Clinical Trial Results:
Extracorporeal Photopheresis Treatment in steroid refractory acute and chronic Graft versus Host Disease
Summary
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EudraCT number |
2015-001550-14 |
Trial protocol |
DK |
Global end of trial date |
16 Oct 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
07 Mar 2018
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First version publication date |
07 Mar 2018
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
ECP-GVHD1
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Bispebjerg hospital
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Sponsor organisation address |
Bispebjerg bakke 23, Copenhagen, Denmark, 2400
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Public contact |
Marietta Nygaard, Bispebjerg hospital, 0045 60626201, marietta.nygaard@regionh.dk
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Scientific contact |
Marietta Nygaard, Bispebjerg hospital, 0045 21494386, marietta.nygaard@regionh.dk
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
12 Feb 2018
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
16 Oct 2016
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
To evaluate the effect of treatment with extracorporeal photopheresis in acute and chronic GVHD.
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Protection of trial subjects |
There were no specific measures to protect subjects, as they received the usual treatment in the department.
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Background therapy |
Given the nature of the disease Graft versus Host Disease and the preceeding bone marrow transplantation, the patients usually recieved many other treatments. This was usually transfusions of red blood cells or platelets, or infection prophylaxis and almost all patients also received one or more immunosuppressive drugs. The latter drugs were given to treat graft versus host disease, but was always administerered at the discretion of the treating physiscian. These drugs may very well have interfered with the effect of extracorporeal photopheresis. | ||
Evidence for comparator |
We used no comparators | ||
Actual start date of recruitment |
07 Jul 2015
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Denmark: 23
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Worldwide total number of subjects |
23
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EEA total number of subjects |
23
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
16
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From 65 to 84 years |
7
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85 years and over |
0
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Recruitment
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Recruitment details |
All patients referred to extracoporeal photopheresis between 07.07.2015 (first patient enrolled) and 16.09.2016 (trial ended prior to plan) were enrolled. | |||||||||
Pre-assignment
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Screening details |
Inclusion criteria were not very strict as it was an observational trial with no control group. All patients referred to extracorporeal photopheresis were screened by sponsor/investigator (MN) and all were enrolled. | |||||||||
Period 1
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Period 1 title |
overall trial (overall period)
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Is this the baseline period? |
Yes | |||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | |||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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chronic | |||||||||
Arm description |
- | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
Uvadex
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Concentrate for emulsion for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Dependent on amount of collected blood
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Arm title
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Acute | |||||||||
Arm description |
PAtietns with acute GvHD | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
Uvadex
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Concentrate for emulsion for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Dependent on amount of collected blood
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Baseline characteristics reporting groups
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Reporting group title |
overall trial
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Reporting group description |
All patients treated with extracorporeal photopheresis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Chronic GvHD
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Subject analysis set type |
Sub-group analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Patients with chronic GvHD
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Subject analysis set title |
Acute GvHD
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Subject analysis set type |
Sub-group analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Patients with acute GvHD
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End points reporting groups
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Reporting group title |
chronic
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Reporting group description |
- | ||
Reporting group title |
Acute
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Reporting group description |
PAtietns with acute GvHD | ||
Subject analysis set title |
Chronic GvHD
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Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Patients with chronic GvHD
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Subject analysis set title |
Acute GvHD
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Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Patients with acute GvHD
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End point title |
Best or overall response | ||||||||||||||||||||||||
End point description |
The overall (cGvHD) or best response (AGvHD) during treatment with ECP
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End point type |
Primary
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End point timeframe |
overall trial
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Statistical analysis title |
None | ||||||||||||||||||||||||
Statistical analysis description |
No statistical analysis were made because the groups are not comparable. They are two different diseases.
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Comparison groups |
Chronic GvHD v Acute GvHD
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Number of subjects included in analysis |
23
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Analysis specification |
Pre-specified
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Analysis type |
other [1] | ||||||||||||||||||||||||
P-value |
< 0.05 [2] | ||||||||||||||||||||||||
Method |
No comparison made | ||||||||||||||||||||||||
Confidence interval |
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Notes [1] - No comparisons can be made. [2] - No comparisons made |
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End point title |
Survival at one year after start of treatment | |||||||||||||||
End point description |
Number of patients alive one year from start of treatment
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End point type |
Secondary
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End point timeframe |
1 year from start of treatment
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No statistical analyses for this end point |
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Adverse events information [1]
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Timeframe for reporting adverse events |
From start of trial and until first amendment, where the reporting of adverse events were changed.
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Adverse event reporting additional description |
Given the nature of the graft versus host disease and the circumstances under which the patients receive treatment, there are expected to be many serious events during the treatment course, these are not expected to be related to the ECP treatment but rather to the bone marrow transplantation and/or the concurrent immunosuppressive drugs.
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Assessment type |
Systematic | ||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
1
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Frequency threshold for reporting non-serious adverse events: 5% | |||
Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: In the amendment it is specified, why the reporting of adverse events is difficult, due to the nature of the studied diseases and the competing uncontrolled therapies. It was never an aim of this study to examine safety. |
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||||||
Date |
Amendment |
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15 Nov 2015 |
Change of the reporting of Adverse events because the patients were influenced by many other drugs and circumstances causing abnormal testings, infections and other complications. |
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Interruptions (globally) |
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Were there any global interruptions to the trial? Yes | |||||||
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Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||||||
The trial was never finished because it turned out to be impossible to perfom it meaningfully |