Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Patients With Steroid Refractory Acute Graft Versus Host Disease
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|ClinicalTrials.gov Identifier: NCT02524847|
Recruitment Status : Completed
First Posted : August 17, 2015
Last Update Posted : October 30, 2019
|Condition or disease||Intervention/treatment||Phase|
|Steroid Refractory Acute Graft Versus Host Disease||Drug: Methoxsalen with ECP||Phase 3|
After the informed consent/assent form (ICF) is signed, the screening assessments will be performed in a single visit to establish the eligibility of the patient, based on inclusion and exclusion criteria, as well as aGvHD grading. Scheduling of the first week of ECP treatments and the arrangements for availability of typed and cross-matched donor packed red blood cells (PRBCs) for transfusion, if required, will be made in advance of patients entering the Treatment Period.
Once eligibility is established, patients will enter the 12-week ECP Treatment Period. The availability of typed and cross-matched donor PRBCs for transfusion during treatment, if needed, should be established prior to the scheduling of ECP treatments.
Patients will be allowed to continue standard aGvHD prophylaxis regimens (e.g., cyclosporine, tacrolimus, methotrexate, mycophenolate mofetil) without the addition of new therapies. Patients will be allowed to discontinue prophylaxis regimens for reasons of toxicity, and will also be allowed to switch to another prophylaxis medication within the same class (e.g., the calcineurin inhibitors cyclosporine and tacrolimus) for reasons of toxicity.
All patients enrolled in this trial will have received corticosteroids for the treatment of aGvHD. After entering the treatment period on study, tapering of steroids by total weekly decrements of 12.5% to 25% of the steroid dose at initiation of ECP therapy is permitted after a sustained response of aGvHD has been observed for at least 3 consecutive days, with the suggested goal to decrease the starting steroid dose by at least 50% 4 weeks after initiation of ECP.
After completion of the 12-week Treatment Period, patients may continue ECP treatment on commercial product at the Principal Investigator's discretion. Acute GvHD status will be assessed 4 weeks after completion of the Treatment Period. Patient survival will be assessed by passive follow-up (chart review) 26 weeks after initiation of ECP treatment.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction With the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft-vs-Host Disease (aGvHD)|
|Actual Study Start Date :||January 20, 2016|
|Actual Primary Completion Date :||July 16, 2019|
|Actual Study Completion Date :||July 16, 2019|
Experimental: Methoxsalen with ECP
All patients will receive methoxsalen and ECP, as part of the same interventional treatment for 12 weeks. Treatments will be given 3 times a week for Weeks 1-4 and 2 times a week for Weeks 5-12.
Drug: Methoxsalen with ECP
Combination product: Methoxsalen sterile solution in conjunction with extracorporeal photopheresis (ECP)
- Complete response plus partial response (CR+PR) [ Time Frame: 4 weeks ]The proportion of patient who achieve an overall response (CR+PR) after 4 weeks of ECP treatment
- Safety parameters (vital signs, laboratory tests, and spontaneously reported adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: 12 weeks ]Safety parameters including vital signs, laboratory tests, and spontaneously reported AEs and SAEs
- Overall response [ Time Frame: 8 weeks ]Proportion of patients who achieve an overall response 8 weeks after initiation of ECP treatment
- Duration of response [ Time Frame: 16 weeks ]Duration of response, defined as the length of time a patient maintains a response through Week 16 of the follow-up period
- Overall response of Glucksberg criteria [ Time Frame: 4 weeks (Day 28), 8 weeks (Day 56), and 12 weeks (Day 84) ]Proportion of patients who achieve an overall response after 4 weeks (Day 28), 8 weeks (Day 56), and 12 weeks (Day 84) of ECP treatment according to the modified Glucksberg Criteria
- Steroid sparing effect [ Time Frame: 12 weeks ]Cumulative dose of daily steroids administered from diagnosis of aGvHD to 12 weeks after initiation of ECP treatment
- Organ specific CR+PR rates [ Time Frame: 4 weeks (Day 28), 8 weeks (Day 56), and 12 weeks (Day 84) ]Organ specific CR+PR rates at 4 weeks (Day 28), 8 weeks (Day 56), and 12 weeks (Day 84) after initiation of ECP treatment
- Very good partial response (VGPR) [ Time Frame: 4 weeks (Day 28), 8 weeks (Day 56), and 12 weeks (Day 84) ]Proportion of patients who achieve a VGPR 4 weeks (Day 28), 8 weeks (Day 56), and 12 weeks (Day 84) after initiation of ECP treatment
- Additional immunosuppressive therapy [ Time Frame: 12 weeks ]Addition of use of alternative immunosuppressive therapies throughout the 12 week Treatment Period
- Nonrelapse mortality [ Time Frame: 12 weeks (Day 84) and 16 weeks (Day 112) ]Nonrelapse mortality 12 weeks (Day 84) and 16 weeks (Day 112) after initiation of ECP treatment
- Performance status [ Time Frame: 4 weeks (Day 28) and 8 weeks (Day 56) ]Performance status (Karnofsky/Lansky score) 4 weeks (Day 28) and 8 weeks (Day 56) after initiation of ECP treatment
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): NCT02524847
|Principal Investigator:||Carrie Kitko, MD||Vanderbilt Medical Center|