Clinical Trial: UVADEX® and ECP for the Treatment of Pediatric Patients With Steroid Refractory Acute Graft Versus Host Disease

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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-Refra

Brief Summary: This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems in pediatric patients with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods.

Detailed Summary:

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 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.

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.

The proportion of patient who achieve an overall response (CR+PR) after 4 weeks of ECP treatment



Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Safety parameters (vital signs, laboratory tests, and spontaneously reported AEs and 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 and 8 weeks ]
    Proportion of patients who achieve an overall response after 4 weeks and 8 weeks 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 and 8 weeks ]
    Organ specific CR+PR rates at 4 weeks and 8 weeks after initiation of ECP treatment


Original Secondary Outcome: Same as current

Information By: Mallinckrodt

Dates:
Date Received: August 13, 2015
Date Started: September 2015
Date Completion: November 2018
Last Updated: November 3, 2016
Last Verified: November 2016