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Conditioning SCID Infants Diagnosed Early

Study acronym: CSIDE
StatusActive, Not Recruiting
PhasePhase 2
Started2018-10-22
View on ClinicalTrials.gov ↗

Amendment history

2026-08-26
minor
Study Status, Contacts/Locations v27
Study sites41→40
2026-08-04
minor
Study Status, Contacts/Locations v26
Study sites42→41
2026-07-27
minor
Study Status, Contacts/Locations v25
Study sites43→42
2026-07-07
minor
Study Status, Contacts/Locations v24
Study sites44→43
2026-02-12
minor
Study Status, Contacts/Locations v23
Study sites45→44
Show 22 earlier versions
2026-01-30
minor
Study Status, Contacts/Locations v22
Study sites47→45
2026-01-27
minor
Study Status, Contacts/Locations v21
Study sites49→47
2025-11-18
notable
Recruiting→Active, Not Recruiting Study Status, Oversight, Contacts/Locations v20
Trial statusRecruiting→Active, Not Recruiting
Primary completion date2029-08-01→2027-12-01
Completion date2030-08-01→2028-12-01
Study sites52→49
2025-09-23
minor
Study Status, Contacts/Locations v19
Study sitesdetails revised at 3 of 52 sites
2025-04-22
minor
Study Identification, Study Status, Sponsor/Collaborators, Study Design, Contacts/Locations v18
Enrollment target64→56
Lead sponsorMichael Pulsipher→Center for International Blood and Marrow Transplant Research
Study sitesdetails revised at 51 of 52 sites
2025-03-20
minor
Study Status v17
Primary completion date2029-01-08→2029-08-01
Completion date2030-01-08→2030-08-01
2025-03-17
minor
Study Identification, Study Status, Sponsor/Collaborators, Oversight, Contacts/Locations v16
Primary completion date2026-08-01→2029-01-08
Completion date2026-08-01→2030-01-08
Lead sponsorMichael Pulsipher, MD→Michael Pulsipher
Study sitesdetails revised at 51 of 52 sites
2023-09-15
minor
Study Status, Contacts/Locations v15
Study sitesdetails revised at 1 of 52 sites
2023-09-08
minor
Study Status, Contacts/Locations v14
Study sites49→52
2023-09-05
minor
Study Status, Contacts/Locations v13
Study sites47→49
2022-12-02
minor
Study Status, Contacts/Locations v12
Study sites42→47
2022-03-02
minor
Study Status, Contacts/Locations v11
Study sitescontacts updated at 2 of 42 sites
2021-10-27
minor
Study Status, Contacts/Locations v10
Study sitescontacts updated at 1 of 42 sites
2021-10-14
minor
Study Status, Contacts/Locations v9
Study sites20→42
2021-09-13
minor
Study Status, Contacts/Locations v8
Study sitescontacts updated at 1 of 20 sites
2021-05-17
minor
Study Status, Contacts/Locations v7
Study sitescontacts updated at 1 of 20 sites
2021-02-25
minor
Study Status, Eligibility v6
Re-verified, no change to tracked fields
2019-12-27
minor
Study Status, Contacts/Locations v5
Study sites6→20
2019-06-06
minor
Study Status, Contacts/Locations v4
Study sites3→6
2018-12-28
notable
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v3
Trial statusNot Yet Recruiting→Recruiting
Start dateestimated 2018-10-15→confirmed 2018-10-22
Study sites2→3
2018-10-20
minor
Study Status, Contacts/Locations v2
Start date2018-09-01→2018-10-15
Study sitescontacts updated at 1 of 2 sites
2018-09-10
minor
Study Status, Eligibility v1
Start date2018-08-01→2018-09-01
2018-08-02
minor
Original filing
The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants. The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has. Donors used for transplant must be unrelated or half-matched related (haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T cells removed, using a newer, experimental approach of a well-established technology. Once the stem cell transplant is completed, patients will be followed for 3 years. Approximately 9-18 months after the transplant, vaccinations will be administered, and a blood test measuring whether your child's body has responded to the vaccine will be collected.
Trial Details
NCT Number NCT03619551
Lead Sponsor Center for International Blood and Marrow Transplant Research
Conditions SCID
Enrollment 56 participants
Start Date 2018-10-22
Primary Completion 2027-12-01 (estimated)
Study Completion 2028-12-01 (estimated)
Updated on ClinicalTrials.gov 2026-08-28