Trial · NCT01380990

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

NCT01380990, Phase 1, Phase 2, Completed, Great Ormond Street Hospital for Children NHS Foundation Trust; results first posted 2021-09-16

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency. Trials, record NCT01380990. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01380990

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
NCT number: NCT01380990
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 36 participants, actual
Lead sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
Conditions: Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
Interventions: Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), Busulfan, Peg-Ada
First posted: 2011-06-27
Last updated: 2021-09-16
Results first posted: 2021-09-16

Source ClinicalTrials.gov

Registered outcomes14

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 allogenic Hematopoietic Stem Cell Transplant (HSCT), or death.
Primary	Vector Copy Number (VCN) in Granulocytes Fraction (Neutrophils)	36 months	Engraftment of transduced cells was assessed using vector gene marking in granulocytes (neutrophils)
Primary	VCN in Peripheral Blood Mononuclear Cells (PBMCs)	36 months	Engraftment of transduced cells was assessed using vector gene marking in PBMCs
Primary	VCN in CD3+ T Cells	36 months	Engraftment of transduced cells was assessed using vector gene marking
Primary	VCN in CD19+ B Cells	36 months	Engraftment of transduced cells was assessed using vector gene marking in CD19+ B Cells
Primary	Change From Baseline in CD3+ T Cell Counts (1 Year)	12 months	Immune reconstitution was assessed by change in CD3+ T Cell counts over time.
Primary	Change From Baseline in CD3+ T Cell Counts (3 Years)	36 months	Immune reconstitution was assessed by change in CD3+ T Cell counts over time.
Primary	ADA Activity in Erythrocytes	36 months	ADA enzyme activity was assessed as a measure of successful engraftment of genetically modified Hematopoietic stem progenitor cells (HSPCs), as it marks sustained gene expression from the normal ADA transgene.
Primary	Reduction in Deoxyadenosine Triphosphate (dATP) in Erythrocytes	36 months	Decreased dATP levels coincide with increased ADA enzyme activity, detoxification was used as a marker of correction of the defective ADA gene. The threshold for detoxification was \<100 μmol/L.

1 to 10 of 14Source ClinicalTrials.gov

See all 14 registered outcomes

Outcomes14

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
PRIMARY	Vector Copy Number (VCN) in Granulocytes Fraction (Neutrophils)	36 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
PRIMARY	VCN in Peripheral Blood Mononuclear Cells (PBMCs)	36 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
PRIMARY	VCN in CD3+ T Cells	36 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
PRIMARY	VCN in CD19+ B Cells	36 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
PRIMARY	Change From Baseline in CD3+ T Cell Counts (1 Year)	12 months	Historical control groups/arms are not included in the analysis population for this outcome measure as CD3+ T cell count data was not collected due to the historical nature of the population	10e9 cells/L	GEOMETRIC_MEAN
PRIMARY	Change From Baseline in CD3+ T Cell Counts (3 Years)	36 months	Historical control groups/arms are not included in the analysis population for this outcome measure as CD3+ T cell count data was not collected due to the historical nature of the population	10e9 cells/L	MEDIAN
PRIMARY	ADA Activity in Erythrocytes	36 months		nmol/h/mg	MEDIAN
PRIMARY	Reduction in Deoxyadenosine Triphosphate (dATP) in Erythrocytes	36 months		umol/L	MEDIAN

1 to 10 of 14Source ClinicalTrials.gov

See all 14 outcomes

Results by group49

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	OTL-101* On-Study Subjects	100	95% Confidence Interval 69.15 to 100	percentage of participants
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	OTL-101* On-Study and CUP Subjects	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	100	95% Confidence Interval 47.82 to 100	percentage of participants
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	HSCT Controls With MRD	100	95% Confidence Interval 71.51 to 100	percentage of participants
Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	All HSCT Controls	100	95% Confidence Interval 79.41 to 100	percentage of participants
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	OTL-101* On-Study Subjects	100	95% Confidence Interval 69.15 to 100	percentage of participants
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	OTL-101* On-Study and CUP Subjects	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	100	95% Confidence Interval 47.82 to 100	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 71.51 to 100	percentage of participants
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)	All HSCT Controls	100	95% Confidence Interval 79.41 to 100	percentage of participants

1 to 10 of 49Source ClinicalTrials.gov

See all 49 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Gene Therapy	20
Overall Study	STARTED	Historical Control Group	16
Overall Study	COMPLETED	Gene Therapy	19
Overall Study	COMPLETED	Historical Control Group	15
Overall Study	NOT COMPLETED	Gene Therapy	1
Overall Study	NOT COMPLETED	Historical Control Group	1

Source ClinicalTrials.gov

Adverse events310

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	Rhinitis	Infections and infestations	OTL-101* On-Study and CUP Subjects	9	20	15
other	Rhinitis	Infections and infestations	OTL-101* On-Study Subjects	5	10	9
other	Viral upper respiratory tract infection	Infections and infestations	OTL-101* On-Study and CUP Subjects	7	20	12
other	Viral upper respiratory tract infection	Infections and infestations	OTL-101* On-Study Subjects	4	10	7
other	Otitis media	Infections and infestations	OTL-101* On-Study and CUP Subjects	3	20	4
other	Otitis media	Infections and infestations	OTL-101* On-Study Subjects	2	10	2
other	Lower respiratory tract infection	Infections and infestations	OTL-101* On-Study and CUP Subjects	2	20	2
other	Lower respiratory tract infection	Infections and infestations	OTL-101* On-Study Subjects	2	10	2
other	Metapneumovirus infection	Infections and infestations	OTL-101* On-Study and CUP Subjects	2	20	2
other	Metapneumovirus infection	Infections and infestations	OTL-101* On-Study Subjects	2	10	2

1 to 10 of 310Source ClinicalTrials.gov

See all 310 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	OTL-101* On-Study and CUP Subjects	0	20
Total, all-cause mortality	OTL-101* On-Study Subjects	0	10
Total, other adverse events	OTL-101* On-Study and CUP Subjects	20	20
Total, other adverse events	OTL-101* On-Study Subjects	10	10
Total, serious adverse events	OTL-101* On-Study and CUP Subjects	11	20
Total, serious adverse events	OTL-101* On-Study Subjects	7	10

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
33974366	DERIVED

Source PubMed

Grants0

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.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
OTL-101* On-Study and CUP Subjects vs OTL-101* On-Study Subjects: deaths from any cause	Trial comparisons
OTL-101* On-Study and CUP Subjects vs OTL-101* On-Study Subjects: serious adverse events	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT01380990

GET https://conductscience.org/api/trials/v1/NCT01380990/results?page=2

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