Trial · NCT01852071
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 11 of 11
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | 12 months | Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with OTL-101 or HSCT |
| Primary | Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | 12 months | Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogeneic Hematopoietic Stem Cell Transplant (HSCT), or death. |
| Secondary | OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) | 24 months | OS is defined as the percentage of subjects alive at 24 months post- treatment with OTL-101 or HSCT |
| Secondary | EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) | 24 months | Event-free survival is defined as the percentage of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death. |
| Secondary | Vector Copy Number (VCN) in Peripheral Blood (PB) Granulocytes. | 24 months | Vector copy number in the PB granulocyte fraction that was T cell depleted, is a surrogate for amount of engrafted genetically modified Hematopoietic stem cell (HSC) that are producing granulocytes every 3-5 days. VCN analysis was performed by Droplet Digital PCR (ddPCR) on DNA extracted from peripheral blood granulocytes. |
| Secondary | VCN in Peripheral Blood Mononuclear Cells (PBMCs) | 24 months | PBMC VCN is a measure of the accumulation of peripheral blood leukocytes arising from engrafted, genetically modified HSC. VCN analysis was performed by ddPCR on DNA extracted from PBMC. |
| Secondary | ADA Activity in Erythrocytes | 24 months | ADA enzyme activity measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV; persistence of ADA enzyme activity over time demonstrates successful engraftment and differentiation of genetically modified HSC. |
| Secondary | Reduction in Deoxyadenosine Nucleotide (dAXP) in Erythrocytes | 24 months | Decreased dAXP levels coincide with increased ADA enzyme activity, detoxification was used to demonstrate functional ADA enzyme production from the introduced ADA transgene. The threshold for detoxification was \<100 μmol/L. |
| Secondary | Change From Baseline in CD3+ T Cell Counts (2 Years) | 24 months | Immune reconstitution was assessed by change in CD3+ T Cell counts at baseline to Month 24. |
| Secondary | Number of Single Integration Sites Representing >30% of the Total Integration Sites (2 Years) | 24 months | Vector Integration Site Analysis (VISA) allowed determination of the distribution of vector integration sites in each subject's genome, as well as the relative clonal abundance. VISA was to be considered abnormal for a subject if, in 2 or more instances during the course of follow-up, a single integration site was found to represent \>30% of the total integration sites detected. |
| Secondary | Severe Infection Rate Excluding the First Three Months After Treatment | 24 months | The infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. Infections that took place in the first 3 months of follow-up post treatment were excluded from calculations to avoid possible bias introduced in the data by the effects of conditioning. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this list from software.
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GET https://conductscience.org/api/trials/v1/NCT01852071/registered