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Extracorporeal Photopheresis Using Theraflex ECP™ for Patients With Refractory Chronic Graft Versus Host Disease (cGVHD)

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ClinicalTrials.gov Identifier: NCT03083574
Recruitment Status : Recruiting
First Posted : March 20, 2017
Last Update Posted : March 20, 2017
Sponsor:
Collaborators:
Macopharma
Belgian Hematological Society
Information provided by (Responsible Party):
Jules Bordet Institute

Brief Summary:
The present project is a prospective, multicenter, non-randomized, phase II trial which aims to evaluate the clinical impact and the safety of extracorporeal photopheresis (ECP) using the Theraflex system in patients with refractory chronic graft versus host disease (cGVHD) after any type of hematopoietic stem cell transplantation or after donor lymphocyte infusion.

Condition or disease Intervention/treatment Phase
Refractory Chronic Graft Versus Host Disease (cGVHD) Device: Photopheresis Theraflex ECP™ Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.


Secondary Outcome Measures :
  1. 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)

  2. Discontinuation of immunosuppressive drugs during the ECP treatment [ Time Frame: During the study (8 years and 2months) ]
  3. 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) ]
  4. Incidence of viral, bacterial, fungal and parasitic infections [ Time Frame: During the study (8 years and 2months) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

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


Contacts
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Contact: Philippe Lewalle, MD, PhD 32-2-5417208 philippe.lewalle@bordet.be

Locations
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Belgium
Ziekenhuis Netwerk Antwerpen Recruiting
Antwerpen, Belgium, 2060
Contact: Pierre Zachee, MD, PhD    32-3-2177111    pierre.zachee@zna.be   
Principal Investigator: Pierre Zachee, MD, PhD         
AZ Sint-Jan Brugge Recruiting
Brugge, Belgium, 8000
Contact: Dominik Selleslag, MD    32-50-453060    dominik.selleslag@azbrugge.be   
Contact: Tom Lodewyck, MD    32-50-453060    tom.lodewyck@azbrugge.be   
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    philippe.lewalle@bordet.be   
Principal Investigator: Philippe Lewalle, MD, PhD         
Universitair Ziekenhuis Antwerpen Recruiting
Edegem, Belgium, 2650
Contact: Zwi Berneman, MD, PhD    32-3-2204111    zwi.berneman@uza.be   
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         
CHU Liège Recruiting
Liège, Belgium, 4000
Contact: Yves Beguin, MD, PhD    32-4-3667201    yves.beguin@chu.ulg.ac.be   
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         
Sponsors and Collaborators
Jules Bordet Institute
Macopharma
Belgian Hematological Society
Investigators
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Principal Investigator: Philippe Lewalle, MD, PhD Jules Bordet Institute

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Responsible Party: Jules Bordet Institute
ClinicalTrials.gov Identifier: NCT03083574     History of Changes
Other Study ID Numbers: BHS-TC-11
First Posted: March 20, 2017    Key Record Dates
Last Update Posted: March 20, 2017
Last Verified: March 2017
Keywords provided by Jules Bordet Institute:
Hematology
Chronic graft versus host disease (cGVHD)
Extracorporeal photopheresis
Hematopoietic stem cell transplantation
Donor lymphocyte infusion
Additional relevant MeSH terms:
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Graft vs Host Disease
Immune System Diseases