Chronic Graft Versus Host DiseaseEarly Phase 1NCT01380535
Extracorporeal Photopheresis (ECP) With Methoxsalen for Chronic Graft Versus Host Disease (cGVHD)
A Early Phase 1 study testing ECP Methoxsalen in people with Chronic Graft Versus Host Disease, run by Mallinckrodt at 32 sites in 8 countries.
Trial summary
Chronic graft-versus-host disease (cGVHD) is a long-lasting complication that can occur after transplants. The transplanted cells seem to fight with the patient's own cells. Extracorporeal photopheresis (ECP) is a fairly new procedure for cGVHD. The participant gets a port to hook up to a machine. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Chronic Graft Versus Host Disease
- What is being tested?
- ECP Methoxsalen
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 32 sitesin 8 countries
- Study dates
- Nov 2011to Mar 2015
- Phase
- Early Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- To be eligible for inclusion in this trial, the patient must have moderate to severe chronic graft-versus-host disease [defined by the National Institutes of Health (NIH) 2015 Consensus Criteria] that started within 3 years after hematopoietic stem cell transplantation (HSCT).
Who cannot join
- Is intolerant to corticosteroids or hypersensitive to methoxsalen
- Received certain treatments during time periods disallowed by protocol
- Has infections and/or requires treatment that (per protocol or in the opinion of the investigator) might compromise:
- safety and well-being of participant or offspring
- safety of study staff
- analysis of results
Trial locations and contacts
32 sites in 8 countries.
32 sites in 8 countries
United States11 sites
Medical City Dallas Hospital,Transplant center
Dallas, Texas, 75230
Parkland Memorial Hospital
Dallas, Texas, 75235
Karmanos Cancer Institute
Detroit, Michigan, 48201
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Indiana Blood and Marrow Transplantation Research
Indianapolis, Indiana, 46237
University of Miami Hospital
Miami, Florida, 33136
Vanderbilt Ingram Cancer Center
Nashville, Tennessee, 37232
Tulane University Health Sciences Center, 1430 Tulane Avenue, Rm 7551,
New Orleans, Louisiana, 70112
New York Presbyterian Hospital
New York, New York, 10065
Methodist healthcare system of san Antonio
San Antonio, Texas, 78229
Stony brook university medical Center
Stony Brook, New York, 11794
Germany9 sites
Universitätsklinik Köln
Cologne, 50937
Universitäts-Klinikum Carl Gustav Carus
Dresden, 01309
Universitatsklinikum Erlangen
Erlangen, 91054
Universitätsklinikum Essen
Essen, 45122
Universitatskrankenhaus Hamburg-Eppendorf
Hamburg, 20246
Universitätsklinikum Leipzig AöR
Leipzig, 04103
Universitätklinikum Mannheim
Mannheim, 68167
Medizinische Universitatsklinik
München, 81377
Uniklinik für Kinder und Jugendmedizin
Tübingen, 72076
France3 sites
CHU de Nantes
Nantes
Hospital Saint Louis
Paris
Hopital Saint Antoine
Saint-Antoine, Paris, 75571
United Kingdom3 sites
University of Birmingham: Queen Elizabeth Hospital
Birmingham
Kings College Hospital
London, Greater London, SE5 9RS
Royal Marsden Hospital
London
Italy2 sites
A.O.U. Policlinico- Vittorio Emanuele
Catania
Universita de Genova - Ospedale S. Martino
Genova
Spain2 sites
Santa Creu I Sant Pau
Barcelona, 08025
Hospital Marques de Valdecilla
Santander, Cantabria, 39008
Austria1 site
General Hospital of Vienna (Medical University of Vienna)
Vienna
Hungary1 site
Egyesített Szent István és Szent László Kórház, Gyáli út 5-7,
Budapest, 1097
About this study
Official title: A Randomized Controlled Study of Extracorporeal Photopheresis (ECP) Therapy With UVADEX for the Treatment of Patients With Moderate to Severe Chronic Graft-versus-Host Disease (cGvHD)
Chronic graft-versus-host disease (cGVHD) is a long-lasting complication that can occur after transplants. The transplanted cells seem to fight with the patient's own cells.
Extracorporeal photopheresis (ECP) is a fairly new procedure for cGVHD. The participant gets a port to hook up to a machine. The machine removes the white blood cells, mixes them with a light-sensitive drug, shines light on it, and puts all the blood back in.
This study will find out if patients respond better if they get ECP with methoxsalen, in addition to the pills normally used to treat cGVHD.
Read the detailed description
The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use.
This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment.
Adult patients with new-onset (≤3 years of hematopoietic stem cell transplantation) moderate or severe cGVHD were randomized 1:1 to 26 weeks of SoC+ECP vs SoC (corticosteroids and cyclosporine A/tacrolimus) between 2011 and 2015. The primary endpoint was overall response rate (ORR), defined as complete or partial response, at week 28 in the intention-to-treat population (ITT).
- Conditions
- Chronic Graft Versus Host Disease
- Being tested
- ECP Methoxsalen, Standard of Care
- Keywords
- Extracorporeal photopheresis therapy, Methoxsalen, cGVHD, hematopoietic stem cell transplantation
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ECP MethoxsalenDrug
Also called Uvadex, Extracorporeal photopheresis (ECP)
Methoxsalen delivered by ECP
Standard of CareDrug
Also called Reference therapy
Tapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus)
| Study group | Role | What they receive |
|---|---|---|
| ECP Methoxsalen + Standard of Care | Experimental | Participants receive methoxsalen administered via ECP in addition to standard of care Receives: Drug: ECP Methoxsalen; Drug: Standard of Care |
| Standard of Care | Active comparator | Participants receive standard of care only Receives: Drug: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With an Overall Response at Week 28
Participants with overall response included those with partial response or complete response, according to study staff who did not know which treatment they received (blinded assessment).
Measured: Week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2011 (estimated)
- Study startNov 2011
- Primary completionMar 2015
- Study completionMar 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Graft Versus Host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Pilot Study for the 2015 NIH Consensus Criteria to inform the design of subsequent studies
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mallinckrodt
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
- Investigators
- Global Clinical Leader, Study director, Mallinckrodt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01380535
- Sponsor's protocol ID
- 10-005
- Other IDs
- 2010-022780-35
- Status last verified
- Feb 2020
- First submitted
- 22 Jun 2011
- Last updated
- 19 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Jagasia M, Scheid C, Socie G, Ayuk FA, Tischer J, Donato ML, Batai A, Chen H, Chen SC, Chin T, Boodee H, Mitri G, Greinix HT. Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD. Blood Adv. 2019 Jul 23;3(14):2218-2229. doi: 10.1182/bloodadvances.2019000145.
PubMed 31332045
Study at a glance
From the registry record, last updated by the sponsor on 19 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Controlled Study of Extracorporeal Photopheresis (ECP) Therapy With UVADEX for the Treatment of Patients With Moderate to Severe Chronic Graft-versus-Host Disease (cGvHD)
- ClinicalTrials.gov ID
- NCT01380535
- Acronym / sponsor ID
- 10-005
- Conditions
- Chronic Graft Versus Host Disease
- Keywords
- Extracorporeal photopheresis therapy, Methoxsalen, cGVHD, hematopoietic stem cell transplantation
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- MallinckrodtIndustry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- Nov 2011 (actual)
- Primary completion
- Mar 2015 (actual)
- Study completion
- Mar 2015 (actual)
- First posted
- 27 Jun 2011 (estimated)
- Results
- Posted 29 Oct 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Chronic graft-versus-host disease (cGVHD) is a long-lasting complication that can occur after transplants. The transplanted cells seem to fight with the patient's own cells.
Extracorporeal photopheresis (ECP) is a fairly new procedure for cGVHD. The participant gets a port to hook up to a machine. The machine removes the white blood cells, mixes them with a light-sensitive drug, shines light on it, and puts all the blood back in.
This study will find out if patients respond better if they get ECP with methoxsalen, in addition to the pills normally used to treat cGVHD.
Detailed description
The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use.
This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment.
Adult patients with new-onset (≤3 years of hematopoietic stem cell transplantation) moderate or severe cGVHD were randomized 1:1 to 26 weeks of SoC+ECP vs SoC (corticosteroids and cyclosporine A/tacrolimus) between 2011 and 2015. The primary endpoint was overall response rate (ORR), defined as complete or partial response, at week 28 in the intention-to-treat population (ITT).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- Pilot Study for the 2015 NIH Consensus Criteria to inform the design of subsequent studies
| Arm | Type | Receives |
|---|---|---|
| ECP Methoxsalen + Standard of Care | Experimental | Drug: ECP Methoxsalen, Drug: Standard of CareParticipants receive methoxsalen administered via ECP in addition to standard of care |
| Standard of Care | Active comparator | Drug: Standard of CareParticipants receive standard of care only |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- To be eligible for inclusion in this trial, the patient must have moderate to severe chronic graft-versus-host disease [defined by the National Institutes of Health (NIH) 2015 Consensus Criteria] that started within 3 years after hematopoietic stem cell transplantation (HSCT).
Key exclusion criteria (6 listed)
- Is intolerant to corticosteroids or hypersensitive to methoxsalen
- Received certain treatments during time periods disallowed by protocol
- Has infections and/or requires treatment that (per protocol or in the opinion of the investigator) might compromise:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ECP MethoxsalenAlso: Uvadex, Extracorporeal photopheresis (ECP)Methoxsalen delivered by ECP | Drug | ECP Methoxsalen + Standard of Care |
| Standard of CareAlso: Reference therapyTapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus) | Drug | ECP Methoxsalen + Standard of Care, Standard of Care |
Primary outcome
Number of Participants With an Overall Response at Week 28
Time frame: Week 28
Participants with overall response included those with partial response or complete response, according to study staff who did not know which treatment they received (blinded assessment).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 31332045Jagasia M, Scheid C, Socie G, Ayuk FA, Tischer J, Donato ML, Batai A, Chen H, Chen SC, Chin T, Boodee H, Mitri G, Greinix HT. Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD. Blood Adv. 2019 Jul 23;3(14):2218-2229. doi: 10.1182/bloodadvances.2019000145.
- Cited article made available by the US National Library of Medicine, National Institutes of HealthLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Global Clinical Leader study director
Mallinckrodt
32 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing ECP Methoxsalen in people with Chronic Graft Versus Host Disease, run by Mallinckrodt at 32 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, Germany, France, United Kingdom, Italy and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Oct 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With an Overall Response at Week 28 (time frame: Week 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01380535, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Feb 2020.