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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03805789




Registration number
NCT03805789
Ethics application status
Date submitted
14/01/2019
Date registered
16/01/2019

Titles & IDs
Public title
The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft-versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant
Scientific title
A Phase 2/3, Multicenter, randOmized, Double-blind, Placebo-controlled, stUdy to evaLuate the Safety and Efficacy of Alpha-1 AntiTrypsin for the prEvention of Graft-versus-host Disease in Patients Receiving Hematopoietic Cell Transplant (MODULAATE Study)
Secondary ID [1] 0 0
2018-000329-29
Secondary ID [2] 0 0
CSL964_2001
Universal Trial Number (UTN)
Trial acronym
MODULAATE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute-graft-versus-host Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - AAT
Treatment: Other - Placebo

Experimental: AAT (low dose) - Open label. AAT is a lyophilized product for intravenous (IV) administration

Experimental: AAT (medium dose) - Open label. AAT is a lyophilized product for IV administration

Experimental: AAT (high dose) - Open label. AAT is a lyophilized product for IV administration

Experimental: AAT (selected dose from open-label) - Double-blind. AAT is a lyophilized product for IV administration

Placebo comparator: Placebo - Albumin solution administered intravenously


Treatment: Other: AAT
AAT is a lyophilized product for IV administration.

Treatment: Other: Placebo
Albumin solution administered intravenously
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The time to Grade II-IV aGVHD or death
Timepoint [1] 0 0
Through 180 days after HCT
Secondary outcome [1] 0 0
Proportion of participants with lower gastrointestinal (GI) aGVHD or Grade III-IV aGVHD in any organ
Timepoint [1] 0 0
Through 180 days after HCT
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Through Day 60 after HCT
Secondary outcome [3] 0 0
Proportion of participants with Grade II-IV aGVHD or death
Timepoint [3] 0 0
Through 100 days and 180 days after HCT
Secondary outcome [4] 0 0
Proportion of participants with lower GI aGVHD
Timepoint [4] 0 0
Through Days 60, 100 and 180 after HCT
Secondary outcome [5] 0 0
Proportion of participants with severe infections defined by NCI-CTCAE >= Grade 3
Timepoint [5] 0 0
Through 100 and 180 days after HCT
Secondary outcome [6] 0 0
Number of deaths (relapse and nonrelapse-related)
Timepoint [6] 0 0
Within 180, 365, and 730 days after HCT
Secondary outcome [7] 0 0
Proportion of participants with Grade III-IV aGVHD or death
Timepoint [7] 0 0
Through Days 60, 100, and 180 days after HCT
Secondary outcome [8] 0 0
Proportion of participants with moderate to severe chronic GVHD
Timepoint [8] 0 0
Within 180, 365, 545, and 730 days after HCT
Secondary outcome [9] 0 0
Proportion of participants who have discontinued immune suppression therapies including standard of care GVHD prophylaxis and steroid treatment
Timepoint [9] 0 0
Within 180 and 365 days after HCT
Secondary outcome [10] 0 0
Time to neutrophil engraftment
Timepoint [10] 0 0
Through 365 days after HCT
Secondary outcome [11] 0 0
Time to GVHD relapse-free survival
Timepoint [11] 0 0
Within 365 and 730 days after HCT
Secondary outcome [12] 0 0
Proportion of participants with relapse of primary malignancies
Timepoint [12] 0 0
Through 180, 365, and 730 days after HCT
Secondary outcome [13] 0 0
Proportion of participants with Grade II-IV aGVHD with an overall (complete + partial) response, complete response and partial response
Timepoint [13] 0 0
Approximately 4 weeks after the initiation of systemic steroids during 8-week Treatment Period
Secondary outcome [14] 0 0
Percent of participants with study drug related adverse events
Timepoint [14] 0 0
Up to 365 days after HCT
Secondary outcome [15] 0 0
Maximum concentration (Cmax) of AAT
Timepoint [15] 0 0
Before and up to 72 after infusion of AAT
Secondary outcome [16] 0 0
Area under the concentration curve (AUC) for AAT
Timepoint [16] 0 0
Before and up to 72 after infusion of AAT
Secondary outcome [17] 0 0
Ctrough of AAT
Timepoint [17] 0 0
Before and up to 72 after infusion of AAT
Secondary outcome [18] 0 0
Clearance (CL) of AAT
Timepoint [18] 0 0
Before and up to 72 after infusion of AAT
Secondary outcome [19] 0 0
Volume of distribution (V) for AAT
Timepoint [19] 0 0
Before and up to 72 after infusion of AAT

Eligibility
Key inclusion criteria
* Male or female participants, >=12 years of age (>= 18 years of age for participants at German sites only), undergoing HCT for hematological malignancies, including leukemia, lymphoma, multiple myeloma, myelodysplastic syndrome, and myeloproliferative neoplasms.
* Planned myeloablative conditioning regimen.
* Participants must have a related or unrelated donor as follows:

* Related donor must be a 6 / 6 match for human leukocyte antigen (HLA)-A, -B, at intermediate (or higher) resolution, and -DR beta 1 (DRB1) at high resolution using deoxyribonucleic acid (DNA)-based typing.
* Unrelated donor must be 7 / 8 or 8 / 8 match for HLA-A, -B, and -C at intermediate (or higher) resolution, and -DRB1 at high resolution using DNA-based typing.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Prior autologous or allogeneic HCT.
* T cell depleted transplant or planned use of anti-T cell antibody therapy either ex vivo or in vivo (ie, anti thymocyte globulin [ATG], alemtuzumab) for GVHD prophylaxis.
* Planned umbilical cord blood transplant.
* Planned use of cyclophosphamide after HCT for GVHD prophylaxis.
* Planned haploidentical donor.

Study design
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/03/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
18/10/2029
Actual
Sample size
Target
310
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Queenlan
Recruitment hospital [1] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Utah
Country [11] 0 0
United States of America
State/province [11] 0 0
Virginia
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington
Country [13] 0 0
Germany
State/province [13] 0 0
Köln
Country [14] 0 0
Italy
State/province [14] 0 0
Catania
Country [15] 0 0
Italy
State/province [15] 0 0
Calabria
Country [16] 0 0
Japan
State/province [16] 0 0
Anjo-shi
Country [17] 0 0
Japan
State/province [17] 0 0
Bunkyo-ku
Country [18] 0 0
Japan
State/province [18] 0 0
Hiroshima
Country [19] 0 0
Japan
State/province [19] 0 0
Nagoya-shi
Country [20] 0 0
Japan
State/province [20] 0 0
Nagoya
Country [21] 0 0
Japan
State/province [21] 0 0
Okayama-shi
Country [22] 0 0
Japan
State/province [22] 0 0
Osaka-shi
Country [23] 0 0
Japan
State/province [23] 0 0
Sapporo
Country [24] 0 0
Korea, Republic of
State/province [24] 0 0
Busan
Country [25] 0 0
Korea, Republic of
State/province [25] 0 0
Incheon
Country [26] 0 0
Korea, Republic of
State/province [26] 0 0
Seoul
Country [27] 0 0
Spain
State/province [27] 0 0
Barcelona
Country [28] 0 0
Spain
State/province [28] 0 0
Salamanca
Country [29] 0 0
Turkey
State/province [29] 0 0
Ankara
Country [30] 0 0
Turkey
State/province [30] 0 0
Battalgazi

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
CSL Behring
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Physician
Address 0 0
CSL Behring
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Registration Coordinator
Address 0 0
Country 0 0
Phone 0 0
+1 610-878-4697
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03805789