ThrombocytopeniaPhase 4MICENCT02032134

Protocol for the Infusion of Buffy Coat-derived Cryopreserved Platelets in Patients With Severe Thrombocytopenia

A Phase 4 study testing Transfusion of Pooled Platelets Cryopreserved In DMSO in people with Thrombocytopenia and Primary Thrombocytopenia,Unspecified, run by Napolitano Mariasanta at 1 site in Italy.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

The increasing need for blood components and the increasingly careful donor selection procedures, make the availability of blood components very reduced, in particular for platelets that are currently administered preferably "fresh" or cryopreserved, only in emergency conditions. Read more about this study

Who is it for?: Ages 18 to 80 years
Conditions the study is for: Thrombocytopenia, Primary Thrombocytopenia,Unspecified, Thrombocytopenia Chemotherapy Induced
What is being tested?: Transfusion of Pooled Platelets Cryopreserved In DMSO
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 5 participants
Where: 1 siteItaly
Study dates: Jul 2015to Jun 2016
Phase: Phase 4
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 to 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Cryopreserved buffy coat pooled platelets will be infused, after being thawed, in inpatients at U.O. of Hematology -Policlinic "P. Giaccone"-Palermo, falling into one of the following categories:
Stable clinical conditions and no complications with platelets ≤ 10,000/microliter;
With active bleeding and ≤ 20,000 platelets/microliter;
In the presence of: high blood pressure, high fever, rapid drop in platelets, infection, chemotherapy, coagulation abnormalities with ≤ 20,000 platelets/microliter.

Who cannot join

Patients
Suffering from congenital immunodeficiency
Allogeneic bone marrow transplant candidates
Bone Marrow Donors for transplantation
Undergoing bone marrow transplant or
Suffering from Hodgkin's disease and treated with purine analogues (Fludarabine, cladribine)

Trial locations and contacts

1 site in 1 country.

Italy1 site

AOUP "P.Giaccone"-Hematology Unit
Palermo, Regione Sicilia, 90127

Other trials in Italy

About this study

Official title: Experimental Protocol for the Infusion of Platelets,Cryopreserved With Dimethyl Sulphoxide (DMSO) and Obtained From Multiple Donors Buffy Coats, in Patients With Severe Thrombocytopenia

Exceeding this limit through the spread of use of cryopreserved platelets in the common clinical practice, might help clinicians to avoid wasting valuable products and face periods of particular shortage of donors Some studies already published in the literature over the last decade (1,2) have demonstrated the safety and efficacy of autologous cryopreserved platelets .

The main objective of the proposed study is to evaluate the efficacy and safety of platelets, cryopreserved with DMSO using a a new method, in patients with severe thrombocytopenia.

This is the in vivo phase of a study where availability and efficacy of cryopreserved platelets has been assessed in vitro.

Read the detailed description

The primary objective of the study is to evaluate the hemostatic efficacy and safety in the recipients of platelets obtained from buffy coat of multiple donors (five), cryopreserved with DMSO using a new patented method, up to 9 months from the collection.

Further goals are the registration of any related- adverse event (idiosyncrasy, allergy, toxicity)

Conditions: Thrombocytopenia, Primary Thrombocytopenia,Unspecified, Thrombocytopenia Chemotherapy Induced
Being tested: Transfusion of Pooled Platelets Cryopreserved In DMSO
Keywords: Cryopreserved platelets, thrombocytopenia, DMSO
Medical subject headings: Thrombocytopenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Transfusion of Pooled Platelets Cryopreserved In DMSOBiological
Also called New Cryopreservation kit (Promedical ®), Buffy Coat Pooled Platelets Cryopreserved In DMSO
Patients with severe thrombocytopenia will be admitted as in patients at the Hematology Unit of the Policlinic "P. Giaccone"- Palermo, if they require platelet transfusion (for bleeding, in preparation for surgery, after chemotherapy); they will receive buffy coat derived cryopreserved platelets,after thawing

Study groups in NCT02032134
Study group	Role	What they receive
Patients with severe thrombocytopenia	Other	Intervention Patients with severe thrombocytopenia with an active bleeding, in preparation for surgery, or after chemotherapy (prophylaxis of bleeding),will receive transfusion of Pooled Platelets Cryopreserved In DMSO With a New System Receives: Biological: Transfusion of Pooled Platelets Cryopreserved In DMSO

What is being measured

The primary outcome is the main question the study is designed to answer.

Efficacy of infused cryopreserved platelets (changes in platelets count will be determined at 1 hour and 24 hours post-transfusion)
To evaluate the availability of cryopreserved platelets after transfusion, the correct count (the number of platelets poured and the body surface, BMI, recipient) of platelets will be determined at 1 hour and 24 hours post transfusion, respectively
Measured: 1 Hour and 24 hours post-transfusion

Secondary outcomes (1)

Safety of cryopreserved platelets
Any transfusion-related side effect (i.e. idiosyncrasy, allergy, toxicity) will be monitored
Measured: During transfusion and within 48 hours after transfusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jul 2015Jun 2016

First posted9 Jan 2014 (estimated)
Study startJul 2015
Primary completionJun 2016
Study completionJun 2016

Why it stopped: recruitment failure within the interval of available CRY BC-PLTs for the study

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (8)Papers the sponsor has linked to this study.
Other trials for ThrombocytopeniaStudies 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: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 5 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02032134
Sponsor's protocol ID: CUP: G33F1100003000
Acronym: MICE
Status last verified: Oct 2017
First submitted: 7 Jan 2014
Last updated: 18 Oct 2017
Results posted: No
Data monitoring committee: Yes

Publications

Wautier JL. Safety and usefulness of autologous cryopreserved platelets. Lancet. 2002 Dec 14;360(9349):1985. doi: 10.1016/S0140-6736(02)11893-9. No abstract available.
PubMed 12493312
Liu GQ, Ding HF, Lu XJ, Xu M, Xing J, Zhao X, Han F, Shang YH. [The efficacy and safety of autologous cryopreserved platelet transfusion in management of thrombocytopenia after chemotherapy in hematological malignancy]. Zhonghua Nei Ke Za Zhi. 2012 Mar;51(3):188-91. Chinese.
PubMed 22781890
Heal JM, Blumberg N. Optimizing platelet transfusion therapy. Blood Rev. 2004 Sep;18(3):149-65. doi: 10.1016/S0268-960X(03)00057-2.
PubMed 15183900
Murphy MF, Brozovic B, Murphy W, Ouwehand W, Waters AH. Guidelines for platelet transfusions. British Committee for Standards in Haematology, Working Party of the Blood Transfusion Task Force. Transfus Med. 1992 Dec;2(4):311-8. doi: 10.1111/j.1365-3148.1992.tb00175.x.
PubMed 1339584
Dumont LJ, Cancelas JA, Dumont DF, Siegel AH, Szczepiorkowski ZM, Rugg N, Pratt PG, Worsham DN, Hartman EL, Dunn SK, O'Leary M, Ransom JH, Michael RA, Macdonald VW. A randomized controlled trial evaluating recovery and survival of 6% dimethyl sulfoxide-frozen autologous platelets in healthy volunteers. Transfusion. 2013 Jan;53(1):128-37. doi: 10.1111/j.1537-2995.2012.03735.x. Epub 2012 Jun 7.
PubMed 22671278
Meyer E, Delaney M, Lin Y, Morris A, Pavenski K, Tinmouth A, Murphy M, Slichter SJ, Heddle N, Dumont LJ. A reporting guideline for clinical platelet transfusion studies from the BEST Collaborative. Transfusion. 2013 Jun;53(6):1328-34. doi: 10.1111/j.1537-2995.2012.03906.x. Epub 2012 Sep 25.
PubMed 23003345
Dumont LJ, Dumont DF, Unger ZM, Siegel A, Szczepiorkowski ZM, Corson JS, Jones MK, Christoffel T, Pellham E, Bailey SL, Slichter SJ; BEST Collaborative. A randomized controlled trial comparing autologous radiolabeled in vivo platelet (PLT) recoveries and survivals of 7-day-stored PLT-rich plasma and buffy coat PLTs from the same subjects. Transfusion. 2011 Jun;51(6):1241-8. doi: 10.1111/j.1537-2995.2010.03007.x. Epub 2011 Jan 7.
PubMed 21214584
Napolitano M, Mancuso S, Raso S, LoCoco L, Arfo PS, De Francisci G, Dieli F, Caccamo N, Reina A, Dolce A, Agliastro R, Siragusa S. Buffy coat-derived platelets cryopreserved using a new method: Results from a pivotal clinical trial on thrombocytopenic patients with acute leukaemia. Transfus Apher Sci. 2019 Dec;58(6):102666. doi: 10.1016/j.transci.2019.10.008. Epub 2019 Nov 5.
PubMed 31753773

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Thrombocytopenia trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Experimental Protocol for the Infusion of Platelets,Cryopreserved With Dimethyl Sulphoxide (DMSO) and Obtained From Multiple Donors Buffy Coats, in Patients With Severe Thrombocytopenia
ClinicalTrials.gov ID: NCT02032134
Acronym / sponsor ID: MICE / CUP: G33F1100003000
Conditions: Thrombocytopenia, Primary Thrombocytopenia,Unspecified, Thrombocytopenia Chemotherapy Induced
Keywords: Cryopreserved platelets, thrombocytopenia, DMSO
Phase: Phase 4
Study type: Interventional
Recruitment status: TerminatedReason stopped: recruitment failure within the interval of available CRY BC-PLTs for the study
Enrolment: 5 participants (actual)
Lead sponsor: Napolitano MariasantaUniversity, hospital or other
Collaborators: Regione Sicilia
Study start: Jul 2015
Primary completion: Jun 2016 (actual)
Study completion: Jun 2016 (actual)
First posted: 9 Jan 2014 (estimated)
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.

The main objective of the proposed study is to evaluate the efficacy and safety of platelets, cryopreserved with DMSO using a a new method, in patients with severe thrombocytopenia.

This is the in vivo phase of a study where availability and efficacy of cryopreserved platelets has been assessed in vitro.

Detailed description

Further goals are the registration of any related- adverse event (idiosyncrasy, allergy, toxicity)

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Patients with severe thrombocytopenia	Other	Biological: Transfusion of Pooled Platelets Cryopreserved In DMSOIntervention Patients with severe thrombocytopenia with an active bleeding, in preparation for surgery, or after chemotherapy (prophylaxis of bleeding),will receive transfusion of Pooled Platelets Cryopreserved In DMSO With a New System

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Cryopreserved buffy coat pooled platelets will be infused, after being thawed, in inpatients at U.O. of Hematology -Policlinic "P. Giaccone"-Palermo, falling into one of the following categories:
Stable clinical conditions and no complications with platelets ≤ 10,000/microliter;
With active bleeding and ≤ 20,000 platelets/microliter;
In the presence of: high blood pressure, high fever, rapid drop in platelets, infection, chemotherapy, coagulation abnormalities with ≤ 20,000 platelets/microliter.

Key exclusion criteria (6 listed)

Patients
Suffering from congenital immunodeficiency
Allogeneic bone marrow transplant candidates
Bone Marrow Donors for transplantation
Undergoing bone marrow transplant or

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
Transfusion of Pooled Platelets Cryopreserved In DMSOAlso: New Cryopreservation kit (Promedical ®), Buffy Coat Pooled Platelets Cryopreserved In DMSOPatients with severe thrombocytopenia will be admitted as in patients at the Hematology Unit of the Policlinic "P. Giaccone"- Palermo, if they require platelet transfusion (for bleeding, in preparation for surgery, after chemotherapy); they will receive buffy coat derived cryopreserved platelets,after thawing	Biological	Patients with severe thrombocytopenia

Primary outcome

Efficacy of infused cryopreserved platelets (changes in platelets count will be determined at 1 hour and 24 hours post-transfusion)
Time frame: 1 Hour and 24 hours post-transfusion
To evaluate the availability of cryopreserved platelets after transfusion, the correct count (the number of platelets poured and the body surface, BMI, recipient) of platelets will be determined at 1 hour and 24 hours post transfusion, respectively

1 secondary outcome

Safety of cryopreserved platelets
Time frame: During transfusion and within 48 hours after transfusion

Results and safety

The study was stopped early (reason given: recruitment failure within the interval of available CRY BC-PLTs for the study). Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2017.
Background: PubMed 12493312Wautier JL. Safety and usefulness of autologous cryopreserved platelets. Lancet. 2002 Dec 14;360(9349):1985. doi: 10.1016/S0140-6736(02)11893-9. No abstract available.
Background: PubMed 22781890Liu GQ, Ding HF, Lu XJ, Xu M, Xing J, Zhao X, Han F, Shang YH. [The efficacy and safety of autologous cryopreserved platelet transfusion in management of thrombocytopenia after chemotherapy in hematological malignancy]. Zhonghua Nei Ke Za Zhi. 2012 Mar;51(3):188-91. Chinese.
Background: PubMed 15183900Heal JM, Blumberg N. Optimizing platelet transfusion therapy. Blood Rev. 2004 Sep;18(3):149-65. doi: 10.1016/S0268-960X(03)00057-2.
Background: PubMed 1339584Murphy MF, Brozovic B, Murphy W, Ouwehand W, Waters AH. Guidelines for platelet transfusions. British Committee for Standards in Haematology, Working Party of the Blood Transfusion Task Force. Transfus Med. 1992 Dec;2(4):311-8. doi: 10.1111/j.1365-3148.1992.tb00175.x.
Background: PubMed 22671278Dumont LJ, Cancelas JA, Dumont DF, Siegel AH, Szczepiorkowski ZM, Rugg N, Pratt PG, Worsham DN, Hartman EL, Dunn SK, O'Leary M, Ransom JH, Michael RA, Macdonald VW. A randomized controlled trial evaluating recovery and survival of 6% dimethyl sulfoxide-frozen autologous platelets in healthy volunteers. Transfusion. 2013 Jan;53(1):128-37. doi: 10.1111/j.1537-2995.2012.03735.x. Epub 2012 Jun 7.
Background: PubMed 23003345Meyer E, Delaney M, Lin Y, Morris A, Pavenski K, Tinmouth A, Murphy M, Slichter SJ, Heddle N, Dumont LJ. A reporting guideline for clinical platelet transfusion studies from the BEST Collaborative. Transfusion. 2013 Jun;53(6):1328-34. doi: 10.1111/j.1537-2995.2012.03906.x. Epub 2012 Sep 25.
Background: PubMed 21214584Dumont LJ, Dumont DF, Unger ZM, Siegel A, Szczepiorkowski ZM, Corson JS, Jones MK, Christoffel T, Pellham E, Bailey SL, Slichter SJ; BEST Collaborative. A randomized controlled trial comparing autologous radiolabeled in vivo platelet (PLT) recoveries and survivals of 7-day-stored PLT-rich plasma and buffy coat PLTs from the same subjects. Transfusion. 2011 Jun;51(6):1241-8. doi: 10.1111/j.1537-2995.2010.03007.x. Epub 2011 Jan 7.
Derived from this study: PubMed 31753773Napolitano M, Mancuso S, Raso S, LoCoco L, Arfo PS, De Francisci G, Dieli F, Caccamo N, Reina A, Dolce A, Agliastro R, Siragusa S. Buffy coat-derived platelets cryopreserved using a new method: Results from a pivotal clinical trial on thrombocytopenic patients with acute leukaemia. Transfus Apher Sci. 2019 Dec;58(6):102666. doi: 10.1016/j.transci.2019.10.008. Epub 2019 Nov 5.

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RecruitingPhase 3
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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.

Sergio Siragusa, MD study director

Hematology Unit- University of Palermo

Mariasanta Napolitano, MD principal investigator

Hematology Unit- University of Palermo

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 4 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: stopped early, as of the sponsor's last update on 18 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Italy. 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?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: recruitment failure within the interval of available CRY BC-PLTs for the study). Results are not available in the source record.

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?

Efficacy of infused cryopreserved platelets (changes in platelets count will be determined at 1 hour and 24 hours post-transfusion) (time frame: 1 Hour and 24 hours post-transfusion). 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 NCT02032134, which also lists 8 linked publications.

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 18 Oct 2017.