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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-000329-29 | EudraCT Number | ||
| 2024-511164-92-00 | EU Trial (CTIS) Number |
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This study is a phase 2 / 3 prospective, double-blind, randomized, multicenter, placebo-controlled study for prevention of acute GVHD (aGVHD) in participants undergoing an unrelated (matched or single allele mismatched) or matched related allogeneic hematopoietic cell transplantation (HCT).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AAT (low dose) | Experimental | Open label. AAT is a lyophilized product for intravenous (IV) administration |
|
| AAT (medium dose) | Experimental | Open label. AAT is a lyophilized product for IV administration |
|
| AAT (high dose) | Experimental | Open label. AAT is a lyophilized product for IV administration |
|
| AAT (selected dose from open-label) | Experimental | Double-blind. AAT is a lyophilized product for IV administration |
|
| Placebo | Placebo Comparator | Albumin solution administered intravenously |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAT | Biological | AAT is a lyophilized product for IV administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| The time to Grade II-IV aGVHD or death | Acute GVHD will be assessed using the Harris scoring system. | Through 180 days after HCT |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants with lower gastrointestinal (GI) aGVHD or Grade III-IV aGVHD in any organ | Through 180 days after HCT | |
| Proportion of participants with severe infections defined by National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) greater than or equal to (>=) Grade 3 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Physician | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HonorHealth Scottsdale Shea Medical Center | Scottsdale | Arizona | 85258 | United States | ||
| Johns Hopkins Hospital |
CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
Proposed research should seek to answer a previously unanswered important medical or scientific question.
Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.
If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
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|
| Placebo | Biological | Albumin solution administered intravenously |
|
| Through Day 60 after HCT |
| Proportion of participants with Grade II-IV aGVHD or death | Through 100 days and 180 days after HCT |
| Proportion of participants with lower GI aGVHD | Through Days 60, 100 and 180 after HCT |
| Proportion of participants with severe infections defined by NCI-CTCAE >= Grade 3 | Through 100 and 180 days after HCT |
| Number of deaths (relapse and nonrelapse-related) | Death by any cause | Within 180, 365, and 730 days after HCT |
| Proportion of participants with Grade III-IV aGVHD or death | Through Days 60, 100, and 180 days after HCT |
| Proportion of participants with moderate to severe chronic GVHD | Moderate to severe chronic GVHD graded according to National Institutes of Health (NIH) scale. | Within 180, 365, 545, and 730 days after HCT |
| Proportion of participants who have discontinued immune suppression therapies including standard of care GVHD prophylaxis and steroid treatment | Within 180 and 365 days after HCT |
| Time to neutrophil engraftment | Time to the first of 3 consecutive days of absolute neutrophil counts ? 500/µL. | Through 365 days after HCT |
| Time to GVHD relapse-free survival | GVHD free, relapse free, survival defined as time to any of the following events: 1) Grade III-IV acute GVHD, 2) moderate-severe chronic GVHD, 3) primary malignancy relapse or 4) death. | Within 365 and 730 days after HCT |
| Proportion of participants with relapse of primary malignancies | Through 180, 365, and 730 days after HCT |
| Proportion of participants with Grade II-IV aGVHD with an overall (complete + partial) response, complete response and partial response | Approximately 4 weeks after the initiation of systemic steroids during 8-week Treatment Period |
| Percent of participants with study drug related adverse events | Up to 365 days after HCT |
| Maximum concentration (Cmax) of AAT | Before and up to 72 after infusion of AAT |
| Area under the concentration curve (AUC) for AAT | Before and up to 72 after infusion of AAT |
| Ctrough of AAT | Before and up to 72 after infusion of AAT |
| Clearance (CL) of AAT | Before and up to 72 after infusion of AAT |
| Volume of distribution (V) for AAT | Before and up to 72 after infusion of AAT |
| St. Petersburg |
| Florida |
| 33701 |
| United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| University of Kansas Cancer Center | Westwood | Kansas | 66205 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| University of Michigan Medical Center | Ann Arbor | Michigan | 48109 | United States |
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
| University Hospital Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| The University of Texas-MD Anderson Cancer Center | San Antonio | Texas | 77030 | United States |
| University of Utah Primary Children's Hospital | Salt Lake City | Utah | 84113 | United States |
| University of Virginia Health System | Charlottesville | Virginia | 22903 | United States |
| Fred Hutchinson Cancer Research Center | Seattle | Washington | 98109 | United States |
| Royal Brisbane and Women's Hospital | Herston | Queenland | 4029 | Australia |
| Uniklinik Köln | Cologne | 50937 | Germany |
| University Hospital Catania | Calabria | Catania | 95123 | Italy |
| Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli | Calabria | 89133 | Italy |
| Anjo Kosei Hospital | Anjo-shi | 4668602 | Japan |
| Tokyo Metropolitan Komagome Hospital | Bunkyō City | 1138677 | Japan |
| Hiroshima University Kasumi Campus | Hiroshima | 7348551 | Japan |
| Aichi Medical Center Nagoya Daiichi Hospital | Nagoya | 4538511 | Japan |
| Nagoya University Hospital | Nagoya | 4668550 | Japan |
| Okayama University Hospital | Okayama | 71008558 | Japan |
| Osaka International Cancer Institute | Osaka | 5418567 | Japan |
| Osaka Metropolitan University Hospital | Osaka | 5458586 | Japan |
| Hokkaido University Hospital | Sapporo | 0608648 | Japan |
| INJE University Haeundae Paik Hospital | Busan | 48108 | South Korea |
| Pusan National University Hospital | Busan | 49241 | South Korea |
| Gachon University Gil Medical Center | Incheon | 21565 | South Korea |
| Seoul National University Hospital | Seoul | 03080 | South Korea |
| Hospital Universitario Valle de Hebron | Barcelona | 08035 | Spain |
| Marqués de Valdecilla University Hospital | Barcelona | 39008 | Spain |
| Salamanca University Hospital | Salamanca | 37007 | Spain |
| Ankara Abdurrahman Yurtaslan | Ankara | 06200 | Turkey (Türkiye) |
| Turgut Ozal Medicine Center | Battalgazi | 44280 | Turkey (Türkiye) |
| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| C000654178 | Respreeza |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000209 | Acute-Phase Proteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000510 | Alpha-Globulins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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