Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID (NCT02999984) | Clinical Trial Compass
CompletedPhase 1/2
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
United States10 participantsStarted 2016-12-16
Plain-language summary
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 (Elongation Factor 1α Short form) Lentiviral Vector (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 Human Leukocyte Antigen (HLA) matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who successfully receive the OTL-101 product, pegademase bovine (PEG-ADA) Enzyme Replacement Therapy (ERT) is discontinued at Day+30 (-3/+15) 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.
Who can participate
Age range30 Days – 17 Years
SexALL
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Inclusion criteria
✓. 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,
✓. Subjects ≥30 days and \<18 years of age,
✓. With a diagnosis of ADA-SCID based on:
✓. Ineligible for matched family allogeneic Bone Marrow (BM) transplantation, defined as the absence of a medically eligible HLA-identical sibling or family donor, with normal immune function, who could serve as an allogeneic bone marrow donor.
✓. Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2.
✓. Subjects and their parents/legal guardians must be willing and able to comply with study restrictions and to remain at the clinic for the required duration during the study period and willing to return to the clinic for the follow up evaluation as specified in the protocol.
Exclusion criteria
✕. Ineligible for autologous Hematopoietic Stem Cell Transplantation (HSCT) as per clinical site criteria.
What they're measuring
1
Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months)
Timeframe: 6 months
2
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
Timeframe: 12 Months
3
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
✕. 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.
✕. Hematologic abnormality, defined as:
✕. Pulmonary abnormality, defined as:
✕. Cardiac abnormality, defined as:
✕. Neurologic abnormality, defined as:
✕. Renal abnormality, defined as:
✕. Hepatic/gastrointestinal abnormality, defined as: