Trial · NCT01852071
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
NCT01852071, Phase 1, Phase 2, Completed, University of California, Los Angeles; results first posted 2021-09-20
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene. Trials, record NCT01852071. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01852071
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
- NCT number
- NCT01852071
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 46 participants, actual
- Lead sponsor
- University of California, Los Angeles
- Conditions
- ADA-SCID
- Interventions
- Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT
- First posted
- 2013-05-13
- Last updated
- 2022-08-03
- Results first posted
- 2021-09-20
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | 12 months | percentage of participants | NUMBER | |
| PRIMARY | Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | 12 months | percentage of participants | NUMBER | |
| SECONDARY | OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) | 24 months | percentage of participants | NUMBER | |
| SECONDARY | EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) | 24 months | percentage of participants | NUMBER | |
| SECONDARY | Vector Copy Number (VCN) in Peripheral Blood (PB) Granulocytes. | 24 months | Historical control groups/arms are not included in the analysis population for this outcome measure as these subjects did not receive gene therapy, and so measurement of VCN levels is not relevant | copies/cell | MEDIAN |
| SECONDARY | VCN in Peripheral Blood Mononuclear Cells (PBMCs) | 24 months | Historical control groups/arms are not included in the analysis population for this outcome measure as these subjects did not receive gene therapy, and so measurement of VCN levels is not relevant | copies/cell | MEDIAN |
| SECONDARY | ADA Activity in Erythrocytes | 24 months | nmol/h/mg | MEDIAN | |
| SECONDARY | Reduction in Deoxyadenosine Nucleotide (dAXP) in Erythrocytes | 24 months | HSCT Controls With MRD arm is not included in the analysis population for this outcome measure as dAXP data was not reported between 17-30 months post-HSCT for any patients in this arm. | umol/mL | MEDIAN |
| SECONDARY | Change From Baseline in CD3+ T Cell Counts (2 Years) | 24 months | cells/μL | MEDIAN | |
| SECONDARY | Number of Single Integration Sites Representing >30% of the Total Integration Sites (2 Years) | 24 months | Historical control groups/arms are not included in the analysis population for this outcome measure as these subjects did not receive gene therapy, and so measurement of VISA is not relevant | number of participants | NUMBER |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group34
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | OTL-101 Gene Therapy | 100 | 95% Confidence Interval 83.16 to 100 | percentage of participants | |
| Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | HSCT Controls Without MRD | 85.71 | 95% Confidence Interval 57.19 to 98.22 | percentage of participants | |
| Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | HSCT Controls With MRD | 100 | 95% Confidence Interval 73.54 to 100 | percentage of participants | |
| Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | All HSCT Control Group | 92.31 | 95% Confidence Interval 74.87 to 99.05 | percentage of participants | |
| Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | OTL-101 Gene Therapy | 100 | 95% Confidence Interval 83.16 to 100 | percentage of participants | |
| Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | HSCT Controls Without MRD | 64.29 | 95% Confidence Interval 35.14 to 87.24 | percentage of participants | |
| Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | HSCT Controls With MRD | 100 | 95% Confidence Interval 73.54 to 100 | percentage of participants | |
| Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) | All HSCT Control Group | 80.77 | 95% Confidence Interval 60.65 to 93.45 | percentage of participants | |
| OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) | OTL-101 Gene Therapy | 100 | 95% Confidence Interval 83.16 to 100 | percentage of participants | |
| OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years) | HSCT Controls Without MRD | 85.71 | 95% Confidence Interval 57.19 to 98.22 | percentage of participants |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Gene Therapy | 20 | |
| Overall Study | STARTED | Historical Control Group | 26 | |
| Overall Study | COMPLETED | Gene Therapy | 20 | |
| Overall Study | COMPLETED | Historical Control Group | 26 | |
| Overall Study | NOT COMPLETED | Gene Therapy | 0 | |
| Overall Study | NOT COMPLETED | Historical Control Group | 0 |
Source ClinicalTrials.gov
Adverse events83
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Neutropenia | Blood and lymphatic system disorders | OTL-101 Gene Therapy | 17 | 20 | 31 |
| other | Anemia | Blood and lymphatic system disorders | OTL-101 Gene Therapy | 13 | 20 | 18 |
| other | Thrombocytopenia | Blood and lymphatic system disorders | OTL-101 Gene Therapy | 3 | 20 | 3 |
| other | Lymphadenopathy | Blood and lymphatic system disorders | OTL-101 Gene Therapy | 2 | 20 | 2 |
| other | Leukopenia | Blood and lymphatic system disorders | OTL-101 Gene Therapy | 1 | 20 | 1 |
| other | Upper respiratory tract infection | Infections and infestations | OTL-101 Gene Therapy | 5 | 20 | 8 |
| other | Rhinovirus infection | Infections and infestations | OTL-101 Gene Therapy | 4 | 20 | 4 |
| other | Otitis media | Infections and infestations | OTL-101 Gene Therapy | 4 | 20 | 8 |
| other | Influenza | Infections and infestations | OTL-101 Gene Therapy | 1 | 20 | 1 |
| other | Nasopharyngitis | Infections and infestations | OTL-101 Gene Therapy | 2 | 20 | 2 |
1 to 10 of 83Source ClinicalTrials.gov
See all 83 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | OTL-101 Gene Therapy | 0 | 20 |
| Total, other adverse events | OTL-101 Gene Therapy | 20 | 20 |
| Total, serious adverse events | OTL-101 Gene Therapy | 9 | 20 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants2
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|---|---|---|---|---|---|
| U01AI100801 | EFS-ADA Lentiviral Vector Transduction of Bone Marrow CD34+ Cells for ADA-SCID | KOHN, DONALD B | UNIVERSITY OF CALIFORNIA LOS ANGELES | NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES | 2012 to 2016 | NIH RePORTER |
| P01HL073104 | Gene Therapy Using Hematopoietic Stem Cells | KOHN, DONALD B | UNIVERSITY OF CALIFORNIA LOS ANGELES | NATIONAL HEART, LUNG, AND BLOOD INSTITUTE | 2003 to 2013 | NIH RePORTER |
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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 trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01852071
GET https://conductscience.org/api/trials/v1/NCT01852071/results?page=2