Trial · NCT02512679
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
NCT02512679, Phase 2, Terminated, Children's Hospital Los Angeles; results first posted 2016-06-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells. Trials, record NCT02512679. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02512679
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
- NCT number
- NCT02512679
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Children's Hospital Los Angeles
- Conditions
Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation, Genetic Diseases, Thalassemia, Pediatrics, Diamond-Blackfan Anemia, Combined Immune Deficiency, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Lymphoproliferative Disease, Metabolic Diseases
- Interventions
- Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3, Cyclophosphamide Dose Level 4
- First posted
- 2015-07-31
- Last updated
- 2017-02-27
- Results first posted
- 2016-06-17
- Limitations and caveats
- De-escalation was not ensued. All enrolled subjects engrafted without toxicity for dose level one.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Neutrophil Engraftment (=/>500 Cells/uL) and Platelet Engraftment (>20K Cell/uL) at 30 Days | 30 days | Absolute Neutrophil Count (ANC) =/\>500;(recovery of white cell count - self sustain platelet above 20,000 per cubic milimeter (20K) - evaluation by Chimerism Study (STR or FISH) at day +30 |
| Primary | Number of Participants With Disease Recurrence at 1 Year Post-transplant | 1 year | assess rate of disease recurrence ("late relapse") due to autologous recovery of recipient hematopoiesis at one year post-HSCT. |
| Primary | Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant | 1 year | Assessment of conditioning regimen related toxicity was evaluated and documented with daily assessment during hospitalization and post-transplant follow-up up to one year. None of the subjects developed VOD necessitating any therapeutic intervention, severe mucositis, or toxicity of the Kidney, Liver or Gastrointestinal. |
| Secondary | Number of Participants Who Developed Graft-Versus-Host-Disease (GVHD) as Determined by the Glucksberg Scale | 1 yr | Clinical evaluation on a daily basis during hospitalization and at each post transplant clinical visit, up to one year, to determine incidence of acute and chronic graft-versus-host disease using Glucksberg grading scale. Acute graft-versus-host disease (aGVHD) develops within the first three months after transplantation and appears as a skin rash, often accompanied by hyperbilirubenemia, abnormal liver enzymes and gastrointestinal symptoms, as diarrhea, nausea and vomiting. Level of aGVHD is graded from 1-4. Chronic GVHD, typically a late complication of Blood and Marrow Transplantation (BMT) characterized by skin changes, sometimes sclerotic changes, with joint contractures, liver function abnormality, gastrointestinal symptoms and sometime other organ involvement such as eyes, lungs, and obliterative bronchiolitis (OB). Chronic GVHD is graded as absent, limited, or extensive. |
| Secondary | Number of Participants Who Were Disease Progression-Free and Death-Free at 1 Year Post-transplant | 1 yr | Evaluation for engraftment, correction of the disease, transplant related complications and event-free survival and overall survival of the subjects post-transplant was undertaken by standard measures and evaluation of disease with disease-specific testing. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Neutrophil Engraftment (=/>500 Cells/uL) and Platelet Engraftment (>20K Cell/uL) at 30 Days | 30 days | Subject enrolled and received Dose Level 1 of Cyclophosphamide and engrafted with Absolute Neutrophil Count (ANC) =/\>500;(recovery of white cell count - self sustain platelet above 20k - evaluation by Chimerism Study (STR or FISH) at day +30. | participants | NUMBER |
| PRIMARY | Number of Participants With Disease Recurrence at 1 Year Post-transplant | 1 year | All subjects who received dose level 1 of Cyclophosphamide did not experience re-occurrence of disease. | participants | NUMBER |
| PRIMARY | Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant | 1 year | Subjects who received dose level 1 of Cyclophosphamide did not experience veno-occlusive disease, organ failure or severe mucositis. | participants | NUMBER |
| SECONDARY | Number of Participants Who Developed Graft-Versus-Host-Disease (GVHD) as Determined by the Glucksberg Scale | 1 yr | Subjects who received dose level 1 of Cyclophosphamide were revaluated for graft-versus-host disease (GVHD) post transplantation. | participants | NUMBER |
| SECONDARY | Number of Participants Who Were Disease Progression-Free and Death-Free at 1 Year Post-transplant | 1 yr | Subjects receiving dose level 1 of Cyclophosphamide event free survival post transplant at 100 days was 95% and 90% 1 year. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Neutrophil Engraftment (=/>500 Cells/uL) and Platelet Engraftment (>20K Cell/uL) at 30 Days | Cyclophosphamide Dose Level 1 | 20 | participants | ||
| Number of Participants With Disease Recurrence at 1 Year Post-transplant | Cyclophosphamide Dose Level 1 | 0 | participants | ||
| Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant | Cyclophosphamide Dose Level 1 | Veno-Occlusive Disease | 0 | participants | |
| Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant | Cyclophosphamide Dose Level 1 | Viral Infection | 8 | participants | |
| Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant | Cyclophosphamide Dose Level 1 | Toxicity of Kidney, Liver, or Gastrointestinal | 0 | participants | |
| Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant | Cyclophosphamide Dose Level 1 | Severe Mucositis | 0 | participants | |
| Number of Participants Who Developed Graft-Versus-Host-Disease (GVHD) as Determined by the Glucksberg Scale | Cyclophosphamide Dose Level 1 | Acute GVHD (Grade 1-2) | 10 | participants | |
| Number of Participants Who Developed Graft-Versus-Host-Disease (GVHD) as Determined by the Glucksberg Scale | Cyclophosphamide Dose Level 1 | Acute GVHD (Grade 3-4) | 0 | participants | |
| Number of Participants Who Developed Graft-Versus-Host-Disease (GVHD) as Determined by the Glucksberg Scale | Cyclophosphamide Dose Level 1 | Chronic GVHD | 0 | participants | |
| Number of Participants Who Were Disease Progression-Free and Death-Free at 1 Year Post-transplant | Cyclophosphamide Dose Level 1 | 100 Day Event Free Survival Post Transplant | 19 | participants |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cyclophosphamide Dose Level 1 | 20 | |
| Overall Study | STARTED | Cyclophosphamide Dose Level 2 | 0 | |
| Overall Study | COMPLETED | Cyclophosphamide Dose Level 1 | 20 | |
| Overall Study | COMPLETED | Cyclophosphamide Dose Level 2 | 0 | |
| Overall Study | NOT COMPLETED | Cyclophosphamide Dose Level 1 | 0 | |
| Overall Study | NOT COMPLETED | Cyclophosphamide Dose Level 2 | 0 |
Source ClinicalTrials.gov
Adverse events5
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 | Cytomegalovirus (CMV) infection | Blood and lymphatic system disorders | Cyclophosphamide Dose Level 1 | 5 | 20 | 5 |
| other | Epstein-Barr Virus (EBV) | Blood and lymphatic system disorders | Cyclophosphamide Dose Level 1 | 1 | 20 | 1 |
| other | Adenovirus | Blood and lymphatic system disorders | Cyclophosphamide Dose Level 1 | 1 | 20 | 1 |
| serious | Death due to pre-exisiting mucormycosis | Respiratory, thoracic and mediastinal disorders | Cyclophosphamide Dose Level 1 | 1 | 20 | 1 |
| serious | Death due to interstitial pneumonia | Respiratory, thoracic and mediastinal disorders | Cyclophosphamide Dose Level 1 | 1 | 20 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cyclophosphamide Dose Level 1 | ||
| Total, other adverse events | Cyclophosphamide Dose Level 1 | 7 | 20 |
| Total, serious adverse events | Cyclophosphamide Dose Level 1 | 2 | 20 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 25459642 | RESULT |
Source PubMed
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/NCT02512679
GET https://conductscience.org/api/trials/v1/NCT02512679/registered