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An Open-Label Study of Defibrotide for the Prevention of Acute Graft-versus-Host-Disease (AGvHD)

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ClinicalTrials.gov Identifier: NCT03339297
Recruitment Status : Recruiting
First Posted : November 13, 2017
Last Update Posted : November 14, 2018
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Brief Summary:
This is a study comparing the defibrotide prophylaxis arm vs standard of care arm for the prevention of aGvHD.

Condition or disease Intervention/treatment Phase
Graft-versus-host Disease Acute-graft-versus-host Disease Drug: Defibrotide Drug: Standard of Care Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : February 21, 2018
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2020


Arm Intervention/treatment
Experimental: Defibrotide Prophylaxis
Standard of Care Immunoprophylaxis + Defibrotide
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.

Active Comparator: Standard of Care
Standard of Care Immunoprophylaxis Alone
Drug: Standard of Care
Administered according to local institutional guidelines, physician preference, and patient need.




Primary Outcome Measures :
  1. Cumulative Incidence of Grade B-D aGvHD by Day +100 post-allogeneic HSCT [ Time Frame: 100 Days post-HSCT ]

Secondary Outcome Measures :
  1. Grade B-D aGvHD-free survival by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  2. Cumulative Incidence of Grade B-D aGvHD by Day +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  3. Cumulative Incidence of Grade C-D aGvHD by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  4. Cumulative Incidence of Relapse by Days +100 and +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  5. Incidence of Systemic Steroid Use in the Treatment of aGvHD by Day +180 post-HSCT [ Time Frame: 180 Days post-HSCT ]
  6. 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 ]
  7. HRQoL as Measured by EuroQOL-5D (EQ-5D) [ Time Frame: 180 Days post-HSCT ]


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Ages Eligible for Study:   1 Year to 74 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03339297


Contacts
Contact: Clinical Trial Disclosure & Transparency 215-832-3750 ClinicalTrialDisclosure@JazzPharma.com

Locations
United States, California
University of California, San Francisco Medical Center Recruiting
San Francisco, California, United States, 94143
United States, Florida
Blood & Marrow Transplant Center Recruiting
Orlando, Florida, United States, 32804
United States, Kansas
University of Kansas Medical Center Recruiting
Westwood, Kansas, United States, 66205
United States, Pennsylvania
Penn State Milton S Hershey Medical Center Recruiting
Hershey, Pennsylvania, United States, 17033
United States, South Carolina
MUSC-Hollings Cancer Center Recruiting
Charleston, South Carolina, United States, 29425
Belgium
UZ Leuven Recruiting
Leuven, Belgium, 3000
France
Hospital d Instructions des Armees Percy Recruiting
Clamart, France, 92141
CHRU Lille Recruiting
Lille, France, 59037
Institut Universitaire du Cancer de Toulouse - Oncopole Recruiting
Toulouse, France, 31059
Spain
Hospital Universitario Virgen del Rocio Recruiting
Sevilla, Spain, 41013
United Kingdom
St James University Hospital Recruiting
Leeds, United Kingdom, LS9 7TF
Sponsors and Collaborators
Jazz Pharmaceuticals

Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03339297     History of Changes
Other Study ID Numbers: JZP963-201
First Posted: November 13, 2017    Key Record Dates
Last Update Posted: November 14, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases
Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors