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

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ClinicalTrials.gov Identifier: NCT03837483
Recruitment Status : Recruiting
First Posted : February 12, 2019
Last Update Posted : September 23, 2019
Sponsor:
Collaborator:
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Information provided by (Responsible Party):
Orchard Therapeutics

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.

Condition or disease Intervention/treatment Phase
Wiskott-Aldrich Syndrome Genetic: OTL-103 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : January 21, 2019
Estimated Primary Completion Date : February 2022
Estimated Study Completion Date : December 2022


Arm Intervention/treatment
Experimental: Gene Therapy
OTL-103, Autologous CD34+ hematopoietic stem cells transduced ex vivo with a lentiviral vector encoding the human WAS gene
Genetic: OTL-103
Autologous hematopoietic stem cells collected from mobilized peripheral blood transduced ex vivo with a lentiviral vector encoding the WAS cDNA




Primary Outcome Measures :
  1. Number of participants with successful engraftment of OTL-103 [ Time Frame: 6 months ]

    Engraftment of of OTL-103 is measured by the following parameters:

    • hematological reconstitution of an absolute neutrophil count > 500 cell/ul
    • a vector copy number of > 0.1 in peripheral blood-derived CD3+ cells
    • WAS protein expression in lymphocytes greater than pre-treatment values


Secondary Outcome Measures :
  1. Number of patients with Vector copy number (VCN)/cell > 0.1 measured in peripheral blood-derived CD3+ cells [ Time Frame: 2 years ]
  2. Percentage of WAS protein expression increased from pre-treatment levels in lymphocytes and platelets [ Time Frame: 2 years ]
  3. Evaluate the number of vaccinations per subject after treatment with gene therapy treatment (OTL-103) [ Time Frame: 2 years ]
  4. The number of subjects presenting with malignancies or abnormal clonal proliferation [ Time Frame: 2 years ]


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Ages Eligible for Study:   up to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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);
    • Family member affected by WAS with life-threatening or fatal clinical events.
  • No human leukocyte antigen (HLA)-identical related donor available for hematopoietic stem cells transplant (HSCT).
  • Parental/guardian/subject-signed informed consent, and subject assent (if appropriate).
  • For all subjects in the reproductive age range, agreement to use highly effective and adequate method of contraception

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03837483


Contacts
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Contact: Orchard Medical Information +44 (0) 20 3808 8286 medinfo@orchard-tx.com
Contact: Orchard Clinical Trials medinfo@orchard-tx.com

Locations
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Italy
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Recruiting
Milan, Italy, 20132
Contact: Alessandro Aiuti    +39 0226434472    aiuti.alessandro@hsr.it   
Sponsors and Collaborators
Orchard Therapeutics
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Investigators
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Study Director: Orchard Clinical Trials Orchard Therapeutics (Europe) Limited

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Responsible Party: Orchard Therapeutics
ClinicalTrials.gov Identifier: NCT03837483     History of Changes
Other Study ID Numbers: OTL-103-4
2018-003842-18 ( EudraCT Number )
First Posted: February 12, 2019    Key Record Dates
Last Update Posted: September 23, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Wiskott-Aldrich Syndrome
Syndrome
Disease
Pathologic Processes
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphopenia
Leukopenia
Leukocyte Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Immunologic Deficiency Syndromes
Immune System Diseases