Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protection from bacteria, viruses, and fungi. They are prone to repeated and persistent infections that can be very serious or life-threatening. If not treated in a way that restores immune function, children with SCID usually live only a year or two. MedDRA version: 20.1 Level: LLT Classification code 10066
Conditions
Interventions
Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells
Product Code: OTL-101
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: There is no recommended INN
Other descriptiv
Sponsors
Great Ormond Street Hospital for Children NHS Trust
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians and, where applicable according to local laws, a signed assent from the child, 2. Subjects =30 days and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Ineligible for autologous haematopoietic stem cell (HSC) procedure. 2. Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the harvest of bone marrow, the administration of busulfan 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 3. Haematologic abnormality, 4. Pulmonary abnormality, 5. Cardiac abnormality, 6. Neurologic abnormality, 7. Renal abnormality, 8. Hepatic/gastrointestinal abnormality, 9. Oncologic disease, 10. Known sensitivity to Busulfan, 11. Confirmation of an infectious disease by deoxyribonucleic acid (DNA) PCR positive at time of assessment for the following: HIV-1, Hepatitis B, Parvovirus B19, 12. The subject is pregnant or has a major congenital anomaly, 13. Is likely to require treatment during the study with drugs that are not permitted by the study protocol, 14. The subject has previously received another form of gene therapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - Efficacy: Evaluate the overall survival and event free survival 12 months post OTL-101 administration. - Safety: The safety and tolerability of OTL-101. ;Secondary Objective: - Evaluate the overall survival and event free survival 24 months post GTMP administration. - Immunoglobulin Replacement therapies prior to and after gene therapy. - Safety and tolerability. - Performance outcomes and quality of life will be measured by the Karnofsky/Lansky scale and questions relevant to general well-being, school attendance and ability to practice sports, respectively. - Evaluation of the frequency of severe infections or opportunistic infectious episodes; defines as infections or severe infections requiring hospitalisation or prolonged hospitalisation and/or documented infections by opportunistic pathogens (i.e. interstitial pneumonia, intractable diarrhoea). - Response to tetanus vaccination. - Immune reconstitution: T and B cell reconstitution.;Primary end point(s): Evaluate the overall survival and event free survival 12 months post OTL-101 administration. Overall survival is defined as the proportion of subjects alive. Event free survival is defined as the proportion of subjects alive with no “event”; an “event” being the resumption of PEG-ADA ERT or the need for a rescue allogenic HSCT, or death.;Timepoint(s) of evaluation of this end point: 12 months post OTL-101 administration | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Evaluate the overall survival and event free survival 24 months post OTL-101 administration - Immunoglobulin Replacement therapies prior to and after gene therapy. - Safety and tolerability. - Performance outcomes and quality of life will be measured by the Karnofsky/Lansky scale and questions relevant to general well-being, school attendance and ability to practice sports. - Evaluation of the frequency of severe infections or opportunistic infectious episodes, defines as infections or severe infections requiring hospitalisation or prolonging hospitalisation and/or documented infections by opportunistic pathogens (i.e. interstitial pneumonia, intractable diarrhoea). - Response to tetanus vaccination. - Immune reconstitution: T and B cell reconstitution.;Timepoint(s) of evaluation of this end point: 24 months post OTL-101 administration | — |
Countries
United Kingdom
Contacts
Public ContactCecile Duret
UCL Institute of Child Health
Outcome results
None listed