Trial · NCT03619551
Conditioning SCID Infants Diagnosed Early
NCT03619551, Phase 2, Active not recruiting, Center for International Blood and Marrow Transplant Research
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Conditioning SCID Infants Diagnosed Early. Trials, record NCT03619551. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03619551
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Conditioning SCID Infants Diagnosed Early
- NCT number
- NCT03619551
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 56 participants, estimated
- Lead sponsor
- Center for International Blood and Marrow Transplant Research
- Conditions
- SCID
- Interventions
- Busulfan, Cell processing for TCRαβ+/CD19+ depletion
- First posted
- 2018-08-08
- Last updated
- 2026-10-26
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Vaccine specific antibody response | 2 years | Humoral immune reconstitution by 2 years post HCT, defined by specific antibody response to tetanus toxoid. Criteria for evaluation of humoral immune response are the following: * Donor T cell chimerism ≥50% * B cell count ≥50 cells/microliter * IVIG independent for ≥12 weeks Subjects meeting the criteria receive 3 doses of tetanus toxoid at least 4 weeks apart, followed by measurement of tetanus titer at least 4-6 weeks after the 3rd dose. Those who achieve tetanus titer of ≥0.15 IU/ml after vaccination will meet the primary endpoint. Patients who have documented humoral immune response at a time prior to 2 years will be considered a success for the primary endpoint, while patients who do not have humoral immune response evaluated by 2 years will be considered failures for the primary endpoint. |
| Secondary | Immune Reconstitution | Up to 3 years | * T cell immune reconstitution at 30 days, 60 days, 3 months, 6 months, 12 months, and 2 years post-HCT. * Naïve T cell generation and thymic output at 3 months, 6 months, 12 months and 2 years post-HCT. * Freedom from immunoglobulin substitution will be assessed on all patients at 9 months, 12 months, 2 years and 3 years post-HCT. Patients who have not received IVIG for at least 12 weeks at the time of assessment will be considered free from immunoglobulin substitution. * Tetanus responses on all patients who complete a trial of vaccination by additional timepoints of 12 months, 18 months and 3 years post-HCT. * Live vaccine responses on all patients who undergo trial of vaccination by 3 years post-HCT |
| Secondary | Engraftment | Neutrophil Engraftment: 42 days post-HCT. Donor cell chimerism up to 2 years post-HCT. | * Neutrophil engraftment will be assessed on all patients and defined as achieving an absolute neutrophil count of \>500 cells/microliter for 3 consecutive days by day 42 post-HCT * Donor cell chimerism (whole blood, sorted CD3 (T-cell), CD19 (B-cell) and CD56 (NK cell) and granulocyte (CD15)) at 42 days, 3 months, 6 months, 12 months, and 2 years post-HCT. Absolute B cell, NK cell and granulocyte counts will be measured. |
| Secondary | Overall Survival | 3 years | Data to track overall survival will be collected at 1, 2 and 3 years post-HCT. |
| Secondary | Event Free Survival | 3 years | Data to track event free survival will be collected at 1, 2 and 3 years post-HCT. Events will be defined as 1) death from any cause, 2) rejection of the graft (T-cell and/or whole blood chimerism \<5% donor), 3) graft failure necessitating a second HCT procedure from the same donor or a different donor, with or without conditioning, 4) DLI given for treatment of falling chimerism. |
| Secondary | Acute graft-versus-host disease (aGVHD) | day 100 and 6 months post HCT | Occurrence of acute (grade II-IV and grade III-IV) GVHD. |
| Secondary | Chronic graft-versus-host disease (cGVHD) | 2 years post HCT | Occurrence of chronic GVHD by 6 months, 12 months and 24 months post-HCT. |
| Secondary | Post-HCT Complications | Up to 2 years post-HCT | * Infections * Targeted regimen related toxicity (severe veno-occlusive disease of the liver, idiopathic pneumonitis syndrome) * Autoimmunity |
| Secondary | Busulfan Pharmacokinetics | Pre-HCT | Blood samples will be collected on busulfan dosing days 1 and 3 at 2.35, 4, 6 and 8 hours from the time of the start of the infusion. The results will be used to estimate individual exposure (AUC and cAUC). |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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/NCT03619551
GET https://conductscience.org/api/trials/v1/NCT03619551/registered