Trial · NCT02524847
Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Participants With Steroid Refractory Acute Graft Versus Host Disease
NCT02524847, Phase 3, Terminated, Therakos, Inc., a Mallinckrodt Company; results first posted 2020-09-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Participants With Steroid Refractory Acute Graft Versus Host Disease. Trials, record NCT02524847. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02524847
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Participants With Steroid Refractory Acute Graft Versus Host Disease
- NCT number
- NCT02524847
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 29 participants, actual
- Lead sponsor
- Therakos, Inc., a Mallinckrodt Company
- Conditions
- Steroid Refractory Acute Graft Versus Host Disease
- Interventions
- Methoxsalen, Extracorporeal Photopheresis (ECP)
- First posted
- 2015-08-17
- Last updated
- 2020-09-11
- Results first posted
- 2020-09-11
- 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.
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4 | 4 weeks | 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 |
| Secondary | Number of Participants With Adverse Events | 16 weeks | Clinically significant changes in vital signs, laboratory values and investigations are reported as adverse events. Summary data are provided below, with details listed in the adverse events module. |
| Secondary | Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8 | 8 weeks | 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 |
| Secondary | Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12 | 12 weeks | 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 |
| Secondary | Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index | 16 weeks | Duration of first response is presented for patients whose disease progressed. Duration of response is defined in the following way: Patients whose response failed: Date at which 1st disease progression occurs - date of 1st response +1. Patients whose response did not relapse: Date of 16 week follow-up or final assessment prior to week 16 (if patient withdrew early) - date of 1st response. |
| Secondary | Overall Response Rate (ORR) According to the Modified Glucksberg Criteria | 4 weeks, 8 weeks, and 12 weeks | ORR is defined as the percentage of patients who achieve an overall response after 4 weeks, 8 weeks, and 12 weeks of ECP treatment according to a scoring algorithm applied to calculate the grade of aGvHD using the modified Glucksberg Criteria. |
| Secondary | Cumulative Dose of Daily Steroids | From diagnosis of aGvHD to 12 Weeks | Steroids administered from diagnosis of aGvHD to 12 Weeks after initiation of ECP treatment |
| Secondary | Number of Patients With Skin Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria | at 4, 8 and 12 weeks | Number of patients whose skin was rated as Stage 0 - 4 using the modified Glucksberg criteria based on the Graft versus Host Disease (GvHD) rash - Stages are defined as 0=No GvHD rash, 1=Maculopapular rash on \<25% body surface area (BSA), 2=Maculopapular rash on 25-50% BSA, 3=Maculopapular rash on \>50% BSA, and 4=Generalized erythroderma plus bullous formation, which are blisters bigger than 5 mm across |
| Secondary | Number of Patients With Liver Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria | at 4, 8 and 12 weeks | Number of patients whose liver was rated as Stage 0 - 4 on the modified Glucksberg criteria - Stages are based on level of bilirubin, defined as: Stage 0 = Bilirubin \< 2.0 mg/dL, Stage 1 = Bilirubin 2.0-3.0 mg/dL, Stage 2 = Bilirubin 3.1-6.0 mg/dL, Stage 3 = Bilirubin 6.1-15.0 mg/dL, and Stage 4 = Bilirubin \> 15.0 mg/dL |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4 | 4 weeks | All participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Adverse Events | 16 weeks | All participants | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8 | 8 weeks | Evaluable Participants (Participants with appropriate efficacy data at the given time point) | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12 | 12 weeks | Evaluable Participants (Participants with appropriate efficacy data at the given time point) | percentage of participants | NUMBER |
| SECONDARY | Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index | 16 weeks | Population meeting the criterion | days | MEDIAN |
| SECONDARY | Overall Response Rate (ORR) According to the Modified Glucksberg Criteria | 4 weeks, 8 weeks, and 12 weeks | All participants with a score at the given time point | percentage of participants | NUMBER |
| SECONDARY | Cumulative Dose of Daily Steroids | From diagnosis of aGvHD to 12 Weeks | All participants | mg | MEDIAN |
| SECONDARY | Number of Patients With Skin Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria | at 4, 8 and 12 weeks | All participants with scores at the given time point | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Patients With Liver Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria | at 4, 8 and 12 weeks | All participants with scores at the given time point | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group41
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4 | Methoxsalen With ECP | 16 | Participants | ||
| Number of Participants With Adverse Events | Methoxsalen With ECP | Any serious AE | 12 | Participants | |
| Number of Participants With Adverse Events | Methoxsalen With ECP | Non-serious TEAE at 5% threshold | 17 | Participants | |
| Number of Participants With Adverse Events | Methoxsalen With ECP | Death for any cause | 3 | Participants | |
| Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8 | Methoxsalen With ECP | 73.7 | 95% Confidence Interval 48.8 to 90.9 | percentage of participants | |
| Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12 | Methoxsalen With ECP | 78.6 | 95% Confidence Interval 49.2 to 95.3 | percentage of participants | |
| Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index | Methoxsalen With ECP | 13.5 | Full Range 4 to 50 | days | |
| Overall Response Rate (ORR) According to the Modified Glucksberg Criteria | Methoxsalen With ECP | Week 4 | 50 | 95% Confidence Interval 29.1 to 70.9 | percentage of participants |
| Overall Response Rate (ORR) According to the Modified Glucksberg Criteria | Methoxsalen With ECP | Week 8 | 63.2 | 95% Confidence Interval 38.4 to 83.7 | percentage of participants |
| Overall Response Rate (ORR) According to the Modified Glucksberg Criteria | Methoxsalen With ECP | Week 12 | 78.6 | 95% Confidence Interval 49.2 to 95.3 | percentage of participants |
1 to 10 of 41Source ClinicalTrials.gov
See all 41 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Methoxsalen With ECP | 29 | |
| Overall Study | Completed Treatment Up to Week 12 | Methoxsalen With ECP | 14 | |
| Overall Study | Started Second Line Treatment | Methoxsalen With ECP | 5 | |
| Overall Study | COMPLETED | Methoxsalen With ECP | 15 | |
| Overall Study | NOT COMPLETED | Methoxsalen With ECP | 14 |
Source ClinicalTrials.gov
Adverse events41
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain | Gastrointestinal disorders | Methoxsalen With ECP | 4 | 29 | |
| other | Nausea | Gastrointestinal disorders | Methoxsalen With ECP | 4 | 29 | |
| other | Hyperglycaemia | Metabolism and nutrition disorders | Methoxsalen With ECP | 3 | 29 | |
| other | Hypocalcaemia | Metabolism and nutrition disorders | Methoxsalen With ECP | 3 | 29 | |
| other | Hyperkalaemia | Metabolism and nutrition disorders | Methoxsalen With ECP | 2 | 29 | |
| other | Hypophosphataemia | Metabolism and nutrition disorders | Methoxsalen With ECP | 2 | 29 | |
| other | Malnutrition | Metabolism and nutrition disorders | Methoxsalen With ECP | 2 | 29 | |
| other | Hypertension | Vascular disorders | Methoxsalen With ECP | 4 | 29 | |
| other | Hypotension | Vascular disorders | Methoxsalen With ECP | 3 | 29 | |
| other | Clostridium difficile infection | Infections and infestations | Methoxsalen With ECP | 2 | 29 |
1 to 10 of 41Source ClinicalTrials.gov
See all 41 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Methoxsalen With ECP | 3 | 29 |
| Total, other adverse events | Methoxsalen With ECP | 17 | 29 |
| Total, serious adverse events | Methoxsalen With ECP | 12 | 29 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02524847
GET https://conductscience.org/api/trials/v1/NCT02524847/results?page=2