Trial · NCT01998633
Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
NCT01998633, Phase 2, Completed, Medical College of Wisconsin; results first posted 2018-06-01
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204). Trials, record NCT01998633. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01998633
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
- NCT number
- NCT01998633
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 47 participants, actual
- Lead sponsor
- Medical College of Wisconsin
- Conditions
- Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
- Interventions
- Hematopoietic Stem Cell Transplant
- First posted
- 2013-12-02
- Last updated
- 2022-12-08
- Results first posted
- 2018-06-01
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Overall Survival (OS) | 1 year and 18 months post-transplant | Overall survival is defined as survival of death from any cause. |
| Secondary | Percentage of Participants With Overall Survival (OS) by Disease Type | 1 year and 18 months post-transplant | Overall survival is defined as survival of death from any cause. |
| Secondary | Percentage of HLH Participants With HLH Reactivation Post-Transplant | 1 year post-transplant | Systemic HLH Reactivation: Post-transplant HLH reactivation is defined by clinical and lab evidence of pathologic inflammation (persistent fever, progressive cytopenias, rising ferritin and soluble IL2Rα, decreasing fibrinogen, hepatosplenomegaly, end-organ damage) not attributable to other causes. Central nervous system (CNS) HLH Reactivation: Reactivation of CNS inflammation in patients with HLH may present with or without altered mental status and is defined by pleocytosis in Cerebrospinal fluid (CSF) or an MRI consistent with CNS inflammation not attributable to other causes. |
| Secondary | Percentage of Participants With Neutrophil Engraftment | Day 42 post-transplant | Time to absolute neutrophil count (ANC) engraftment is defined as the first of three measurements on different days that the patient has an absolute neutrophil count of ≥ 500x10\^6/liter following conditioning regimen induced nadir. |
| Secondary | Percentage of Participants With Platelet Engraftment | Day 100 post-transplant | Platelet engraftment is defined as the first day of a minimum of three measurements on different days that the patient has achieved a platelet count \> 20,000 / microliter AND the patient is platelet transfusion independent for a minimum of seven days following conditioning regimen induced nadir. |
| Secondary | Percentage of Participants Alive With Sustained Engraftment | 1 year post-transplant | Sustained engraftment is defined as the occurrence of whole blood donor chimerism \> 5% by Day 42 accompanied by the absence of any primary or secondary graft failure. Primary graft failure is defined as \< 5% donor chimerism by Day +42, second stem cell infusion, DLI (except in the case of donor CTLs given for infection control), or second HCT following original HCT. Secondary graft failure is defined as \< 5% donor chimerism following initial engraftment. |
| Secondary | Percentage of Participants Alive With Sustained Engraftment by Disease Type | 1 year post-transplant | Sustained engraftment is defined as the occurrence of whole blood donor chimerism \> 5% by Day 42 accompanied by the absence of any primary or secondary graft failure. Primary graft failure is defined as \< 5% donor chimerism by Day +42, second stem cell infusion, DLI (except in the case of donor CTLs given for infection control), or second HCT following original HCT. Secondary graft failure is defined as \< 5% donor chimerism following initial engraftment. |
| Secondary | Number of Participants With Acute Graft-Versus-Host Disease (GVHD) | 1 year post-transplant | Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995: Skin stage: 0: No rash 1. Rash \<25% of body surface area 2. Rash on 25-50% of body surface area 3. Rash on \> 50% of body surface area 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level)\*: 0: \<2 mg/dL 1. 2-3 mg/dL 2. 3.01-6 mg/dL 3. 6.01-15.0 mg/dL 4. \>15 mg/dL GI stage\*: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus \* If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1. GVHD grade: 0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4 |
| Secondary | Percentage of Participants With Grade II-IV and Grade III-IV Acute GVHD | Day 100 and 6 months post-transplant | Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995: Skin stage: 0: No rash 1. Rash \<25% of body surface area 2. Rash on 25-50% of body surface area 3. Rash on \> 50% of body surface area 4. Generalized erythroderma with bullous formation Liver stage (based on bilirubin level)\*: 0: \<2 mg/dL 1. 2-3 mg/dL 2. 3.01-6 mg/dL 3. 6.01-15.0 mg/dL 4. \>15 mg/dL GI stage\*: 0: No diarrhea or diarrhea \<500 mL/day 1. Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD 2. Diarrhea 1000-1499 mL/day 3. Diarrhea \>1500 mL/day 4. Severe abdominal pain with or without ileus \* If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1. GVHD grade: 0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4 |
| Secondary | Number of Participants With Chronic GVHD | 1 year post-transplant | Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Overall Survival (OS) | 1 year and 18 months post-transplant | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants With Overall Survival (OS) by Disease Type | 1 year and 18 months post-transplant | percentage of participants | NUMBER | |
| SECONDARY | Percentage of HLH Participants With HLH Reactivation Post-Transplant | 1 year post-transplant | Participants with HLH | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Neutrophil Engraftment | Day 42 post-transplant | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants With Platelet Engraftment | Day 100 post-transplant | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants Alive With Sustained Engraftment | 1 year post-transplant | percentage of participants | NUMBER | |
| SECONDARY | Percentage of Participants Alive With Sustained Engraftment by Disease Type | 1 year post-transplant | percentage of participants | NUMBER | |
| SECONDARY | Number of Participants With Acute Graft-Versus-Host Disease (GVHD) | 1 year post-transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Percentage of Participants With Grade II-IV and Grade III-IV Acute GVHD | Day 100 and 6 months post-transplant | percentage of participants | NUMBER | |
| SECONDARY | Number of Participants With Chronic GVHD | 1 year post-transplant | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Overall Survival (OS) | Hematopoietic Stem Cell Transplant | 1 year OS | 80.4 | 95% Confidence Interval 68.6 to 88.2 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) | Hematopoietic Stem Cell Transplant | 18 month OS | 66.7 | 95% Confidence Interval 52.9 to 77.3 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) by Disease Type | Hemophagocytic Lymphohistiocytosis (HLH) | 1 year OS | 82.4 | 95% Confidence Interval 68.4 to 90.6 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) by Disease Type | Other Primary Immune Deficiencies (PID) | 1 year OS | 75.0 | 95% Confidence Interval 47.4 to 89.5 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) by Disease Type | Hemophagocytic Lymphohistiocytosis (HLH) | 18 month OS | 68.0 | 95% Confidence Interval 51.8 to 79.7 | percentage of participants |
| Percentage of Participants With Overall Survival (OS) by Disease Type | Other Primary Immune Deficiencies (PID) | 18 month OS | 62.5 | 95% Confidence Interval 32.7 to 82.0 | percentage of participants |
| Percentage of HLH Participants With HLH Reactivation Post-Transplant | Hematopoietic Stem Cell Transplant | 14.7 | 95% Confidence Interval 5.2 to 28.7 | percentage of participants | |
| Percentage of Participants With Neutrophil Engraftment | Hematopoietic Stem Cell Transplant | 100.0 | 95% Confidence Interval 90.8 to 100.0 | percentage of participants | |
| Percentage of Participants With Platelet Engraftment | Hematopoietic Stem Cell Transplant | 88.9 | 95% Confidence Interval 73.3 to 95.6 | percentage of participants | |
| Percentage of Participants Alive With Sustained Engraftment | Hematopoietic Stem Cell Transplant | 39.1 | 95% Confidence Interval 25.1 to 54.6 | percentage of participants |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Hematopoietic Stem Cell Transplant | 47 | |
| Overall Study | COMPLETED | Hematopoietic Stem Cell Transplant | 46 | |
| Overall Study | NOT COMPLETED | Hematopoietic Stem Cell Transplant | 1 |
Source ClinicalTrials.gov
Adverse events14
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 |
|---|---|---|---|---|---|---|
| serious | Febrile neutropenia | Blood and lymphatic system disorders | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
| serious | Haemolytic anaemia | Blood and lymphatic system disorders | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
| serious | Cardiac arrest | Cardiac disorders | Hematopoietic Stem Cell Transplant | 2 | 46 | 2 |
| serious | Ventricular hypertrophy | Cardiac disorders | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
| serious | Death | General disorders | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
| serious | Serum sickness | Immune system disorders | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
| serious | Sepsis | Infections and infestations | Hematopoietic Stem Cell Transplant | 2 | 46 | 2 |
| serious | Post transplant lymphoproliferative disorders | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
| serious | Cerebrovascular accident | Nervous system disorders | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
| serious | Haemorrhagic stroke | Nervous system disorders | Hematopoietic Stem Cell Transplant | 1 | 46 | 1 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Hematopoietic Stem Cell Transplant | ||
| Total, other adverse events | Hematopoietic Stem Cell Transplant | 0 | 46 |
| Total, serious adverse events | Hematopoietic Stem Cell Transplant | 14 | 46 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants2
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|---|---|---|---|---|---|
| U24CA076518 | A Data Resource for Blood and Marrow Transplants and Adoptive Cellular Therapy Research | SHAW, BRONWEN; MOSKOP, AMY M | MEDICAL COLLEGE OF WISCONSIN | NATIONAL CANCER INSTITUTE | 1998 to 2025 | NIH RePORTER |
| U10HL069294 | Blood & Marrow Transplant Clinical Trials Network Data Coordinating Center | HOROWITZ, MARY MARESCA | MEDICAL COLLEGE OF WISCONSIN | NATIONAL HEART, LUNG, AND BLOOD INSTITUTE | 2011 to 2016 | NIH RePORTER |
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/NCT01998633
GET https://conductscience.org/api/trials/v1/NCT01998633/results?page=2