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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03837483
Other study ID # OTL-103-4
Secondary ID 2018-003842-18
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date January 21, 2019
Est. completion date September 2027

Study information

Verified date March 2023
Source Fondazione Telethon
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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 the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 10
Est. completion date September 2027
Est. primary completion date September 2025
Accepts healthy volunteers No
Gender All
Age group N/A to 65 Years
Eligibility Inclusion Criteria: - 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 Criteria: - 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

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
OTL-103
Autologous hematopoietic stem cells collected from mobilized peripheral blood transduced ex vivo with a lentiviral vector encoding the WAS cDNA

Locations

Country Name City State
Italy Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Milan
United States Children's Healthcare of Atlanta, Inc Atlanta Georgia

Sponsors (2)

Lead Sponsor Collaborator
Fondazione Telethon Ospedale San Raffaele

Countries where clinical trial is conducted

United States,  Italy, 

Outcome

Type Measure Description Time frame Safety issue
Primary Annualized rate of severe infections from 6 to 18 months after gene therapy compared with 1 year before gene therapy 18 months
Primary Annualized rate of moderate and severe bleeding episodes up to 1 year after gene therapy compared with 1 year before gene therapy 12 months
Secondary Evaluation of the overall survival 36 months
Secondary Number of patients with Vector copy number (VCN)/cell > 0.1 measured in peripheral blood-derived CD3+ cells 2 years
Secondary Percentage of WAS protein expression increased from pre-treatment levels in lymphocytes 2 years
Secondary Percentage of WAS protein expression increased from pre-treatment levels in platelets 2 years
Secondary Number of participants with successful engraftment of OTL-103 Engraftment of of OTL-103 is measured by hematological reconstitution of an absolute neutrophil count > 500 cell/ul 6 months
Secondary The number of subjects presenting with malignancies or abnormal clonal proliferation 2 years
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