Severe Combined ImmunodeficiencyPhase 1/2NCT01129544
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
A Phase 1/2 study testing Gene transfer in people with Severe Combined Immunodeficiency, run by David Williams at 3 sites in United States.
Trial summary
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat transplants, incomplete cure of the immune system, exposure to chemotherapy, and graft… Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Severe Combined Immunodeficiency
- What is being tested?
- Gene transfer
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2010to Mar 2023
- 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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of SCID-X1 based on immunophenotype (<200 CD3+ autologous T cells, and confirmed by DNA sequencing)
- AND
- Lack an HLA identical (A, B, C, DR, DQ) related donor
- AND either one of the following:
- 1. Patients in good clinical condition who do not have a readily available HLA identical (A,B,C,DR,DQ) unrelated donor (readily available defined as: a donor confirmed within 6 weeks of searching, with ability to transplant within 3 months of diagnosis).
- 2. Patients with an active, therapy-resistant infection or other medical conditions that significantly increase the risk of allogeneic transplant. Examples of "therapy-resistant infections that significantly increase the risk of allogeneic transplant" include but are not limited to:
Show all 12Show fewer
- interstitial pneumonia due to adenovirus or parainfluenzae virus.
- protracted diarrhea requiring total parenteral nutrition.
- disseminated BCG infection.
- virus-induced lymphoproliferative disease.
- any active opportunistic infection (eg, due to Pneumocystis jiroveci, cytomegalovirus,cryptosporidium) that does not improve on medical management.
- active and progressive pulmonary disease requiring mechanic ventilation. Inclusion of patients with disease-related organ dysfunction is justified by the known poor outcome with standard treatment and the potential life-saving nature of the treatment proposed. Patients who are on high-dose steroids or other immunosuppressive agents will also be considered eligible, because use of these drugs is common in patients with SCID and maternal T cell engraftment or who present with severe interstitial lung disease. Use of immunosuppressive drugs does not affect efficacy of hematopoietic cell transplantation, and therefore should not affect efficacy of gene transfer.
Who cannot join
- No available molecular diagnosis confirming SCID-X1.
- Patients who have an available HLA-identical related donor.
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease
- Patients with evidence of infection with HIV-1
- Previous gene transfer
- Major (life-threatening) congenital anomalies. Examples of "major (life-threatening) congenital anomalies" include, but are not limited to: unrepaired cyanotic heart disease, hypoplastic lungs, anencephaly or other major CNS malformations, other severe non-repairable malformations of the gastrointestinal or genitourinary tracts that significantly impair organ function.
- Other conditions which in the opinion of the P.I. or co-investigators, contra-indicate collection and/or infusion of transduced cells or indicate patient's inability to follow the protocol. These may include for example clinical ineligibility to receive anesthesia, severe deterioration of clinical condition of the patient after collection of bone marrow but before infusion of transduced cells, or documented refusal or inability of the family to return for scheduled visits. There may be other unforeseen rare circumstances that would result in exclusion of the patient, such as sudden loss of legal guardianship.
- Although the presentation of the disease may be variable in type, the severity of the immunodeficiency is uniform. The gene transfer protocol will be instituted in the place of haploidentical transplant for those patients who do not have a matched family donor or in whom an unrelated donor transplant is not indicated for the reasons specified above. Apart from the gene transfer protocol, the patients will not undergo additional procedures that would not form part of an equivalent haploidentical transplantation regimen, and will not receive conditioning chemotherapy.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Children's Hospital Boston
Boston, Massachusetts, 02116
Cincinnati Children's Medical Center
Cincinnati, Ohio, 45229
Mattel Children's Hospital - UCLA
Los Angeles, California, 90095
About this study
Official title: Multi-site Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene Transfer
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat transplants, incomplete cure of the immune system, exposure to chemotherapy, and graft versus host disease.
The idea behind gene transfer is to replace the broken gene by putting a piece of genetic material (DNA) that has the normal gene into the child's cells. Gene transfer can only be done if we know which gene is missing or broken in the patient. For SCID-X1, gene transfer has been done in the laboratory and in two previous clinical trials by inserting the normal gene into stem cells from bone marrow. The bone marrow is the "factory" inside the bones that creates blood and immune cells. So fixing the gene in the bone marrow stem cells should fix the immune problem, without giving chemotherapy and without risk of graft versus host disease, because the child's own cells are used, rather than another person's. Out of the 20 subjects enrolled in the two previous trials, 18 are alive with better immune systems after gene transfer. Two of the surviving subjects received gene corrected cells over 10 years ago.
Gene transfer is still research for two reasons. One is that not enough children have been studied to tell if the procedure is consistently successful. Of the 20 children enrolled in the previous two trials, one child did not have correction of the immune system, and died of complications after undergoing stem cell transplant. The second important reason why gene transfer is research is that we are still learning about the side effects of gene transfer and how to do gene transfer safely. In the last two trials, 5 children have experienced a serious side effect. These children developed leukemia related to the gene transfer itself. Leukemia is a cancer of the white blood cells, a condition where a few white blood cells grow out of control. Of these children, 4 of the 5 have received chemotherapy (medication to treat cancer) and are currently in remission (no leukemia can be found by sensitive testing), whereas one died of gene transfer-related leukemia.
Read the detailed description
Severe combined immunodeficiencies (SCID) are a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function. They arise from a variety of molecular defects which affect lymphocyte development and function. The most common form of SCID is an X-linked form (SCID-X1) which accounts for 40-50% of all cases. SCID-X1 is caused by defects in the common cytokine receptor chain, which was originally identified as a component of the high affinity interleukin-2 receptor (IL-2RG), but is now known to be an essential component of the IL-4, -7, -9 -15, and -21 cytokine receptor complexes. Classic SCID-X1 has an extremely poor prognosis without treatment. Death usually occurs in the first year of life from infectious complications unless definitive treatment can be administered. Until the recent advent of somatic gene therapy, hematopoietic stem cell transplantation (HSCT) offered the only curative option for patients with any form of SCID. If a genotypically matched sibling donor is available, HSCT is a highly successful procedure. However a genotypically matched family donor is only available for approximately 30% of patients. For the remaining individuals, alternative donor transplants, principally from matched unrelated or haploidentical parental donors have been performed. These approaches are still problematic with toxicity from ablative therapy, graft-versus-host disease and incomplete lymphoid reconstitution. Recent gene transfer trials have documented the efficacy of gene transfer in this disease, albeit with toxicity related to insertional mutagenesis. A new generation of self-inactivating (SIN) vectors has been developed which lack all enhancer-promoter elements of the LTR U3 region and are also devoid of all gammaretroviral coding regions. A SIN vector expressing the IL-2RG gene, pSRS11.EFS.IL2RG.pre* has been developed and has shown a reduction in mutagenic potential compared to LTR configuration in non-clinical studies. The current study is a phase I/II trial of somatic gene therapy for patients with SCID-X1.
- Conditions
- Severe Combined Immunodeficiency
- Being tested
- Gene transfer
- Keywords
- Severe combined immunodeficiency, SCID, XSCID, SCID-X1
- Medical subject headings
- Severe Combined Immunodeficiency, X-Linked Combined Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene transferBiological
Three procedures: 1) Bone marrow harvest from the patient's posterior iliac crests. 2.) Chemotherapy conditioning with Busulfan 3)One time infusion of patient's transduced bone marrow cells.
| Study group | Role | What they receive |
|---|---|---|
| Gene Transfer | Experimental | open label single arm study Receives: Biological: Gene transfer |
What is being measured
The primary outcome is the main question the study is designed to answer.
CD3 Cell Count Post Infusion
Immunological reconstitution defined as absolute CD3 cells of >300/μl and PHA stimulation index >15 at 6 months post infusion
Measured: 6 Months Post Gene Transfer
Incidence of Life Threatening Adverse Reactions Related to the Gene Therapy Procedure
Follow up time for each of the 8 enrolled patients is as follows: Patient #1 -12 yrs, Patient#2 -11 yrs, Patient #6- 9 yrs, Patient #7- 8 yrs, Patient #8 - 4.5 yrs. Patient #3 \& #5 failed primary endpoint and had allo transplant, Patient #4 retreated with Gene therapy and is 6 years post 2nd infusion.
Measured: 4.5-12 years post infusion of the gene therapy product
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2010 (estimated)
- Study startApr 2010
- Primary completionMar 2023
- Study completionMar 2023
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 (2)Papers the sponsor has linked to this study.
- Other trials for Severe Combined ImmunodeficiencyStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- David Williams
- Sponsor type
- University, hospital or other
- Collaborators
- Boston Children's Hospital, Children's Hospital Medical Center, Cincinnati, University of California, Los Angeles
- Responsible party
- Sponsor-investigator, David Williams, Boston Children's Hospital
- Investigators
- Jennifer Whangbo, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01129544
- Sponsor's protocol ID
- IND14067
- Status last verified
- Mar 2026
- First submitted
- 21 May 2010
- Last updated
- 21 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z.
PubMed 30261899Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernandez KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A modified gamma-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588.
PubMed 25295500
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-site Phase I/II Trial Evaluating the Treatment of SCID-X1 Patients With Retrovirus-mediated Gene Transfer
- ClinicalTrials.gov ID
- NCT01129544
- Acronym / sponsor ID
- IND14067
- Conditions
- Severe Combined Immunodeficiency
- Keywords
- Severe combined immunodeficiency, SCID, XSCID, SCID-X1
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- David WilliamsUniversity, hospital or other
- Collaborators
- Boston Children's Hospital, Children's Hospital Medical Center, Cincinnati, University of California, Los Angeles
- Study start
- Apr 2010 (actual)
- Primary completion
- Mar 2023 (actual)
- Study completion
- Mar 2023 (actual)
- First posted
- 24 May 2010 (estimated)
- Results
- Posted 21 Apr 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat transplants, incomplete cure of the immune system, exposure to chemotherapy, and graft versus host disease.
The idea behind gene transfer is to replace the broken gene by putting a piece of genetic material (DNA) that has the normal gene into the child's cells. Gene transfer can only be done if we know which gene is missing or broken in the patient. For SCID-X1, gene transfer has been done in the laboratory and in two previous clinical trials by inserting the normal gene into stem cells from bone marrow. The bone marrow is the "factory" inside the bones that creates blood and immune cells. So fixing the gene in the bone marrow stem cells should fix the immune problem, without giving chemotherapy and without risk of graft versus host disease, because the child's own cells are used, rather than another person's. Out of the 20 subjects enrolled in the two previous trials, 18 are alive with better immune systems after gene transfer. Two of the surviving subjects received gene corrected cells over 10 years ago.
Gene transfer is still research for two reasons. One is that not enough children have been studied to tell if the procedure is consistently successful. Of the 20 children enrolled in the previous two trials, one child did not have correction of the immune system, and died of complications after undergoing stem cell transplant. The second important reason why gene transfer is research is that we are still learning about the side effects of gene transfer and how to do gene transfer safely. In the last two trials, 5 children have experienced a serious side effect. These children developed leukemia related to the gene transfer itself. Leukemia is a cancer of the white blood cells, a condition where a few white blood cells grow out of control. Of these children, 4 of the 5 have received chemotherapy (medication to treat cancer) and are currently in remission (no leukemia can be found by sensitive testing), whereas one died of gene transfer-related leukemia.
Detailed description
Severe combined immunodeficiencies (SCID) are a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function. They arise from a variety of molecular defects which affect lymphocyte development and function. The most common form of SCID is an X-linked form (SCID-X1) which accounts for 40-50% of all cases. SCID-X1 is caused by defects in the common cytokine receptor chain, which was originally identified as a component of the high affinity interleukin-2 receptor (IL-2RG), but is now known to be an essential component of the IL-4, -7, -9 -15, and -21 cytokine receptor complexes. Classic SCID-X1 has an extremely poor prognosis without treatment. Death usually occurs in the first year of life from infectious complications unless definitive treatment can be administered. Until the recent advent of somatic gene therapy, hematopoietic stem cell transplantation (HSCT) offered the only curative option for patients with any form of SCID. If a genotypically matched sibling donor is available, HSCT is a highly successful procedure. However a genotypically matched family donor is only available for approximately 30% of patients. For the remaining individuals, alternative donor transplants, principally from matched unrelated or haploidentical parental donors have been performed. These approaches are still problematic with toxicity from ablative therapy, graft-versus-host disease and incomplete lymphoid reconstitution. Recent gene transfer trials have documented the efficacy of gene transfer in this disease, albeit with toxicity related to insertional mutagenesis. A new generation of self-inactivating (SIN) vectors has been developed which lack all enhancer-promoter elements of the LTR U3 region and are also devoid of all gammaretroviral coding regions. A SIN vector expressing the IL-2RG gene, pSRS11.EFS.IL2RG.pre* has been developed and has shown a reduction in mutagenic potential compared to LTR configuration in non-clinical studies. The current study is a phase I/II trial of somatic gene therapy for patients with SCID-X1.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gene Transfer | Experimental | Biological: Gene transferopen label single arm study |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Diagnosis of SCID-X1 based on immunophenotype (<200 CD3+ autologous T cells, and confirmed by DNA sequencing)
- AND
- Lack an HLA identical (A, B, C, DR, DQ) related donor
- 1. Patients in good clinical condition who do not have a readily available HLA identical (A,B,C,DR,DQ) unrelated donor (readily available defined as: a donor confirmed within 6 weeks of searching, with ability to transplant within 3 months of diagnosis).
- 2. Patients with an active, therapy-resistant infection or other medical conditions that significantly increase the risk of allogeneic transplant. Examples of "therapy-resistant infections that significantly increase the risk of allogeneic transplant" include but are not limited to:
Key exclusion criteria (8 listed)
- No available molecular diagnosis confirming SCID-X1.
- Patients who have an available HLA-identical related donor.
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease
- Patients with evidence of infection with HIV-1
- Previous gene transfer
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 |
|---|---|---|
| Gene transferThree procedures: 1) Bone marrow harvest from the patient's posterior iliac crests. 2.) Chemotherapy conditioning with Busulfan 3)One time infusion of patient's transduced bone marrow cells. | Biological | Gene Transfer |
Primary outcome
CD3 Cell Count Post Infusion
Time frame: 6 Months Post Gene Transfer
Immunological reconstitution defined as absolute CD3 cells of >300/μl and PHA stimulation index >15 at 6 months post infusion
Incidence of Life Threatening Adverse Reactions Related to the Gene Therapy Procedure
Time frame: 4.5-12 years post infusion of the gene therapy product
Follow up time for each of the 8 enrolled patients is as follows: Patient #1 -12 yrs, Patient#2 -11 yrs, Patient #6- 9 yrs, Patient #7- 8 yrs, Patient #8 - 4.5 yrs. Patient #3 \& #5 failed primary endpoint and had allo transplant, Patient #4 retreated with Gene therapy and is 6 years post 2nd infusion.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30261899Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z.
- Derived from this study: PubMed 25295500Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernandez KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A modified gamma-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588.
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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.
Jennifer Whangbo, MD principal investigator
Boston Children's Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Gene transfer in people with Severe Combined Immunodeficiency, run by David Williams at 3 sites in United States.
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 12 inclusion and 8 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 21 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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 21 Apr 2026). 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?
CD3 Cell Count Post Infusion (time frame: 6 Months Post Gene Transfer). The study lists 2 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 NCT01129544, which also lists 2 linked publications.
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 21 Apr 2026.