitoring
Not Provided
Not Provided
 
Gene Therapy for WAS
Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
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.
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Wiskott-Aldrich Syndrome
Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
5
Same as current
January 9, 2017
January 13, 2016   (Final data collection date for primary outcome measure)

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 after 3 month search
  • lack of a 10/10 or 9/10 antigen HLA-matched unrelated donor after 3 month search
  • lack of a HLA-matched 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 or 9/10 antigen HLA-matched unrelated donor or with HLA-matched cord blood
  • contraindication to leukapheresis
  • contraindication to bone marrow harvest
  • contraindication to administration of conditioning medication
  • HIV positive patient
Sexes Eligible for Study: Male
Child, Adult, Older Adult
No
Contact information is only displayed when the study is recruiting subjects
France
 
 
NCT01347346
GTG003.08
2009-011152-22 ( EudraCT Number )
Yes
Not Provided
Not Provided
Genethon
Genethon
Hôpital Necker-Enfants Malades
Not Provided
Genethon
May 2018

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