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

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Brief Title

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

Official Title

A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS)

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

Study Phase

Phase 3

Study Type


Primary Outcome

Annualized rate of severe infections from 6 to 18 months after gene therapy compared with 1 year before gene therapy

Secondary Outcome

 Evaluation of the overall survival


Wiskott-Aldrich Syndrome



Study Arms / Comparison Groups

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


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

January 21, 2019

Completion Date

January 2024

Primary Completion Date

July 2023

Eligibility Criteria

        Inclusion Criteria:

          -  Age: up to 65 years

          -  Diagnosis of WAS defined by genetic mutation and at least one of the following

               -  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

          -  Previous Gene Therapy




N/A - 65 Years

Accepts Healthy Volunteers



Orchard Clinical Trials, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Orchard Therapeutics


 Ospedale San Raffaele

Study Sponsor

Orchard Clinical Trials, Study Director, Orchard Therapeutics (Europe) Limited

Verification Date

February 2022