Extracorporeal Photopheresis Using Theraflex ECP™ for Patients With Refractory Chronic Graft Versus Host Disease (cGVHD)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03083574|
Recruitment Status : Recruiting
First Posted : March 20, 2017
Last Update Posted : March 20, 2017
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Refractory Chronic Graft Versus Host Disease (cGVHD)||Device: Photopheresis Theraflex ECP™||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study to Assess the Safety and the Efficacy of Extracorporeal Photopheresis Using the Theraflex ECP™ for Patients With Refractory Chronic Graft Versus Host Disease (cGVHD)|
|Study Start Date :||September 2014|
|Estimated Primary Completion Date :||September 2022|
|Estimated Study Completion Date :||September 2023|
Experimental: Photopheresis Theraflex ECP™
All patients will initially be treated by 6 cycles of extracorporeal photopheresis (i.e. extracorporeal photopheresis on two consecutive days) administered every 2 weeks. Patients will then be evaluated after 3 months and treatment continuation will be decided based on response.
Device: Photopheresis Theraflex ECP™
The Macopharma (Theraflex ECP™) approach is based on a multistep procedure involving (1) standard mononuclear cell apheresis, (2) injection of the 8-Mop in the apheresis bag, (3) UVA exposure of the bag in the Macogenic illumination device, and (4) reinfusion of the cells into the patients.
- Response rates of chronic GVHD to the Theraflex ECP treatment. [ Time Frame: During the study (8 years and 2months) ]Percentage of patients reaching complete response, percentage of patients reaching partial response.
- Duration of response. [ Time Frame: During the study (8 years and 2months) ]Time from achieving at least a partial response to the time of progression.
- GVHD-partial response survival. [ Time Frame: During the study (8 years and 2months) ]Time from partial response to either the first progression of GVHD or the date of death, whichever occurs first.
- GVHD-free Interval. [ Time Frame: During the study (8 years and 2months) ]Interval from the date of complete response to the date of the first progression of GVHD.
- GVHD-free survival. [ Time Frame: During the study (8 years and 2months) ]Time fromcomplete response to either the first progression of GVHD or the date of death, whichever occurs first.
- Percentage of steroid dose saving. [ Time Frame: During the study (8 years and 2months) ]Percentage of dose reduction from the date of first ECP to the lowest dose of steroids taken by the patient during a minimum of 3 months (except in case of dose reduction due to adverse events related to steroids)
- Discontinuation of immunosuppressive drugs during the ECP treatment [ Time Frame: During the study (8 years and 2months) ]
- Occurrence of adverse events and serious adverse events related to extracorporeal photopheresis. [ Time Frame: During the study (1 year of follow up after the last treatment) ]
- Incidence of viral, bacterial, fungal and parasitic infections [ Time Frame: During the study (8 years and 2months) ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||Child, Adult, Older Adult|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Patients must have chronic GVHD (cGVHD) occurring after any type of HSC transplantation : with any type of donor (HLA-identical siblings or HLA-matched or mismatched family or unrelated donor); with any type of conditioning (full-intensity, reduced-intensity, nonmyeloablative, no conditioning); with any type of HSC (bone marrow, PBSC, cord blood) or after donor lymphocyte infusion.
- Patients must have cGVHD primarily affecting at least one of the following organs: skin; oral mucosal; eye; liver; lung; joints; fascia. Gastro-intetinal (GI) cGVHD alone is not a sufficient inclusion criterion.
- Patients must have cGVHD that has already been treated with first-line systemic therapy for at least 1 month at effective doses. First-line systemic therapy must have included at least prednisolone 1 mg/kg/day or equivalent. In case of formal contraindication to steroid therapy, first-line systemic therapy must have included therapeutic doses of at least one of the following drugs: tacrolimus or ciclosporine (if patient not treated with a calcineurin inhibitor at onset of cGVHD), sirolimus, everolimus, mycophenolate mofetyl.
- Patients must require further salvage therapy for cGVHD because of either refractoriness or contraindication/intolerance to current therapy.
Need for salvage therapy is defined by any of the following criteria :
- the development of 1 or more new sites of disease while being treated for chronic GVHD
- progression of existing sites of disease while receiving treatment for chronic GVHD
- failure to improve despite at least 1 month of standard treatment for chronic GVHD,
relapse/progression of cGVHD while tapering current treatment for cGVHD.
- Patients may have received any number of previous lines of treatment for cGVHD.
- Concomitant treatment with other immunosuppressors is allowed if they were started and maintained at constant dosage for at least one month before the start of ECP. Shorter delay can be accepted for patients with highly progressive GVHD requiring salvage therapy.
- Signed informed consent.
- Any age.
- Weight > 15 Kg (because of leukapheresis). Weight <15 Kg is acceptable if a suitable method of leukapheresis has been developed and approved at site.
- Patient has received any investigational agent for chronic GVHD in the past 4 weeks.
- Patient has started a new line of systemic therapy for cGVHD in the past 4 weeks. Shorter delay can be accepted for patients with highly progressive GVHD requiring salvage therapy.
- Known sensitivity to psoralen compounds such as 8-methoxypsoralen
- Comorbidities that may result in photosensitivity (coexisting skin cancer or photosensitive disease (such as porphyria, lupus, albinism…)
- Aphakia. MOP is contraindicated in patients with aphakia, because of the significantly increased risk of retinal damage due to the absence of lenses
- Known allergy to one of the components used in apheresis (e.g., heparin and citrate).
- History of heparin-induced thrombocytopenia or patients with serious coagulation disorders.
- Unable to tolerate the apheresis procedure including extracorporeal volume shifts because of uncompensated congestive heart failure, pulmonary edema, severe lung disease, severe renal failure, hepatic encephalopathy, or any other reason.
- Bilirubin > 25 mg/L.
- Absolute neutrophil count < 1.0 x 109 / L despite use of growth factors
- Platelet count < 20 x 109 / L despite platelet transfusion
- HIV seropositivity.
- Uncontrolled infection
- Relapse or progression of the hematological malignancy.
- Eastern Cooperative Oncology Group (ECOG) score > 2.
- Pregnancy or breastfeeding
- Patient is a fertile man or woman who is unwilling to use contraceptive techniques during and for 12 months following treatment.
- Any serious illness with expected survival less than 6 months.
- Any clinically significant medical or other condition that in the investigator's opinion could interfere with the administration of photopheresis or interpretation of study results, or compromise the safety or wellbeing of the patient.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03083574
|Contact: Philippe Lewalle, MD, PhDemail@example.com|
|Ziekenhuis Netwerk Antwerpen||Recruiting|
|Antwerpen, Belgium, 2060|
|Contact: Pierre Zachee, MD, PhD 32-3-2177111 firstname.lastname@example.org|
|Principal Investigator: Pierre Zachee, MD, PhD|
|AZ Sint-Jan Brugge||Recruiting|
|Brugge, Belgium, 8000|
|Contact: Dominik Selleslag, MD 32-50-453060 email@example.com|
|Contact: Tom Lodewyck, MD 32-50-453060 firstname.lastname@example.org|
|Principal Investigator: Dominik Selleslag, MD|
|Sub-Investigator: Tom Lodewyck, MD|
|Institut Jules Bordet||Recruiting|
|Brussels, Belgium, 1000|
|Contact: Philippe Lewalle, MD, PhD 32-2-5417208 email@example.com|
|Principal Investigator: Philippe Lewalle, MD, PhD|
|Universitair Ziekenhuis Antwerpen||Recruiting|
|Edegem, Belgium, 2650|
|Contact: Zwi Berneman, MD, PhD 32-3-2204111 firstname.lastname@example.org|
|Principal Investigator: Zwi Berneman, MD, PhD|
|Universitair Ziekenhuis Gent||Not yet recruiting|
|Gent, Belgium, 8000|
|Contact: Lucien Noens, MD, PhD 32-9-3323464 Lucien.Noens@Ugent.be|
|Contact: Tessa Kerre, MD, PhD 32-9-3323464 Tessa.Kerre@Ugent.be|
|Principal Investigator: Tessa Kerre, MD, PhD|
|Sub-Investigator: Lucien Noens, MD, PhD|
|Universitair Ziekenhuis Brussel||Recruiting|
|Jette, Belgium, 1090|
|Contact: Rik Schots, MD, PhD 32-2-4763105 Rik.Schots@uzbrussel.be|
|Contact: Ann De Becker, MD 32-2-4763105 Ann.Debecker@uzbrussel.be|
|Principal Investigator: Rik Schots, MD, PhD|
|Sub-Investigator: Ann De Becker, MD|
|Liège, Belgium, 4000|
|Contact: Yves Beguin, MD, PhD 32-4-3667201 email@example.com|
|Contact: Frédéric Baron, MD, PhD 32-4-3667201 F.Baron@ulg.ac.be|
|Principal Investigator: Yves Beguin, MD, PhD|
|Sub-Investigator: Frederic Baron, MD, PhD|
|Sub-Investigator: Evelyne Willems, MD|
|Cliniques Universitaires Saint-Luc||Recruiting|
|Woluwe-Saint-Lambert, Belgium, 1200|
|Contact: Xavier Poiré, MD 32-2-7641809 Xavier.Poire@uclouvain.be|
|Principal Investigator: Xavier Poiré, MD|
|Principal Investigator:||Philippe Lewalle, MD, PhD||Jules Bordet Institute|
|Responsible Party:||Jules Bordet Institute|
|Other Study ID Numbers:||
|First Posted:||March 20, 2017 Key Record Dates|
|Last Update Posted:||March 20, 2017|
|Last Verified:||March 2017|
Chronic graft versus host disease (cGVHD)
Hematopoietic stem cell transplantation
Donor lymphocyte infusion
Graft vs Host Disease
Immune System Diseases