Overview

This trial is active, not recruiting.

Condition chronic graft versus host disease
Treatments ecp(methoxsalen)+corticosteroids+cyclosporine or tacrolimus, corticosteroids/cyclosporine/tacrolimus
Phase phase 1
Sponsor Mallinckrodt
Collaborator Parexel
Start date November 2011
End date March 2017
Trial size 60 participants
Trial identifier NCT01380535, 10-005, 2010-022780-35

Summary

The purpose of this study is to evaluate the safety and effectiveness of extracorporeal photopheresis therapy when added to standard drug therapies administered to patients with moderate to severe chronic graft-versus-host disease.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking single blind (outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
ECP UVADEX dose (TBD) administered during Wks 2-10 (2x/wk), Wks 11-18 (2x/wk every 2 wks), and Wks 19-26 (2x/wk every 4 wks) along with corticosteroids at a dose of 1.0 mg/kg prednisone, or equivalent, daily, tapered to 0.125 mg/kg daily by Wk 24 and maintained at that dose until Wk 28.
ecp(methoxsalen)+corticosteroids+cyclosporine or tacrolimus Uvadex
ECP UVADEX dose (TBD) administered during Wks 2-10 (2x/wk), Wks 11-18 (2x/wk every 2 wks), and Wks 19-26 (2x/wk every 4 wks) along with corticosteroids at a dose of 1.0 mg/kg prednisone, or equivalent, daily, tapered to 0.125 mg/kg daily by Wk 24 and maintained at that dose until Wk 28.
(Active Comparator)
1.0 mg/kg prednisone, or equivalent, daily, tapered to 0.125 mg/kg daily by Wk 24 and maintained at that dose until Wk 28 administered with cyclosporine or Tacrolimus (dose of cyclosporine and Tacrolimus should be consistent with local institutional practice).
corticosteroids/cyclosporine/tacrolimus
1.0 mg/kg prednisone, or equivalent, daily, tapered to 0.125 mg/kg daily by Wk 24 and maintained at that dose until Wk 28 administered with cyclosporine or Tacrolimus (dose of cyclosporine/Tacrolimus should be consistent with local institutional practice).

Primary Outcomes

Measure
Overall response (complete or partial response) in cGvHD according to NIH Response Criteria
time frame: week 28

Secondary Outcomes

Measure
Proportion of patients in each treatment group achieving response rates (complete or partial) by organ system involvement
time frame: Change from baseline to week 28
Correlation between total skin score and skin assessment (% of skin involvement)
time frame: up to week 28
Quality of Life (QoL) Questionnaire SF 36 short version scores
time frame: change in baseline to week 28
QoL questionnaire, Bone marrow Transplant scores
time frame: change from baseline to week 28
Relapse of underlying malignancy
time frame: up to week 28
Results from clinical laboratory tests
time frame: up to week 28
Adverse Events Reported
time frame: up to week 28

Eligibility Criteria

Male or female participants from 18 years up to 99 years old.

Inclusion Criteria: - Have new onset of moderate or severe cGvHD as assessed by the NIH Consensus Criteria Clinical Assessment (staging and severity) with onset within 2 years of transplantation (Patients with prior acute GvHD should be on a stable dose of <0.5 mg/kg daily prednisone, or equivalent, for at least 2 weeks prior to study entry. Prior ECP for patients with acute GvHD is permitted in the study);willing to start 1.0mg/kg prednisone: Be using adequate birth control; If a woman, must have negative pregnancy test result at screening: Be able and willing to comply with all study procedures including providing informed consent Exclusion Criteria: - Be intolerant to corticosteroids; Received treatment with >2.0 mg daily prednisone, or equivalent, for cGvHD for more than 7 days prior to baseline visit; received treatment with prednisone for mild cGVHD with >.5mg/kg for > 14 days, Have evidence of known infection with human immunodeficiency virus (HIV), active Hepatitis B infection, or have uncontrolled infection requiring treatment at the time of study entry; Requires treatment with budesonide and similar low absorption oral steroids and steroid enema preparations; Receiving treatment with mycophenolate mofetil (MMF),PUVA, tyrosine kinase inhibitors, anti-tumor necrosis factor (TNF) agents, sirolimus, and bortezomib; Receiving treatment with alemtuzumab, antithymocyte globulin (ATG) or other similar long-acting agents used for treatment of acute or chronic GvHD or administered during the conditioning regimen <90 days prior to randomization

Additional Information

Official title A Randomized Controlled Study of Extracorporeal Photopheresis (ECP) Therapy With UVADEX for the Treatment of Patients With Moderate to Severe Chronic Graft-versus-Host Disease (cGvHD)
Description This is an open-label study (patients and study staff will know the identity of treatments assigned during the study) in patients with chronic graft-versus-host disease (cGvHD). Chronic graft-versus-host disease (a donator-versus-recipient-disease) is a complication that can occur after a blood stem cell or bone marrow transplant with cells from a related or unrelated donator. During cGvHD, the transplanted cells attack the recipient's body. Patients with cGVHD who meet entry criteria for the study will be randomly assigned to receive standard of care treatment for 26 weeks or standard or care treatment with extracorporeal photopheresis (ECP) for 26 weeks. Standard of care treatment consists of orally (taken by mouth) administered corticosteroids (drugs that reduce inflammation) and cyclosporine (CsA)or Tacrolimus ( drugs that suppress the patient's immune response). ECP therapy is a process that takes place in a device where the investigational drug UVADEX (methoxsalen) is injected into a germ-free bag mixed with the patient's white blood cells. After the blood cells have absorbed the drug and are exposed to ultraviolet A (UVA) radiation, the blood cells are injected back into the patient's body. During the study, a third party assessor at each study center who will be blinded (will not know) to treatment will assess the condition each patient's skin and oral mucosa at protocol-specified visits and will complete a total skin score for all patients . The study will consist of 3 phases: a screening phase, an open label treatment phase and an end-of-study (or early withdrawal) phase. The duration of patient participation will be 28 weeks and patient safety will be monitored throughout the study. An additional non-interventional 2 year follow-up will begin after the 28 week/withdrawal end-of-study phase.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Mallinckrodt.