Trials Sponsors Conditions Drugs Pricing

Conditions

Steroid Refractory Acute Graft Versus Host Disease

Outcome Measures

Primary Outcomes (1)

Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4
OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows: * CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment * PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
4 weeks

Secondary Outcomes (8)

Number of Participants With Adverse Events
16 weeks
Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8
8 weeks
Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12
12 weeks
Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index
16 weeks
Overall Response Rate (ORR) According to the Modified Glucksberg Criteria
4 weeks, 8 weeks, and 12 weeks
+3 more secondary outcomes

Study Arms (1)

Methoxsalen with ECP

EXPERIMENTAL

Participants receive methoxsalen 20 µg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12.

Drug: MethoxsalenProcedure: Extracorporeal Photopheresis (ECP)

Interventions

MethoxsalenDrug

Sterile solution used in conjunction with photopheresis procedure.

Also known as: UVADEX®

Methoxsalen with ECP

Extracorporeal Photopheresis (ECP)Procedure

Also known as: THERAKOS® CELLEX® Photopheresis System

Eligibility Criteria

Age1 Year - 21 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

Male or female 1 to 21 years of age at the time of consent
Steroid-refractory grade B-D aGvHD.

Sponsors & Collaborators

Therakos, Inc., a Mallinckrodt Company
Industrylead

Study Sites (32)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Yale University

New Haven, Connecticut, 06520, United States

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Children's Healthcare of Atlanta, Emory - Children's Center

Atlanta, Georgia, 30322, United States

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

Boston Children's Hospital

MeSH Terms

Interventions

MethoxsalenPhotopheresis

Intervention Hierarchy (Ancestors)

FurocoumarinsCoumarinsBenzopyransPyransHeterocyclic Compounds, 1-RingHeterocyclic CompoundsHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds, 3-RingPUVA TherapyUltraviolet TherapyPhototherapyTherapeutics

Limitations and Caveats

This study did have a notable limitation in its single-group study design. This may limit a more robust assessment vs standard of care alone for primary endpoint of overall response and secondary endpoints steroid sparing and disease progression.

Results Point of Contact

Title: Medical Information Call Center
Organization: Mallinckrodt Pharmaceuticals

Study Officials

Clinical Team Leader
Mallinckrodt
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restrictive Agreement: Yes

Enrollment

29 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 3
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

August 13, 2015

First Posted

August 17, 2015

Study Start

January 20, 2016

Actual (reported)

Study Stopped

Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Participants With Steroid Refractory Acute Graft Versus Host Disease

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (8)

Study Arms (1)

Methoxsalen with ECP

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (32)

MeSH Terms

Interventions

Intervention Hierarchy (Ancestors)

Limitations and Caveats

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations

Study Stopped

Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Participants With Steroid Refractory Acute Graft Versus Host Disease

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (8)

Study Arms (1)

Methoxsalen with ECP

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (32)

MeSH Terms

Interventions

Intervention Hierarchy (Ancestors)

Limitations and Caveats

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Documents

Locations