Steroid Refractory Acute Graft Versus Host Disease Clinical Trial
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)
This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems (ECP) in pediatric participants with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods.
Screening:
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 participant, 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 participants
entering the Treatment Period.
Treatment Period:
Once eligibility is established, participants 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.
Participants will be allowed to continue standard aGvHD prophylaxis regimens (e.g.,
cyclosporine, tacrolimus, methotrexate, mycophenolate mofetil) without the addition of new
therapies. Participants 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 participants 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.
Follow-Up Period:
After completion of the 12-week Treatment Period, participants 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. Participant survival will be
assessed by passive follow-up (chart review) 26 weeks after initiation of ECP treatment.
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| Status | Clinical Trial | Phase | |
|---|---|---|---|
| No longer available |
NCT03172455 -
Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
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