Graft Versus Host Diseasecryo-ECPNCT03361254
Cryopreservation of White Blood Cells Before Their UVA Irradiation for Graft Versus Host Disease Treatment
A study testing cryopreservation in people with Graft Versus Host Disease, run by Central Hospital, Nancy, France.
Trial summary
Extracorporeal photopheresis (ECP) is a worldwide recognized treatment of acute and chronic mild to moderate graft versus host disease (GVHD), in second or further line of treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Graft Versus Host Disease
- What is being tested?
- cryopreservation
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- 31 Mar 2020to 30 Sep 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult or child who received an allogeneic hematopoietic stem cell transplantation, with a medical follow-up at the University Hospital of Nancy, or at the other University Hospitals in the great East region (Dijon, Reims, Besançon) in which ECP is not accessible.
- Eligible for ECP for the treatment of acute or chronic steroid refractory GVHD, after pluridisciplinary concertations
- Patients (or parents) who received full information on the research and who signed a consent for this research
- Non inclusion Criteria:
- Children under 10 kg of weight
- Patients or parents who did not sign a consent
- Patients who present an absolute contra-indication for apheresis: recent pulmonary embolism, deep vein thrombosis, myocardial infarction, active and evolutive bacterial or fungal infection, hemodynamics instability, deep anemia (Hb < 8g/dl) or deep thrombocytopenia (< 20 g/L) despite transfusions, severe hypocalcemia.
- Women in age to procreate without contraception
Who cannot join
None listed.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Division and Cryopreservation of Autologous Blood Nuclear Cells for Intensive Regimen of Extracorporeal Photopheresis : Interest for the Treatment of Patients With Steroid Resistant Graft Versus Host Disease
Extracorporeal photopheresis (ECP) is a worldwide recognized treatment of acute and chronic mild to moderate graft versus host disease (GVHD), in second or further line of treatment. Contrary to immunosuppressive drugs, ECP is not associated with side effects such as opportunistic infections, and is not associated with a higher frequency of relapse of the initial hematological disease. High intensity of ECP regimen (1 to 3 sessions per week, in case of chronic or acute GVHD) seems to be correlated to a higher efficacy. However, high intensity of ECP treatment is often difficult to sustain, because of frequent logistical problems to perform aphereses, such as venous access failure, infections of central line, deep blood cytopenias that require many transfusions before performing aphereses. Merlin et al. first described the feasibility of white blood cells cryopreservation before UVA irradiation, in vitro, then in vivo. We also recently reported the feasibility and efficacy of cryopreserved ECP in a series of 20 patients (adults and children), with acute and chronic GVHD, who had recurrent contraindications to aphereses, that prevented the realization of an intensive program of ECP. No adverse events occurred, and efficacy seemed to be similar to "classical" ECP (35% of complete overall response, and 40% of partial response). White blood cells (WBC) were divided after collection on Optia or Cellex apheresis machines: one was immediately treated with 8-MOP (methoxsalen) and ultraviolet A (UVA) irradiation, while the other was cryopreserved, and further (a few days later) thawed, sensitized with 8-MOP and irradiated before injection to the patient.
The aim of this study is to analyze this method in a prospective way, with complete biological data collection, of apoptosis, cytokines release etc…, necessary to the full description of cryopreservation of white blood cells before their irradiation and reinjection to the patient.
We will propose this technique of cryopreservation to every patient with an indication of ECP for acute or chronic GVHD in Nancy Hospital for 18 months.
Read the detailed description
The main objective of this prospective interventional pilot study is to demonstrate the ability of this technique to follow high intensity regimen recommendations, as mentioned above:
- 3 ECP sessions (thawed or fresh) per week during 3 months for grade III to IV acute GVHD, then
- 2 ECP sessions (thawed or fresh) per week during 3 months for grade II acute GVHD
- 1 ECP session per week for chronic GVHD during 3 months
The other objectives are:
- To describe the safety and efficacy of cryo ECP. Efficacy will be assessed with a composite criteria test, comporting 5 items, which will be compared for each patient with a paired cohort of historical patients treated in Nancy University Hospital since 2010 (match will be made on type and grade of GVHD, and age under and over 18 year-old)
- To fully describe the biological consequences of cryopreservation of WBC before UVA irradiation: apoptosis rate and time, lymphocyte functional tests (lymphoblastic transformation test), cytokines release, number of lymphocytes, and monocytes at the time of reinjection, bacteriological tests, residual diméthylsulfoxyde (DMSO) quantification
- To demonstrate the decrease in red blood cells and platelets transfusion number, due to the sparing effect of cryo ECP on aphereses number.
- Conditions
- Graft Versus Host Disease
- Being tested
- cryopreservation
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cryopreservationBiological
cryopreservation to every patient with an indication of ECP for acute or chronic GVHD in Nancy Hospital for 18 months
What is being measured
The primary outcome is the main question the study is designed to answer.
intensity of ECP regimen
Number of ECP (fresh and thawed) sessions during 3 months
Measured: 3 months after the first ECP session
Secondary outcomes (6)
systemic infections during 3 months after cryo-ECP (safety)
Measured: at 6 weeks and 3 months after the first ECP session
occurrence of death after cryo-ECP (safety)
Measured: at 6 weeks and 3 months after the first ECP session
occurrence of relapse of the initial hematological disease after cryo-ECP (safety)
Measured: at 6 weeks and 3 months after the first ECP session
Steroid sparing effect (efficacy)
Measured: at 6 weeks and 3 months after the first ECP session
GVHD grade (severity) (efficacy)
Measured: at 6 weeks and 3 months after the first ECP session
immunosuppressive drugs used (efficacy)
Measured: at 6 weeks and 3 months after the first ECP session
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2017
- Study start31 Mar 2020 (estimated)
- Primary completion30 Jun 2020 (estimated)
- Study completion30 Sep 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by Central Hospital, Nancy, FranceThe sponsor's other registered studies.
- 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- demonstrate the ability of this technique to follow high intensity regimen recommendations
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Central Hospital, Nancy, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03361254
- Sponsor's protocol ID
- APJC2015/CRYOPCE-POCHON/NK
- Acronym
- cryo-ECP
- Status last verified
- Jan 2020
- First submitted
- 22 Nov 2017
- Last updated
- 27 Jan 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Division and Cryopreservation of Autologous Blood Nuclear Cells for Intensive Regimen of Extracorporeal Photopheresis : Interest for the Treatment of Patients With Steroid Resistant Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT03361254
- Acronym / sponsor ID
- cryo-ECP / APJC2015/CRYOPCE-POCHON/NK
- Conditions
- Graft Versus Host Disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Central Hospital, Nancy, FranceUniversity, hospital or other
- Study start
- 31 Mar 2020 (estimated)
- Primary completion
- 30 Jun 2020 (estimated)
- Study completion
- 30 Sep 2020 (estimated)
- First posted
- 4 Dec 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Extracorporeal photopheresis (ECP) is a worldwide recognized treatment of acute and chronic mild to moderate graft versus host disease (GVHD), in second or further line of treatment. Contrary to immunosuppressive drugs, ECP is not associated with side effects such as opportunistic infections, and is not associated with a higher frequency of relapse of the initial hematological disease. High intensity of ECP regimen (1 to 3 sessions per week, in case of chronic or acute GVHD) seems to be correlated to a higher efficacy. However, high intensity of ECP treatment is often difficult to sustain, because of frequent logistical problems to perform aphereses, such as venous access failure, infections of central line, deep blood cytopenias that require many transfusions before performing aphereses. Merlin et al. first described the feasibility of white blood cells cryopreservation before UVA irradiation, in vitro, then in vivo. We also recently reported the feasibility and efficacy of cryopreserved ECP in a series of 20 patients (adults and children), with acute and chronic GVHD, who had recurrent contraindications to aphereses, that prevented the realization of an intensive program of ECP. No adverse events occurred, and efficacy seemed to be similar to "classical" ECP (35% of complete overall response, and 40% of partial response). White blood cells (WBC) were divided after collection on Optia or Cellex apheresis machines: one was immediately treated with 8-MOP (methoxsalen) and ultraviolet A (UVA) irradiation, while the other was cryopreserved, and further (a few days later) thawed, sensitized with 8-MOP and irradiated before injection to the patient.
The aim of this study is to analyze this method in a prospective way, with complete biological data collection, of apoptosis, cytokines release etc…, necessary to the full description of cryopreservation of white blood cells before their irradiation and reinjection to the patient.
We will propose this technique of cryopreservation to every patient with an indication of ECP for acute or chronic GVHD in Nancy Hospital for 18 months.
Detailed description
The main objective of this prospective interventional pilot study is to demonstrate the ability of this technique to follow high intensity regimen recommendations, as mentioned above:
- 3 ECP sessions (thawed or fresh) per week during 3 months for grade III to IV acute GVHD, then
- 2 ECP sessions (thawed or fresh) per week during 3 months for grade II acute GVHD
- 1 ECP session per week for chronic GVHD during 3 months
The other objectives are:
- To describe the safety and efficacy of cryo ECP. Efficacy will be assessed with a composite criteria test, comporting 5 items, which will be compared for each patient with a paired cohort of historical patients treated in Nancy University Hospital since 2010 (match will be made on type and grade of GVHD, and age under and over 18 year-old)
- To fully describe the biological consequences of cryopreservation of WBC before UVA irradiation: apoptosis rate and time, lymphocyte functional tests (lymphoblastic transformation test), cytokines release, number of lymphocytes, and monocytes at the time of reinjection, bacteriological tests, residual diméthylsulfoxyde (DMSO) quantification
- To demonstrate the decrease in red blood cells and platelets transfusion number, due to the sparing effect of cryo ECP on aphereses number.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Model description
- demonstrate the ability of this technique to follow high intensity regimen recommendations
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Adult or child who received an allogeneic hematopoietic stem cell transplantation, with a medical follow-up at the University Hospital of Nancy, or at the other University Hospitals in the great East region (Dijon, Reims, Besançon) in which ECP is not accessible.
- Eligible for ECP for the treatment of acute or chronic steroid refractory GVHD, after pluridisciplinary concertations
- Patients (or parents) who received full information on the research and who signed a consent for this research
- Children under 10 kg of weight
- Patients or parents who did not sign a consent
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| cryopreservationcryopreservation to every patient with an indication of ECP for acute or chronic GVHD in Nancy Hospital for 18 months | Biological | – |
Primary outcome
intensity of ECP regimen
Time frame: 3 months after the first ECP session
Number of ECP (fresh and thawed) sessions during 3 months
6 secondary outcomes
systemic infections during 3 months after cryo-ECP (safety)
Time frame: at 6 weeks and 3 months after the first ECP session
occurrence of death after cryo-ECP (safety)
Time frame: at 6 weeks and 3 months after the first ECP session
occurrence of relapse of the initial hematological disease after cryo-ECP (safety)
Time frame: at 6 weeks and 3 months after the first ECP session
Steroid sparing effect (efficacy)
Time frame: at 6 weeks and 3 months after the first ECP session
GVHD grade (severity) (efficacy)
Time frame: at 6 weeks and 3 months after the first ECP session
immunosuppressive drugs used (efficacy)
Time frame: at 6 weeks and 3 months after the first ECP session
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2020.
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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.
Questions about this trial
What is this clinical trial studying?
A study testing cryopreservation in people with Graft Versus Host Disease, run by Central Hospital, Nancy, France.
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 8 inclusion and 0 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: status not confirmed, as of the sponsor's last update on 27 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Cécile POCHON, Dr. (00 33 3 83 15 46 28, c.pochon@chru-nancy.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
intensity of ECP regimen (time frame: 3 months after the first ECP session). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Jan 2020.