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Trial record 38 of 1177 for:    adenosine

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

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ClinicalTrials.gov Identifier: NCT04049084
Recruitment Status : Enrolling by invitation
First Posted : August 7, 2019
Last Update Posted : August 7, 2019
Sponsor:
Collaborators:
University of California, Los Angeles
Great Ormond Street Hospital for Children NHS Foundation Trust
Information provided by (Responsible Party):
Orchard Therapeutics

Brief Summary:
This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human adenosine deaminase (ADA) gene (EFS-ADA LV), as part of the OTL-101 clinical development program. No investigational medicinal product will be administered to these patients as part of the OTL-101-6 study.

Condition or disease Intervention/treatment
Adenosine Deaminase Deficiency Severe Combined Immunodeficiency (SCID) Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)

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Study Type : Observational
Estimated Enrollment : 70 participants
Observational Model: Other
Time Perspective: Other
Official Title: An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)
Estimated Study Start Date : September 2019
Estimated Primary Completion Date : August 2035
Estimated Study Completion Date : August 2035



Intervention Details:
  • Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
    ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)


Primary Outcome Measures :
  1. Overall Survival [ Time Frame: 15 years post-treatment ]
    Establishing efficacy through overall survival

  2. Event-Free Survival [ Time Frame: 15 years post-treatment ]
    Establishing efficacy through event-free survival

  3. Incidence of Adverse Events [ Time Frame: 15 years post-treatment ]
    Long-Term Safety incidence of AEs and SAEs



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This observational LTFU study is open for enrollment to ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA lentiviral vector as part of the OTL-101 clinical development program. Patients treated in clinical trials as part of IND #15440, study OTL-101-5 (EudraCT No 2017-001275-23) or study 10-MI-29 (EudraCT No 2010-024253-36), as well as in Expanded Access or Compassionate Use Programs, will be eligible for enrollment.
Criteria

Inclusion Criteria:

A patient is eligible for enrollment in the study if all of the following criteria are met:

  1. the patient has been treated with an autologous ex vivo gene therapy product based on the EFS-ADA LV, as part of the OTL-101 clinical development program;
  2. the patient displays persistent detectable gene marking, as determined by the Investigator;
  3. the patient or, if applicable, the patient's parent(s)/legal guardian(s), are able and willing to provide informed consent.

Exclusion Criteria:

  • There are no exclusion criteria for participation in this observational LTFU study.

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): NCT04049084


Locations
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United States, California
Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
Los Angeles, California, United States, 90095
United Kingdom
UCL Great Ormond Street Institute of Child Health
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Orchard Therapeutics
University of California, Los Angeles
Great Ormond Street Hospital for Children NHS Foundation Trust
Investigators
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Study Director: Orchard Clinical Trials Orchard Therapeutics

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Responsible Party: Orchard Therapeutics
ClinicalTrials.gov Identifier: NCT04049084     History of Changes
Other Study ID Numbers: OTL-101-6
First Posted: August 7, 2019    Key Record Dates
Last Update Posted: August 7, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Orchard Therapeutics:
OTL-101
Additional relevant MeSH terms:
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Adenosine
Agammaglobulinemia
Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Blood Protein Disorders
Hematologic Diseases
Lymphoproliferative Disorders
Lymphatic Diseases
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Arrhythmia Agents
Vasodilator Agents
Purinergic P1 Receptor Agonists
Purinergic Agonists
Purinergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action