A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-01-21

Study Completion Date

2027-09-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Wiskott-Aldrich Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Gene Therapy

OTL-103, Autologous CD34+ hematopoietic stem cells transduced ex vivo with a lentiviral vector encoding the human WAS gene

Group Type EXPERIMENTAL

OTL-103

Intervention Type GENETIC

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

Interventions

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OTL-103

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

Intervention Type GENETIC

Eligibility Criteria

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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

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No