Trial · NCT03513328
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
NCT03513328, Phase 1, Phase 2, Completed, University of Florida; results first posted 2023-09-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation. Trials, record NCT03513328. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03513328
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
- NCT number
- NCT03513328
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- University of Florida
- Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
- Interventions
- Thiotepa--single daily dose, Thiotepa--escalated dose
- First posted
- 2018-05-01
- Last updated
- 2023-09-15
- Results first posted
- 2023-09-15
- Limitations and caveats
- Due to change in clinical practice, only patients with HLA matched donors were enrolled in this trial. Study was closed prematurely due to lack of enrollment.
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Assessment of Minimum Effective Dose (MED) of Thiotepa | Day 42 | Assess the MED of thiotepa in combination with reduced-dose busulfan, fludarabine and rATG required to achieve engraftment in \>90% subjects undergoing hematopoietic stem cell transplantation for non-malignant disorders. |
| Secondary | Percentage of Subjects With Graft Rejection/Failure. | Day 42; Day 365 | Percentage of all subjects who initiated conditioning regimen and have sustained engraftment failure. |
| Secondary | Percentage of Subjects Without Disease Recurrence Who Are Alive at 24 Months Post Transplant | Month 24 | Percentage of subjects who initiated conditioning regimen and are without evidence of underlying disease (DFS). |
| Secondary | Percentage of Subjects Alive at 24 Months Post Transplant (OS) | Month 24 | Percentage of subjects who initiated conditioning regimen and are alive at 24 months post transplant (OS). |
| Secondary | Evaluation of Transplant-related Mortality | Month 12 | Percentage of subjects who initiated conditioning regimen and who died due to a cause unrelated to the underlying disease. |
| Secondary | Number of Participants With Grade 2-4 Acute Graft-versus-host Disease (GVDH) | Month 12 | Graft-versus host disease symptoms measured using Modified Glucksberg Staging Criteria. (Scale 0-4; with 4 being most severe) |
| Secondary | Percentage of Participants With Chronic Graft-versus-host Disease (cGVHD) | Month 24 | Measures the frequency of chronic graft-vs-host disease in Group A participants |
| Secondary | Percentage of Participants With Transplant-related Complications | 24 months | Complications gathered via CIBMTR (Center for International Blood \& Marrow Transplant Research) post-transplant form was tabulated and described by treatment received. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Assessment of Minimum Effective Dose (MED) of Thiotepa | Day 42 | MED dose of thiotepa in group A was determined to be 5 mg/kg in combination with Fludarabine and rATG. All six patients engrafted by day 42 using this dose. No subsequent escalation was done. | mg/kg | NUMBER |
| SECONDARY | Percentage of Subjects With Graft Rejection/Failure. | Day 42; Day 365 | Risk of rejection was determined in Group A. None of the patients rejected the graft = 0% (95% CI 0-46%) either at Day 42 or Day 365 | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Subjects Without Disease Recurrence Who Are Alive at 24 Months Post Transplant | Month 24 | At 24 month OS and DFS was 100% (95% CI 54-100%) in group A, the OS and DFS could not have been evaluated for group B due to lack of enrollment | Percentage of patients | NUMBER |
| SECONDARY | Percentage of Subjects Alive at 24 Months Post Transplant (OS) | Month 24 | At 24 month OS and DFS was 100% (95% CI 54-100%) in group A, the OS and DFS could not have been evaluated for group B due to lack of enrollment | Percentage of patients | NUMBER |
| SECONDARY | Evaluation of Transplant-related Mortality | Month 12 | Transplant related mortality at 12 months post transplant was 0% (95% CI 0-46%) in Group A. Transplant-related mortality could not have been evaluated for group B due to lack of enrollment | Percentage of patients | NUMBER |
| SECONDARY | Number of Participants With Grade 2-4 Acute Graft-versus-host Disease (GVDH) | Month 12 | 1 of 6 patients developed GVHD grade 2-4. | Number of patients | NUMBER |
| SECONDARY | Percentage of Participants With Chronic Graft-versus-host Disease (cGVHD) | Month 24 | cGVHD in 2 of 6 participants | Percentage of patients | NUMBER |
| SECONDARY | Percentage of Participants With Transplant-related Complications | 24 months | Percentages of patient at risk for complications during first 2 years post transplant | Percentage of patients | NUMBER |
Source ClinicalTrials.gov
Results by group15
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Assessment of Minimum Effective Dose (MED) of Thiotepa | Group A--Thiotepa Single Dose | 5 | mg/kg | ||
| Percentage of Subjects With Graft Rejection/Failure. | Group A--Thiotepa Single Dose | 0 | Percentage of participants | ||
| Percentage of Subjects Without Disease Recurrence Who Are Alive at 24 Months Post Transplant | Group A--Thiotepa Single Dose | 100 | 95% Confidence Interval 54 to 100 | Percentage of patients | |
| Percentage of Subjects Alive at 24 Months Post Transplant (OS) | Group A--Thiotepa Single Dose | 100 | 95% Confidence Interval 54 to 100 | Percentage of patients | |
| Evaluation of Transplant-related Mortality | Group A--Thiotepa Single Dose | 0 | 95% Confidence Interval 0 to 46 | Percentage of patients | |
| Number of Participants With Grade 2-4 Acute Graft-versus-host Disease (GVDH) | Group A--Thiotepa Single Dose | 1 | Number of patients | ||
| Percentage of Participants With Chronic Graft-versus-host Disease (cGVHD) | Group A--Thiotepa Single Dose | 33 | 95% Confidence Interval 0.4 to 78 | Percentage of patients | |
| Percentage of Participants With Transplant-related Complications | Group A--Thiotepa Single Dose | Mucositis - 1st year post transplant | 100 | 95% Confidence Interval 54 to 100 | Percentage of patients |
| Percentage of Participants With Transplant-related Complications | Group A--Thiotepa Single Dose | Viral reactivations - first year post transplant | 67 | 95% Confidence Interval 22 to 96 | Percentage of patients |
| Percentage of Participants With Transplant-related Complications | Group A--Thiotepa Single Dose | Covid 19 Infection first year post transplant | 17 | 95% Confidence Interval 0 to 64 | Percentage of patients |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Group A--Thiotepa Single Dose | 6 | |
| Overall Study | STARTED | Group A--Thiotepa Escalated Dose | 0 | |
| Overall Study | STARTED | Group B--Thiotepa Single Dose | 0 | |
| Overall Study | STARTED | Group B--Thiotepa Escalated Dose | 0 | |
| Overall Study | COMPLETED | Group A--Thiotepa Single Dose | 6 | |
| Overall Study | COMPLETED | Group A--Thiotepa Escalated Dose | 0 | |
| Overall Study | COMPLETED | Group B--Thiotepa Single Dose | 0 | |
| Overall Study | COMPLETED | Group B--Thiotepa Escalated Dose | 0 | |
| Overall Study | NOT COMPLETED | Group A--Thiotepa Single Dose | 0 | |
| Overall Study | NOT COMPLETED | Group A--Thiotepa Escalated Dose | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events36
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Mucositis | Gastrointestinal disorders | Group A--Thiotepa Single Dose | 3 | 6 | 3 |
| other | Mucositis | Gastrointestinal disorders | Group A--Thiotepa Escalated Dose | 0 | 0 | 0 |
| other | Mucositis | Gastrointestinal disorders | Group B--Thiotepa Single Dose | 0 | 0 | 0 |
| other | Mucositis | Gastrointestinal disorders | Group B--Thiotepa Escalated Dose | 0 | 0 | 0 |
| other | Salmonella Bacteremia | Infections and infestations | Group A--Thiotepa Single Dose | 1 | 6 | 1 |
| other | Salmonella Bacteremia | Infections and infestations | Group A--Thiotepa Escalated Dose | 0 | 0 | 0 |
| other | Salmonella Bacteremia | Infections and infestations | Group B--Thiotepa Single Dose | 0 | 0 | 0 |
| other | Salmonella Bacteremia | Infections and infestations | Group B--Thiotepa Escalated Dose | 0 | 0 | 0 |
| other | Coag Negative Staph Bacteremia | Infections and infestations | Group A--Thiotepa Single Dose | 1 | 6 | 1 |
| other | Coag Negative Staph Bacteremia | Infections and infestations | Group A--Thiotepa Escalated Dose | 0 | 0 | 0 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Group A--Thiotepa Single Dose | 0 | 6 |
| Total, all-cause mortality | Group A--Thiotepa Escalated Dose | 0 | 0 |
| Total, all-cause mortality | Group B--Thiotepa Single Dose | 0 | 0 |
| Total, all-cause mortality | Group B--Thiotepa Escalated Dose | 0 | 0 |
| Total, other adverse events | Group A--Thiotepa Single Dose | 6 | 6 |
| Total, other adverse events | Group A--Thiotepa Escalated Dose | 0 | 0 |
| Total, other adverse events | Group B--Thiotepa Single Dose | 0 | 0 |
| Total, other adverse events | Group B--Thiotepa Escalated Dose | 0 | 0 |
| Total, serious adverse events | Group A--Thiotepa Single Dose | 0 | 6 |
| Total, serious adverse events | Group A--Thiotepa Escalated Dose | 0 | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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/NCT03513328
GET https://conductscience.org/api/trials/v1/NCT03513328/events?page=2