Status:
ACTIVE_NOT_RECRUITING
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
Lead Sponsor:
Fondazione Telethon
Collaborating Sponsors:
Ospedale San Raffaele
Conditions:
Wiskott-Aldrich Syndrome
Eligibility:
All Genders
Up to 65 years
Phase:
PHASE3
Brief Summary
This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for ...
Eligibility Criteria
Inclusion
- Age: up to 65 years
- Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
- Severe Wiskott-Aldrich Syndrome (WAS) gene mutation, defined by literature data (genotype/phenotype studies).;
- Absent WASP expression, assessed by flow cytometry;
- Severe clinical score (Zhu clinical score ≥ 3);
- No human leukocyte antigen (HLA)-identical related donor available for hematopoietic stem cells transplant (HSCT).
Exclusion
- End-organ dysfunction, severe active infection not responsive to treatment or other severe disease or clinical condition which, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
- Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome.
- Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukaemia , or other serious haematological disorders
- Documented human immunodeficiency virus (HIV) infection
- Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin
- Symptomatic herpes zoster, not responsive to specific treatment
- Evidence of acute tuberculosis
- Acute or chronic stable Hepatitis B
- Presence of positive Hepatitis C RNA test result at screening
- Patients not eligible for mobilization protocols in order to obtain CD34+ cells
- Previous Gene Therapy
Key Trial Info
Start Date :
January 21 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2027
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT03837483
Start Date
January 21 2019
End Date
September 1 2027
Last Update
September 29 2025
Active Locations (2)
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1
Children's Healthcare of Atlanta, Inc
Atlanta, Georgia, United States, 30329
2
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, Italy, 20132