Observational LTFU study for pts previously treated with ex vivo GT

• Research type

  Research Study

• Full 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)

• IRAS ID

  264553

• Contact name

  Claire Booth

• Contact email

  c.booth@ucl.ac.uk

• Sponsor organisation

  Orchard Therapeutics (Europe) Ltd.

• Clinicaltrials.gov Identifier

  N/A, N/A

• Duration of Study in the UK

  15 years, 0 months, 0 days

• Research summary

  This is a long-term follow-up study funded by the sponsor, Orchard Therapeutics (a biotechnology company), and defined as a non-interventional study. The participants in the OTL-101-6 will not receive any IMP. These participants will be patients previously treated with autologous ex vivo gene therapy products for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), as part of the OTL-101 clinical programme. Participants will be followed up to 15 years from treatment with the previously administered gene therapy product to collect information about their health in order to understand the long-term safety and efficacy of the treatment received.

  Approximately 70 patients will participate in this study. Enrollment in this study is not expected to lead to additional pre-specified clinical assessments or visits beyond those of routine clinical practice for ADA-SCID patients treated with gene therapy.

  Participants will be asked to undergo assessments performed under routine clinical practice, which may include collection of the following information from their medical records:
  o Demographics and disease history (collected only at the start of this study)
  o Height and weight
  o Medication use and medical treatments
  o Adverse events and safety concerns
  o Quality of life (QoL) and performance status measures (These assessments are used to evaluate the particpant's average well-being and ability to perform activities of daily living)
  o Laboratory assessments

  The above information will be collected annually for 15 years post gene therapy as follows:
  • During on-site visits at site or their local health care provider for the first 5 years after gene therapy treatment
  • During phone calls by site or their local health care provider for the next 10 years (this information may be collected at in person visits, although this is not a requirement).

  A patient diary will be provided to the participant's parents which can be used to record any adverse events or safety concerns that happen in between routine study visits.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  19/LO/0983

• Date of REC Opinion

  24 Sep 2019

• REC opinion

  Further Information Favourable Opinion