Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE1/PHASE2
6 participants
INTERVENTIONAL
2011-03-31
2019-11-13
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
NCT03019809
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
NCT00160355
Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
NCT00885833
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
NCT01319851
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
NCT01565616
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
study treatment
autologous CD34 positive cells transduced with a lentiviral vector containing the human WAS gene
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
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
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
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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 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
MALE
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Great Ormond Street Hospital for Children NHS Foundation Trust
OTHER
Institute of Child Health
OTHER
Genethon
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Great Ormond Street Hospital
London, , United Kingdom
Royal Free Hospital
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Magnani A, Semeraro M, Adam F, Booth C, Dupre L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24.
Morris EC, Fox T, Chakraverty R, Tendeiro R, Snell K, Rivat C, Grace S, Gilmour K, Workman S, Buckland K, Butler K, Chee R, Salama AD, Ibrahim H, Hara H, Duret C, Mavilio F, Male F, Bushman FD, Galy A, Burns SO, Gaspar HB, Thrasher AJ. Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. Blood. 2017 Sep 14;130(11):1327-1335. doi: 10.1182/blood-2017-04-777136. Epub 2017 Jul 17.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
GTG002.07
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.