Trial · NCT01700036
A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease
NCT01700036, Phase 2, Completed, University of Michigan Rogel Cancer Center; results first posted 2018-01-05
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease. Trials, record NCT01700036. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01700036
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Pilot Study of Alpha-1-Antitrypsin (AAT) in Steroid Refractory Acute Graft vs Host Disease
- NCT number
- NCT01700036
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 40 participants, actual
- Lead sponsor
- University of Michigan Rogel Cancer Center
- Conditions
- Graft vs Host Disease
- Interventions
- Alpha-1-Antitrypsin (AAT)
- First posted
- 2012-10-04
- Last updated
- 2018-11-27
- Results first posted
- 2018-01-05
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Patients That Respond to Treatment | 4 weeks | To estimate the proportion of patients with steroid refractory acute Graft vs Host Disease) GvHD who respond (achieve either PR or CR) to Alpha-1 Antitrypsin (AAT) at a dose of 60mg/kg twice weekly for 8 doses Partial Response (PR) is defined as a decrease of at least one grade in the severity of GVHD without deterioration of any organ systems by d28 of therapy. Complete Response (CR) is defined as the resolution of all manifestations of GVHD by d28 of treatment. All organs must have a Stage 0. |
| Secondary | Percentage of Patients Who Achieve a Complete Response to Treatment | 4 weeks | To estimate the proportion of patients in complete remission without additional therapy at four weeks after the last dose of AAT Complete remission is defined as the resolution of all manifestations of GVHD by d28 of treatment. All organs must have a Stage 0. |
| Secondary | Percentage of Patients With Documented Infection | 30 days | To estimate the incidence of infection during and following treatment of steroid refractory acute GVHD with AAT |
| Secondary | The Percentage of Patients Alive at 6 Months for Patients in CR or PR | 6 months | Survival at 6 months in patients receiving AAT treatment for steroid refractory acute GVHD for patients that achieved a CR or PR. Partial Response (PR) is defined as a decrease of at least one grade in the severity of GVHD without deterioration of any organ systems by d28 of therapy. Complete Response (CR) is defined as the resolution of all manifestations of GVHD by d28 of treatment. All organs must have a Stage 0. |
| Secondary | Mean Fold Change in Plasma Biomarkers | Baseline, 2 weeks, and 4 weeks | The fold change in levels of plasma biomarkers IL-6, TNF-alpha and ST2 will be measured pre-treatment and after the administration of AAT treatment for steroid refractory GvHD. |
| Secondary | Mean Plasma Levels for Alpha-1-Antitrypsin (AAT) | 4 weeks | Plasma levels of Alpha-1-Antitrypsin(AAT)will be measured. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Patients That Respond to Treatment | 4 weeks | percentage of patients | NUMBER | |
| SECONDARY | Percentage of Patients Who Achieve a Complete Response to Treatment | 4 weeks | percentage of patients | NUMBER | |
| SECONDARY | Percentage of Patients With Documented Infection | 30 days | percentage of patients | NUMBER | |
| SECONDARY | The Percentage of Patients Alive at 6 Months for Patients in CR or PR | 6 months | 40 patients were enrolled and 26 patients achieved a CR or PR. | percentage of patients | NUMBER |
| SECONDARY | Mean Fold Change in Plasma Biomarkers | Baseline, 2 weeks, and 4 weeks | fold change from baseline | MEAN | |
| SECONDARY | Mean Plasma Levels for Alpha-1-Antitrypsin (AAT) | 4 weeks | mg/dl | MEAN |
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 |
|---|---|---|---|---|---|
| Percentage of Patients That Respond to Treatment | Treatment Arm | 65 | 95% Confidence Interval 48 to 79 | percentage of patients | |
| Percentage of Patients Who Achieve a Complete Response to Treatment | Treatment Arm | 35 | 95% Confidence Interval 21 to 52 | percentage of patients | |
| Percentage of Patients With Documented Infection | Treatment Arm | 32.5 | percentage of patients | ||
| The Percentage of Patients Alive at 6 Months for Patients in CR or PR | Treatment Arm | 50 | 95% Confidence Interval 34 to 73 | percentage of patients | |
| Mean Fold Change in Plasma Biomarkers | Treatment Arm | IL-6 Fold Change at 2 weeks | 6.3 | Standard Error 3.6 | fold change from baseline |
| Mean Fold Change in Plasma Biomarkers | Treatment Arm | IL-6 Fold Change at 4 weeks | 4 | Standard Error 1.9 | fold change from baseline |
| Mean Fold Change in Plasma Biomarkers | Treatment Arm | TNF-alpha Fold Change at 2 weeks | 1.7 | Standard Error 0.8 | fold change from baseline |
| Mean Fold Change in Plasma Biomarkers | Treatment Arm | TNF-alpha Fold Change at 4 weeks | 1.1 | Standard Error 0.2 | fold change from baseline |
| Mean Fold Change in Plasma Biomarkers | Treatment Arm | ST2 Fold Change at 2 weeks | 1.2 | Standard Error 0.4 | fold change from baseline |
| Mean Fold Change in Plasma Biomarkers | Treatment Arm | ST2 Fold Change at 4 weeks | 1.5 | Standard Error 0.7 | fold change from baseline |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Treatment Arm | 40 | |
| Overall Study | COMPLETED | Treatment Arm | 40 | |
| Overall Study | NOT COMPLETED | Treatment Arm | 0 |
Source ClinicalTrials.gov
Adverse events70
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 | Anemia | Blood and lymphatic system disorders | Treatment Arm | 4 | 40 | 4 |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Treatment Arm | 1 | 40 | 1 |
| other | Thrombotic thrombocytopenic purpura | Blood and lymphatic system disorders | Treatment Arm | 3 | 40 | 3 |
| other | Atrial fibrillation | Cardiac disorders | Treatment Arm | 1 | 40 | 1 |
| other | Sinus bradycardia | Cardiac disorders | Treatment Arm | 1 | 40 | 1 |
| other | Sinus tachycardia | Cardiac disorders | Treatment Arm | 1 | 40 | 1 |
| other | Supraventricular tachycardia | Cardiac disorders | Treatment Arm | 1 | 40 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Treatment Arm | 1 | 40 | 1 |
| other | Ascites | Gastrointestinal disorders | Treatment Arm | 1 | 40 | 1 |
| other | Colitis | Gastrointestinal disorders | Treatment Arm | 1 | 40 | 1 |
1 to 10 of 70Source ClinicalTrials.gov
See all 70 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Arm | 8 | 40 |
| Total, other adverse events | Treatment Arm | 31 | 40 |
| Total, serious adverse events | Treatment Arm | 11 | 40 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 29437593 | DERIVED |
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/NCT01700036
GET https://conductscience.org/api/trials/v1/NCT01700036/events?page=2