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Trial record 1 of 1 for:    NCT02999984
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Autologous Cryopreserved CD34+ Hematopoietic Cells Transduced With EFS-ADA Lentivirus for ADA SCID

This study is currently recruiting participants.
See Contacts and Locations
Verified January 2017 by Donald B. Kohn, M.D., University of California, Los Angeles
Sponsor:
Collaborators:
Orchard Therapeutics Limited
University College, London
California Institute for Regenerative Medicine
Information provided by (Responsible Party):
Donald B. Kohn, M.D., University of California, Los Angeles
ClinicalTrials.gov Identifier:
NCT02999984
First received: December 19, 2016
Last updated: January 4, 2017
Last verified: January 2017
  Purpose
This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS LV encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an HLA-matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product will be infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who have successfully received the OTL-101 product, PEG-ADA ERT will be discontinued at Day+30 (+/-3) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.

Condition Intervention Phase
Severe Combined Immunodeficiency Due to ADA Deficiency Biological: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With ADA Deficiency Severe Combined Immunodeficiency

Resource links provided by NLM:


Further study details as provided by Donald B. Kohn, M.D., University of California, Los Angeles:

Primary Outcome Measures:
  • Overall survival [ Time Frame: 12 months ]
    The proportion of subjects alive at the Month 12 visit.


Secondary Outcome Measures:
  • Event-free survival [ Time Frame: 24 months ]

Estimated Enrollment: 10
Study Start Date: December 2016
Estimated Study Completion Date: June 2020
Estimated Primary Completion Date: June 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Biological: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Infusion of OTL-101 after reduced intensity conditioning
Other Name: OTL-101

Detailed Description:

This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem cells transduced ex vivo with EFS LV encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years, who are not eligible for an HLA-matched sibling/family donor and meeting the inclusion/exclusion criteria.

The aim of this study is to assess the success of treatment for overall survival and event free survival.

Eligible subjects will be hospitalized to undergo the harvesting of autologous CD34+ cells. To enable the release of the cell product for infusion, the product must meet various quality control criteria for safety, identity, viability, purity and potency. If OTL-101 meets the acceptance criteria and is released, the subjects will be readmitted for conditioning prior to infusion of OTL-101.

For subjects who have successfully received the OTL-101 product, PEG-ADA ERT will be discontinued at Day+30 (+/-3) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples at Months 1, 3, 6, 9, 12, 18, and 24. Any medically-indicated interventions will be determined at these visits. After Month 24 visit, the subjects will have completed the study and may enter a long term registry.

  Eligibility

Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Eligible subjects aged ≥30 days and <18 years
  • Diagnosis of ADA-SCID
  • Ineligible for matched family allogeneic bone marrow transplantation

Exclusion Criteria:

  • Ineligible for autologous HSCT as per clinical site criteria
  • Other conditions which, in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the harvest of bone marrow, the administration of conditioning and the infusion of transduced cells, or which indicate an inability of the subject or subject's parent/legal guardian to comply with the protocol
  • Hematologic abnormality, defined as:

    • Anemia
    • Neutropenia
    • Thrombocytopenia
    • Coagulation abnormality
    • Cytogenetic abnormalities
    • Prior allogeneic HSCT with cytoreductive conditioning.
  • Pulmonary abnormality
  • Cardiac abnormality
  • Neurologic abnormality
  • Renal abnormality
  • Hepatic/gastrointestinal abnormality
  • Oncologic disease other than dermatofibrosarcoma protuberans (DFSP)
  • Known sensitivity to conditioning agents
  • Confirmation of an infectious disease (HIV-1, Hepatitis B, Parvovirus B19)
  • Pregnancy or major congenital anomaly
  • Is likely to require treatment during the study with drugs that are not permitted by the study protocol
  • Previous gene therapy procedure
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02999984

Contacts
Contact: Theodore B Moore, M.D. (310) 825-6708 tbmoore@mednet.ucla.edu
Contact: Satiro De Oliveira, MD t(310) 825-6708 sdeoliveira@mednet.ucla.edu

Locations
United States, California
University of California, Los Angeles Recruiting
Los Angeles, California, United States, 90095
Contact: Theodore B. Moore, M.D.    310-825-6708    tbmoore@mednet.ucla.edu   
Contact: Kit Shaw, Ph.D.    310-825-6188    kshaw@mednet.ucla.edu   
Principal Investigator: Theodore B. Moore, M.D.         
Sub-Investigator: Satiro De Oliveira, M.D.         
Sub-Investigator: Donald B. Kohn, M.D.         
Sponsors and Collaborators
University of California, Los Angeles
Orchard Therapeutics Limited
University College, London
California Institute for Regenerative Medicine
Investigators
Study Director: Donald B. Kohn, MD University of California, Los Angeles
  More Information

Additional Information:
Publications:
Responsible Party: Donald B. Kohn, M.D., Principal Investigator, University of California, Los Angeles
ClinicalTrials.gov Identifier: NCT02999984     History of Changes
Other Study ID Numbers: UCLA IRB#15-001678
Study First Received: December 19, 2016
Last Updated: January 4, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Severe Combined Immunodeficiency
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases

ClinicalTrials.gov processed this record on July 26, 2017