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Trial record 1 of 1 for:    NCT03339297
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An Open-Label Study of Defibrotide for the Prevention of Acute Graft-versus-Host-Disease (AGvHD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03339297
Recruitment Status : Completed
First Posted : November 13, 2017
Last Update Posted : July 1, 2020
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE November 8, 2017
First Posted Date  ICMJE November 13, 2017
Last Update Posted Date July 1, 2020
Actual Study Start Date  ICMJE February 21, 2018
Actual Primary Completion Date May 12, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 29, 2017)
Cumulative Incidence of Grade B-D aGvHD by Day +100 post-allogeneic HSCT [ Time Frame: 100 Days post-HSCT ]
Original Primary Outcome Measures  ICMJE
 (submitted: November 8, 2017)
Cumulative Incidence of Grade B-D aGvHD by Day +100 post-allogeneic HCST [ Time Frame: 100 Days post-HSCT ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 29, 2017)
  • Grade B-D aGvHD-free survival by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Cumulative Incidence of Grade B-D aGvHD by Day +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Cumulative Incidence of Grade C-D aGvHD by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Cumulative Incidence of Relapse by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Incidence of Systemic Steroid Use in the Treatment of aGvHD by Day +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Health-related Quality of Life (HRQoL) as Measured by Functional Assessment of Cancer Therapy-Bone Marrow Transplant-Trial Outcomes Index (FACT-BMT-TOI) [ Time Frame: 180 Days post-HSCT ]
  • HRQoL as Measured by EuroQOL-5D (EQ-5D) [ Time Frame: 180 Days post-HSCT ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 8, 2017)
  • Grade B-D aGvHD-free survival by Days +100 and +180 post-HCST [ Time Frame: 180 Days post-HSCT ]
  • Cumulative Incidence of Grade B-D aGvHD by Day +180 post-HCST [ Time Frame: 180 Days post-HSCT ]
  • Cumulative Incidence of Grade C-D aGvHD by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Cumulative Incidence of Relapse by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Incidence of Systemic Steroid Use in the Treatment of aGvHD by Day +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  • Health-related Quality of Life (HRQoL) as Measured by Functional Assessment of Cancer Therapy-Bone Marrow Transplant-Trial Outcomes Index (FACT-BMT-TOI) [ Time Frame: 180 Days post-HSCT ]
  • HRQoL as Measured by EuroQOL-5D (EQ-5D) [ Time Frame: 180 Days post-HSCT ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-Label Study of Defibrotide for the Prevention of Acute Graft-versus-Host-Disease (AGvHD)
Official Title  ICMJE A Phase 2, Prospective, Randomized, Open-Label Study on the Efficacy of Defibrotide Added to Standard of Care Immunoprophylaxis for the Prevention of Acute Graft-versus-Host-Disease in Adult and Pediatric Patients After Allogeneic Hematopietic Stem Cell Transplant
Brief Summary This is a study comparing the defibrotide prophylaxis arm vs standard of care arm for the prevention of aGvHD.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Graft-versus-host Disease
  • Acute-graft-versus-host Disease
Intervention  ICMJE
  • Drug: Defibrotide
    6.25 mg/kg via 2-hour IV infusion every 6 hours
  • Drug: Standard of Care
    Administered according to local institutional guidelines, physician preference, and patient need.
Study Arms  ICMJE
  • Experimental: Defibrotide Prophylaxis
    Standard of Care Immunoprophylaxis + Defibrotide
    Interventions:
    • Drug: Defibrotide
    • Drug: Standard of Care
  • Active Comparator: Standard of Care
    Standard of Care Immunoprophylaxis Alone
    Intervention: Drug: Standard of Care
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 14, 2020)
152
Original Estimated Enrollment  ICMJE
 (submitted: November 8, 2017)
150
Actual Study Completion Date  ICMJE May 12, 2020
Actual Primary Completion Date May 12, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Patient must be ≥1 year and <75 years of age at screening and undergoing allogeneic HSCT.
  2. Patient must be diagnosed with acute leukemia in morphologic complete remission (CR1 or CR2) or with MDS with no circulating blasts and with less than 5% blasts in the bone marrow
  3. Patient must have planned to receive either a myeloablative or reduced-intensity conditioning regimen and have an unrelated donor who is HLA matched or single-allele mismatched
  4. Patient must receive the following medical regimen as part of standard of care immunoprophylaxis for GvHD in either study arm at doses and regimen determined by local institutional guidelines, physician preference, and patient need:

    MTX or MMF + calcineurin inhibitor (CSA or TAC) +/- ATG (ATG use is limited to 30% of patients).

  5. Graft must be a CD3+ T-cell replete PBSC graft or non-manipulated BM graft.
  6. Adult patients must be able to understand and sign a written informed consent. For pediatric patients, the parent/legal guardian or representative must be able to understand and sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.

Exclusion Criteria:

  1. Patient has had a prior autologous or allogeneic HSCT.
  2. Patient is using or plans to use an investigational agent for the prevention of GvHD.
  3. Patient is receiving or plans to receive other investigational therapy and/or is enrolled or plans to enroll in a separate clinical study.
  4. Patient, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
  5. Patient has a psychiatric illness that would prevent the patient or legal guardian or representative from giving informed consent and/or assent.
  6. Patient has a serious active disease or co-morbid medical condition, as judged by the investigator, which would interfere with the conduct of this study.
  7. Patient is pregnant or lactating and does not agree to stop breastfeeding.
  8. Any other condition that would cause a risk to the patient if he/she participated in the trial.
  9. Patient has a known history of hypersensitivity to defibrotide or any of the excipients.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 74 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Belgium,   Bulgaria,   Canada,   Croatia,   France,   Germany,   Greece,   Italy,   Poland,   Portugal,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03339297
Other Study ID Numbers  ICMJE JZP963-201
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Jazz Pharmaceuticals
Study Sponsor  ICMJE Jazz Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Jazz Pharmaceuticals
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP