Adenosine Deaminase DeficiencyPhase 1/2NCT01380990

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

A Phase 1/2 study testing Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT) and Busulfan in people with Adenosine Deaminase Deficiency and Severe Combined Immunodeficiencies (SCID), run by Great Ormond Street Hospital for Children NHS Foundation Trust at 1 site in United Kingdom.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages Up to 15 years
Conditions the study is for: Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
What is being tested?: Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT) and Busulfan
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 36 participants
Where: 1 siteUnited Kingdom
Study dates: 15 Nov 2012to 23 Dec 2019
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Up to 15 years
All sexes
No healthy volunteers
Child

Who may be able to join

Diagnosis of ADA-SCID confirmed by DNA sequencing or by confirmed absence of <3% of ADA enzymatic activity in peripheral blood (or for neonates) in umbilical cord blood erythrocytes and/or leukocytes or in cultured fetal cells derived from either chorionic villus biopsy or amniocentesis, prior to institution of Polyethylene glycol-modified ADA (PEG-ADA) replacement therapy
Patients who lack a fully Human leukocyte antigen (HLA)-matched family donor
Patients (male or female) <5 years of age OR Patients (male or female) ≥ 5 years to 15 years of age who have preserved thymic function as evidenced by presence of >10 % naïve T cells (CD4+45RA+27+ cells)
Parental/guardian signed informed consent
Diagnosis of ADA-SCID confirmed by DNA sequencing OR by confirmed absence of <3% of ADA enzymatic activity in peripheral blood or (for neonates) in umbilical cord blood erythrocytes and/or leucocytes or in cultured foetal cells derived from either chorionic villus biopsy or amniocentesis, prior to institution of PEG-ADA replacement therapy
Patients (male or female) between 0-18 years at time of treatment
Patient treated with allogeneic haematopoietic stem cell transplantation since 2000

Who cannot join

Cytogenetic abnormalities on peripheral blood
Evidence of active malignant disease
Known sensitivity to busulfan
If applicable, confirmed pregnancy (to be tested in patients above 12 years old)
Gene Therapy (CUP)
A group of patients were treated under CUP (GOSH special license) either because the study was not yet open and patients needed urgent treatment, or because they were outside of the inclusion/exclusion criteria or received Investigational Medicinal Product (IMP) followed a different process (ie, received in two infusions). Patients followed the same protocol steps and study visits.
Historical Control Group

Trial locations and contacts

1 site in 1 country.

United Kingdom1 site

Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH

Other trials in United Kingdom

About this study

Official title: Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals

This is a historically controlled, non-randomized Phase I/II clinical trial to assess the safety and efficacy of autologous transplantation of CD34+ hematopoietic stem/progenitor cells (HSPCs), obtained from infants affected by ADA-SCID, following transduction of the HSPCs with a lentiviral vector (LV) carrying the human ADA complementary DNA (cDNA) under the control of the elongation factor 1 alpha shortened (EFS) promoter. Subjects treated in the trial receive the infusion of autologous, transduced cells following marrow cytoreduction with busulfan. The outcomes are compared to those observed in a historical control group of patients who received an allogeneic hematopoietic stem cell transplant (HSCT).

This Phase I/II clinical trial will be performed at Great Ormond Street Hospital (GOSH), London, United Kingdom.

Conditions: Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
Being tested: Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), Busulfan, Peg-Ada
Keywords: Gene therapy, Hematopoietic stem and progenitor cells, Lentiviral vector, ADA-SCID
Medical subject headings: Severe combined immunodeficiency due to adenosine deaminase deficiency, Severe Combined Immunodeficiency

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Infusion of autologous EFS-ADA LV CD34+ cellsGenetic
Also called OTL-101*
Autologous EFS-ADA LV CD34+ cells (OTL-101*) are infused intravenously
Haematopoietic Stem Cell Transplantation (HSCT)Other
Historical data from a database of ADA-SCID patients treated with allogeneic HSCT from GOSH will be collected as comparator group.
BusulfanDrug
Busulfan is used for non-myeloablative conditioning
Peg-AdaDrug
Peg-Ada enzyme replacement therapy is discontinued at Day +3- (-3/+15 days) after successful engraftment

Study groups in NCT01380990
Study group	Role	What they receive
Gene Therapy	Experimental	Infusion of autologous EFS-ADA LV CD34+ cells Receives: Genetic: Infusion of autologous EFS-ADA LV CD34+ cells; Drug: Busulfan; Drug: Peg-Ada
Historical Control Group	Other	Historical data from ADA-SCID patients who were treated with Hematopoietic Stem Cell Transplantation (HSCT) Receives: Other: Haematopoietic Stem Cell Transplantation (HSCT)

What is being measured

The primary outcome is the main question the study is designed to answer.

Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with OTL-101* or HSCT
Measured: 12 months
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
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.
Measured: 12 months
Vector Copy Number (VCN) in Granulocytes Fraction (Neutrophils)
Engraftment of transduced cells was assessed using vector gene marking in granulocytes (neutrophils)
Measured: 36 months
VCN in Peripheral Blood Mononuclear Cells (PBMCs)
Engraftment of transduced cells was assessed using vector gene marking in PBMCs
Measured: 36 months
VCN in CD3+ T Cells
Engraftment of transduced cells was assessed using vector gene marking
Measured: 36 months
VCN in CD19+ B Cells
Engraftment of transduced cells was assessed using vector gene marking in CD19+ B Cells
Measured: 36 months
Change From Baseline in CD3+ T Cell Counts (1 Year)
Immune reconstitution was assessed by change in CD3+ T Cell counts over time.
Measured: 12 months
Change From Baseline in CD3+ T Cell Counts (3 Years)
Immune reconstitution was assessed by change in CD3+ T Cell counts over time.
Measured: 36 months
ADA Activity in Erythrocytes
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.
Measured: 36 months
Reduction in Deoxyadenosine Triphosphate (dATP) in Erythrocytes
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.
Measured: 36 months
Frequency of Vector Integration Into Known Protooncogenes (3 Years)
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.
There were no instances of clonal proliferation in the course of the 36 month follow-up for on-study and CUP subjects; hence, a detailed analysis of the frequency of clonal expansion associated with vector integration near proto-oncogenes was not generated.
Measured: 36 months

Secondary outcomes (3)

OS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)
Overall survival (OS) is defined as the percentage of subjects alive at 36 months post- treatment with OTL-101* or HSCT
Measured: 36 months
EvFS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)
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.
Measured: 36 months
Infection Rate
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.
Measured: 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

15 Nov 201223 Dec 2019

First posted27 Jun 2011 (estimated)
Study start15 Nov 2012
Primary completion23 Dec 2019
Study completion23 Dec 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Adenosine Deaminase DeficiencyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 36 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01380990
Sponsor's protocol ID: 10-MI-29
Status last verified: Aug 2021
First submitted: 23 Jun 2011
Last updated: 16 Sep 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No

Publications

Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11.
PubMed 33974366

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruiting
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Active, not recruitingPhase 1/2
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
Adenosine Deaminase Severe Combined Immune Deficiency
RecruitingObservational study
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
Enrolling by invitationObservational study
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Immunologic Deficiency Syndromes

All Adenosine Deaminase Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 16 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals
ClinicalTrials.gov ID: NCT01380990
Acronym / sponsor ID: 10-MI-29
Conditions: Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
Keywords: Gene therapy, Hematopoietic stem and progenitor cells, Lentiviral vector, ADA-SCID
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 36 participants (actual)
Lead sponsor: Great Ormond Street Hospital for Children NHS Foundation TrustUniversity, hospital or other
Collaborators: Orchard Therapeutics
Study start: 15 Nov 2012 (actual)
Primary completion: 23 Dec 2019 (actual)
Study completion: 23 Dec 2019 (actual)
First posted: 27 Jun 2011 (estimated)
Results: Posted 16 Sep 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This Phase I/II clinical trial will be performed at Great Ormond Street Hospital (GOSH), London, United Kingdom.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Gene Therapy	Experimental	Genetic: Infusion of autologous EFS-ADA LV CD34+ cells, Drug: Busulfan, Drug: Peg-AdaInfusion of autologous EFS-ADA LV CD34+ cells
Historical Control Group	Other	Other: Haematopoietic Stem Cell Transplantation (HSCT)Historical data from ADA-SCID patients who were treated with Hematopoietic Stem Cell Transplantation (HSCT)

Who may qualify

Ages: Up to 15 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (7 listed)

Diagnosis of ADA-SCID confirmed by DNA sequencing or by confirmed absence of <3% of ADA enzymatic activity in peripheral blood (or for neonates) in umbilical cord blood erythrocytes and/or leukocytes or in cultured fetal cells derived from either chorionic villus biopsy or amniocentesis, prior to institution of Polyethylene glycol-modified ADA (PEG-ADA) replacement therapy
Patients who lack a fully Human leukocyte antigen (HLA)-matched family donor
Patients (male or female) <5 years of age OR Patients (male or female) ≥ 5 years to 15 years of age who have preserved thymic function as evidenced by presence of >10 % naïve T cells (CD4+45RA+27+ cells)
Parental/guardian signed informed consent
Diagnosis of ADA-SCID confirmed by DNA sequencing OR by confirmed absence of <3% of ADA enzymatic activity in peripheral blood or (for neonates) in umbilical cord blood erythrocytes and/or leucocytes or in cultured foetal cells derived from either chorionic villus biopsy or amniocentesis, prior to institution of PEG-ADA replacement therapy

Key exclusion criteria (7 listed)

Cytogenetic abnormalities on peripheral blood
Evidence of active malignant disease
Known sensitivity to busulfan
If applicable, confirmed pregnancy (to be tested in patients above 12 years old)
Gene Therapy (CUP)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Infusion of autologous EFS-ADA LV CD34+ cellsAlso: OTL-101Autologous EFS-ADA LV CD34+ cells (OTL-101) are infused intravenously	Genetic	Gene Therapy
Haematopoietic Stem Cell Transplantation (HSCT)Historical data from a database of ADA-SCID patients treated with allogeneic HSCT from GOSH will be collected as comparator group.	Other	Historical Control Group
BusulfanBusulfan is used for non-myeloablative conditioning	Drug	Gene Therapy
Peg-AdaPeg-Ada enzyme replacement therapy is discontinued at Day +3- (-3/+15 days) after successful engraftment	Drug	Gene Therapy

Primary outcome

Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
Time frame: 12 months
Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with OTL-101* or HSCT
Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
Time frame: 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.
Vector Copy Number (VCN) in Granulocytes Fraction (Neutrophils)
Time frame: 36 months
Engraftment of transduced cells was assessed using vector gene marking in granulocytes (neutrophils)
VCN in Peripheral Blood Mononuclear Cells (PBMCs)
Time frame: 36 months
Engraftment of transduced cells was assessed using vector gene marking in PBMCs
VCN in CD3+ T Cells
Time frame: 36 months
Engraftment of transduced cells was assessed using vector gene marking
VCN in CD19+ B Cells
Time frame: 36 months
Engraftment of transduced cells was assessed using vector gene marking in CD19+ B Cells
Change From Baseline in CD3+ T Cell Counts (1 Year)
Time frame: 12 months
Immune reconstitution was assessed by change in CD3+ T Cell counts over time.
Change From Baseline in CD3+ T Cell Counts (3 Years)
Time frame: 36 months
Immune reconstitution was assessed by change in CD3+ T Cell counts over time.
ADA Activity in Erythrocytes
Time frame: 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.
Reduction in Deoxyadenosine Triphosphate (dATP) in Erythrocytes
Time frame: 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.
Frequency of Vector Integration Into Known Protooncogenes (3 Years)
Time frame: 36 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. There were no instances of clonal proliferation in the course of the 36 month follow-up for on-study and CUP subjects; hence, a detailed analysis of the frequency of clonal expansion associated with vector integration near proto-oncogenes was not generated.

3 secondary outcomes

OS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)
Time frame: 36 months
EvFS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)
Time frame: 36 months
Infection Rate
Time frame: 36 months

Results and safety

Results posted to the registry on 16 Sep 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Gene Therapy	20	19
Historical Control Group	16	15

Primary outcome: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

Time frame: 12 months. Number (95% Confidence Interval). Unit: percentage of participants

Measure	OTL-101* On-Study Subjects n = 10	OTL-101* On-Study and CUP Subjects n = 20	HSCT Controls Without MRD n = 5	HSCT Controls With MRD n = 11	All HSCT Controls n = 16
Value	100 (69.15 to 100)	100 (83.16 to 100)	100 (47.82 to 100)	100 (71.51 to 100)	100 (79.41 to 100)

OTL-101* On-Study Subjects vs HSCT Controls Without MRD: Difference in percentages: 0
OTL-101* On-Study Subjects vs HSCT Controls With MRD: Difference in percentages: 0
OTL-101* On-Study Subjects vs All HSCT Controls: Difference in percentages: 0

Primary outcome: Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

Time frame: 12 months. Number (95% Confidence Interval). Unit: percentage of participants

Measure	OTL-101* On-Study Subjects n = 10	OTL-101* On-Study and CUP Subjects n = 20	HSCT Controls Without MRD n = 5	HSCT Controls With MRD n = 11	All HSCT Controls n = 16
Value	100 (69.15 to 100)	100 (83.16 to 100)	100 (47.82 to 100)	100 (71.51 to 100)	100 (79.41 to 100)

OTL-101* On-Study Subjects vs HSCT Controls Without MRD: Difference in percentages: 0
OTL-101* On-Study Subjects vs HSCT Controls With MRD: Difference in percentages: 0
OTL-101* On-Study Subjects vs All HSCT Controls: Difference in percentages: 0

Primary outcome: Vector Copy Number (VCN) in Granulocytes Fraction (Neutrophils)

Time frame: 36 months. Median (Full Range). Unit: copies/cell

Measure	OTL-101* On-Study Subjects n = 9	OTL-101* On-Study and CUP Subjects n = 18
Value	0.240 (0.15 to 0.79)	0.280 (0.03 to 1.60)

Population analysed: 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

Primary outcome: VCN in Peripheral Blood Mononuclear Cells (PBMCs)

Time frame: 36 months. Median (Full Range). Unit: copies/cell

Measure	OTL-101* On-Study Subjects n = 9	OTL-101* On-Study and CUP Subjects n = 18
Value	0.600 (0.20 to 1.67)	0.625 (0.20 to 1.67)

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were reported from the time of participant consent, approximately 1-month pre-treatment, up until 3-years post-treatment with OTL-101*. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
OTL-101* On-Study and CUP Subjects	11 of 20 (55%)	20 of 20 (100%)	0 of 20
OTL-101* On-Study Subjects	7 of 10 (70%)	10 of 10 (100%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 33974366Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11.

Related trials for Adenosine Deaminase Deficiency

Not yet recruiting
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
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Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Claire Booth, Dr principal investigator

Great Ormond Street Hospital NHS Foundation Trust

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Sep 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) (time frame: 12 months). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01380990, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2021.