NCT03311503 Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
| NCT ID | NCT03311503 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | David Williams |
| Condition | Severe Combined Immunodeficiency, X Linked |
| Study Type | INTERVENTIONAL |
| Enrollment | 12 participants |
| Start Date | 2018-02-26 |
| Primary Completion | 2028-01-01 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 12 participants in total. It began in 2018-02-26 with a primary completion date of 2028-01-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized
Eligibility Criteria
Inclusion Criteria: \- 1. Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA \<10% of the lower limit of normal for the laboratory) AND confirmed by a mutation in IL2RG 2. Lack of an HLA identical (A, B, C, DR, DQ) related donor 3. Age 5 years old or younger 4. Signed informed consent 5. Documentation of willingness to follow up for 15 years post-infusion as currently required by the FDA 6. If the patient has previously undergone allogeneic transplant, lack of donor T cell engraftment must be documented. 7\. Age at least 8 weeks by the time of busulfan administration Exclusion Criteria: 1. Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite a minimum of 2 weeks of appropriate therapy and is associated with significant organ dysfunction (including but not limited to abnormalities listed below). 1. Mechanical ventilation including continuous positive airway pressure 2. Abnormal liver function defined by AST and ALT \>10 times the upper range of normal OR Bilirubin \>2 mg/dL 3. Shortening fraction on echocardiogram \<25% or ejection fraction \<50% 4. Renal failure defined as glomerular filtration rate \<30 ml/min/1.73 m2 or dialysis dependence 2. Uncontrolled seizure disorder 3. Encephalopathy 4. Documented coexistence of any disorder known to affect DNA repair 5. Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease 6. Patients with evidence of infection with HIV-1 7. 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 central nervous system malformations, other severe non-repairable malformations of the gastrointestinal or genitourinary tracts that significantly impair organ function. 8. 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 deterioriation 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 \-
Contact & Investigator
Sung-Yun Pai, MD
STUDY CHAIR
National Institutes of Health (NIH)
Frequently Asked Questions
Who can join the NCT03311503 clinical trial?
This trial is open to male participants only, aged 0 Years or older, up to 5 Years, studying Severe Combined Immunodeficiency, X Linked. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03311503 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT03311503 currently recruiting?
Yes, NCT03311503 is actively recruiting participants. Contact the research team at colleen.dansereau@childrens.harvard.edu for enrollment information.
Where is the NCT03311503 trial being conducted?
This trial is being conducted at Los Angeles, United States, Atlanta, United States, Boston, United States, Cincinnati, United States.
Who is sponsoring the NCT03311503 clinical trial?
NCT03311503 is sponsored by David Williams. The principal investigator is Sung-Yun Pai, MD at National Institutes of Health (NIH). The trial plans to enroll 12 participants.