Thrombotic Thrombocytopenic Purpura, AcquiredROSCAPLINCT05262881
A Retrospective, Observational Study on the Response to Caplacizumab Treatment in aTTP Patients: the Italian Experience (ROSCAPLI)
An observational study of Thrombotic Thrombocytopenic Purpura, Acquired, run by Catholic University of the Sacred Heart at 1 site in Italy.
Trial summary
This study is a multicenter, retrospective, observational study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) treated with plasma exchange (PEX) in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura, Acquired
- How many people
- 50 participants
- Where
- 1 siteItaly
- Study dates
- 1 Mar 2022to 30 Oct 2022
- Phase
- Observational
- 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
Study population: The study population consists of patients treated for acute aTTP episodes in the hematology, internal medicine, and critical care centers between Q4-2019 and Q1-2021 in Italy.
Who may be able to join
- The patients included in this study should have received caplacizumab for treatment in the period between Q4-2019 and the end of February 2021 while the end of follow up observation is scheduled for Q1-2021 (to observe at least one month of post treatment follow up). The diagnosis should be based on either clinical/laboratory parameters inclusive of measurement of ADAMTS13 level <10%) or the PLASMIC score (platelets, lysis, active cancer, stem cell or solid organ transplant, MCV, INR, and creatinine) with intermediate and high risk (sore>5) already computed or retrospectively calculated as previously detailed for centers that did not measure the ADAMTS13 level.
Who cannot join
- Patients treated with uncertain aTTP diagnosis according to the above inclusion criteria and manifesting clinical signs like aTTP but characterized by a different pathogenesis (e.g. cancer, sepsis)
Trial locations and contacts
1 site in 1 country.
Italy1 site
Fondazione Policlinico Universitario Agostino Gemelli, IRCCS
Rome, 00168
About this study
This study is a multicenter, retrospective, observational study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) treated with plasma exchange (PEX) in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021. The retrospective study will measure: Age, sex, BMI, blood pressure at diagnosis, Platelet count at diagnosis and at the follow up visits, Hb level at diagnosis at the follow up visits, White blood cell count at diagnosis at the follow up visits, Creatinine at diagnosis at the follow up visits, schistocytes count at diagnosis at the follow up visits, LDH at diagnosis at the follow up visits, Coombs' assay at diagnosis, alanine-leucine-amino-transferase (ALT) at diagnosis at the follow up visits, total bilirubin at diagnosis at the follow up visits, Troponin above ULN at any point, ADAMTS13 activity (where measured) at diagnosis at the follow up visits, Anti-ADAMTS13 antibodies (where measured) at diagnosis at the follow up visits.
The primary objective in this study is the description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab , in addition to PEX and immunosuppression in the real-world setting. The secondary objectives include: number of exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of therapy; rate of relapse, defined as a TTP event occurring more than 30 days after the end of daily plasma exchange; refractoriness; defined by the lack of a doubling of platelet count after 4 days of treatment and a lactate dehydrogenase level that remained above the upper limit of the normal range, TTP-related mortality and evaluation of adverse events.
Read the detailed description
This study is a multicenter, retrospective, observational study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) treated with plasma exchange (PEX) in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021.
Study design
The retrospective study will measure the following vital, clinical chemistry, hematological, and hemostatic parameters, according to the consensus guidelines of aTTP diagnosis and as a part of routine diagnostic work-up:
- Age, sex, BMI, blood pressure at diagnosis
- Platelet count at diagnosis and at the follow up visits
- Hb level at diagnosis at the follow up visits
- White blood cell count at diagnosis at the follow up visits
- Creatinine at diagnosis at the follow up visits
- schistocytes count at diagnosis at the follow up visits
- LDH at diagnosis at the follow up visits
- Coombs' assay at diagnosis
- alanine-leucine-amino-transferase (ALT) at diagnosis at the follow up visits
- total bilirubin at diagnosis at the follow up visits
- Troponin above ULN at any point
- ADAMTS13 activity (where measured) at diagnosis at the follow up visits
- Anti-ADAMTS13 antibodies (where measured) at diagnosis at the follow up visits The clinical signs will be parametrized by the PLASMIC score and the Glasgow score, where calculated in the various centers. The PLASMIC score and the Glasgow score will be evaluated according to the obtained values (0-4, 5-6, and 7; <13 and 13-15, respectively). All the clinical and laboratory parameters will be taken in each center and reported in the CRF.
OBJECTIVES The primary objective in this study is the description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab , in addition to PEX and immunosuppression in the real-world setting. The Secondary objectives include: Number of exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of therapy; Rate of relapse, defined as a TTP event occurring more than 30 days after the end of daily plasma exchange;Refractoriness; defined by the lack of a doubling of platelet count after 4 days of treatment and a lactate dehydrogenase level that remained above the upper limit of the normal range, TTP-related mortality and evaluation of adverse events.
Data collection and management A customized eCRF (RedCap) will be created for the study and only the principal investigator and collaborating investigators of the center will have access to patient medical records during the study.
STATISTICAL CONSIDERATIONS Qualitative variables will be expressed by absolute and percentage frequency. Quantitative variables will be previously assessed for their distribution by the Shapiro-Wilk test. Normally distributed data will be described by mean and standard deviation (SD), whilst non-normally distributed variable by median and interquartile range (IQR).
INFORMED CONSENT Informed consent form will be collected for all except for all patients whom death is ascertained
- Medical subject headings
- Thrombotic thrombocytopenic purpura, acquired
What is being measured
The primary outcome is the main question the study is designed to answer.
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets
Platelet response was defined as recovery of platelets ≥ 150,000/µL.
Measured: 18 months
Secondary outcomes (1)
study of critical parameters
- Number and percentage of subjects with TTP-Related Death
- Number and percentage of subjects with relapse of TTP, defined as a TTP event occurring more than 30 days after the end of daily plasma exchanges
- assessment of all SAEs of thromboembolic events
- Number and percentage of subjects with exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of daily plasma exchanges that required reinitiation of daily exchanges
- Number and percentage of subjects with refractory TTP (); and the time to normalization (i.e., to a level below the defined upper limit of the normal range) of three organ-damage markers (LDH, cardiac troponin I, and serum creatinine).
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2022
- Study start1 Mar 2022 (estimated)
- Primary completion30 Sep 2022 (estimated)
- Study completion30 Oct 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (18)Papers the sponsor has linked to this study.
- Other trials for Thrombotic Thrombocytopenic Purpura, AcquiredStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Catholic University of the Sacred Heart
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Raimondo De Cristofaro, Catholic University of the Sacred Heart
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05262881
- Sponsor's protocol ID
- 4305
- Acronym
- ROSCAPLI
- Status last verified
- Feb 2022
- First submitted
- 21 Feb 2022
- Last updated
- 2 Mar 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Scully M, Hunt BJ, Benjamin S, Liesner R, Rose P, Peyvandi F, Cheung B, Machin SJ; British Committee for Standards in Haematology. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012 Aug;158(3):323-35. doi: 10.1111/j.1365-2141.2012.09167.x. Epub 2012 May 25. No abstract available.
PubMed 22624596Rizzo C, Rizzo S, Scire E, Di Bona D, Ingrassia C, Franco G, Bono R, Quintini G, Caruso C. Thrombotic thrombocytopenic purpura: a review of the literature in the light of our experience with plasma exchange. Blood Transfus. 2012 Oct;10(4):521-32. doi: 10.2450/2012.0122-11. Epub 2012 Jun 27. No abstract available.
PubMed 22790258Bell WR, Braine HG, Ness PM, Kickler TS. Improved survival in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Clinical experience in 108 patients. N Engl J Med. 1991 Aug 8;325(6):398-403. doi: 10.1056/NEJM199108083250605.
PubMed 2062331Nguyen L, Li X, Duvall D, Terrell DR, Vesely SK, George JN. Twice-daily plasma exchange for patients with refractory thrombotic thrombocytopenic purpura: the experience of the Oklahoma Registry, 1989 through 2006. Transfusion. 2008 Feb;48(2):349-57. doi: 10.1111/j.1537-2995.2007.01530.x. Epub 2007 Nov 19.
PubMed 18028271Balduini CL, Gugliotta L, Luppi M, Laurenti L, Klersy C, Pieresca C, Quintini G, Iuliano F, Re R, Spedini P, Vianelli N, Zaccaria A, Pogliani EM, Musso R, Bobbio Pallavicini E, Quarta G, Galieni P, Fragasso A, Casella G, Noris P, Ascari E; Italian TTP Study Group. High versus standard dose methylprednisolone in the acute phase of idiopathic thrombotic thrombocytopenic purpura: a randomized study. Ann Hematol. 2010 Jun;89(6):591-6. doi: 10.1007/s00277-009-0877-5. Epub 2009 Dec 23.
PubMed 20033409Ziman A, Mitri M, Klapper E, Pepkowitz SH, Goldfinger D. Combination vincristine and plasma exchange as initial therapy in patients with thrombotic thrombocytopenic purpura: one institution's experience and review of the literature. Transfusion. 2005 Jan;45(1):41-9. doi: 10.1111/j.1537-2995.2005.03146.x.
PubMed 15647017Beloncle F, Buffet M, Coindre JP, Munoz-Bongrand N, Malot S, Pene F, Mira JP, Galicier L, Guidet B, Baudel JL, Subra JF, Tanguy-Schmidt A, Pourrat J, Azoulay E, Veyradier A, Coppo P; Thrombotic Microangiopathies Reference Center. Splenectomy and/or cyclophosphamide as salvage therapies in thrombotic thrombocytopenic purpura: the French TMA Reference Center experience. Transfusion. 2012 Nov;52(11):2436-44. doi: 10.1111/j.1537-2995.2012.03578.x. Epub 2012 Mar 8.
PubMed 22404639Sayani FA, Abrams CS. How I treat refractory thrombotic thrombocytopenic purpura. Blood. 2015 Jun 18;125(25):3860-7. doi: 10.1182/blood-2014-11-551580. Epub 2015 Mar 17.
PubMed 25784681Lim W, Vesely SK, George JN. The role of rituximab in the management of patients with acquired thrombotic thrombocytopenic purpura. Blood. 2015 Mar 5;125(10):1526-31. doi: 10.1182/blood-2014-10-559211. Epub 2015 Jan 8. No abstract available.
PubMed 25573992Vazquez-Mellado A, Pequeno-Luevano M, Cantu-Rodriguez OG, Villarreal-Martinez L, Jaime-Perez JC, Gomez-De-Leon A, De La Garza-Salazar F, Gonzalez-Llano O, Colunga-Pedraza P, Sotomayor-Duque G, Gomez-Almaguer D. More about low-dose rituximab and plasma exchange as front-line therapy for patients with thrombotic thrombocytopenic purpura. Hematology. 2016 Jun;21(5):311-6. doi: 10.1080/10245332.2015.1133008. Epub 2016 Feb 24.
PubMed 26907228Scully M, McDonald V, Cavenagh J, Hunt BJ, Longair I, Cohen H, Machin SJ. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011 Aug 18;118(7):1746-53. doi: 10.1182/blood-2011-03-341131. Epub 2011 Jun 2.
PubMed 21636861Page EE, Kremer Hovinga JA, Terrell DR, Vesely SK, George JN. Rituximab reduces risk for relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2016 Jun 16;127(24):3092-4. doi: 10.1182/blood-2016-03-703827. Epub 2016 Apr 8. No abstract available.
PubMed 27060171Hie M, Gay J, Galicier L, Provot F, Presne C, Poullin P, Bonmarchand G, Wynckel A, Benhamou Y, Vanhille P, Servais A, Bordessoule D, Coindre JP, Hamidou M, Vernant JP, Veyradier A, Coppo P; French Thrombotic Microangiopathies Reference Centre. Preemptive rituximab infusions after remission efficiently prevent relapses in acquired thrombotic thrombocytopenic purpura. Blood. 2014 Jul 10;124(2):204-10. doi: 10.1182/blood-2014-01-550244. Epub 2014 May 28.
PubMed 24869941Rottenstreich A, Hochberg-Klein S, Rund D, Kalish Y. The role of N-acetylcysteine in the treatment of thrombotic thrombocytopenic purpura. J Thromb Thrombolysis. 2016 May;41(4):678-83. doi: 10.1007/s11239-015-1259-6.
PubMed 26245827Eskazan AE. Bortezomib therapy in patients with relapsed/refractory acquired thrombotic thrombocytopenic purpura. Ann Hematol. 2016 Oct;95(11):1751-6. doi: 10.1007/s00277-016-2804-x. Epub 2016 Sep 3.
PubMed 27590601
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective, Observational Study on the Response to Caplacizumab Treatment in aTTP Patients: the Italian Experience (ROSCAPLI)
- ClinicalTrials.gov ID
- NCT05262881
- Acronym / sponsor ID
- ROSCAPLI / 4305
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Catholic University of the Sacred HeartUniversity, hospital or other
- Study start
- 1 Mar 2022 (estimated)
- Primary completion
- 30 Sep 2022 (estimated)
- Study completion
- 30 Oct 2022 (estimated)
- First posted
- 2 Mar 2022 (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.
This study is a multicenter, retrospective, observational study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) treated with plasma exchange (PEX) in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021. The retrospective study will measure: Age, sex, BMI, blood pressure at diagnosis, Platelet count at diagnosis and at the follow up visits, Hb level at diagnosis at the follow up visits, White blood cell count at diagnosis at the follow up visits, Creatinine at diagnosis at the follow up visits, schistocytes count at diagnosis at the follow up visits, LDH at diagnosis at the follow up visits, Coombs' assay at diagnosis, alanine-leucine-amino-transferase (ALT) at diagnosis at the follow up visits, total bilirubin at diagnosis at the follow up visits, Troponin above ULN at any point, ADAMTS13 activity (where measured) at diagnosis at the follow up visits, Anti-ADAMTS13 antibodies (where measured) at diagnosis at the follow up visits.
The primary objective in this study is the description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab , in addition to PEX and immunosuppression in the real-world setting. The secondary objectives include: number of exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of therapy; rate of relapse, defined as a TTP event occurring more than 30 days after the end of daily plasma exchange; refractoriness; defined by the lack of a doubling of platelet count after 4 days of treatment and a lactate dehydrogenase level that remained above the upper limit of the normal range, TTP-related mortality and evaluation of adverse events.
Detailed description
This study is a multicenter, retrospective, observational study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) treated with plasma exchange (PEX) in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021.
Study design
The retrospective study will measure the following vital, clinical chemistry, hematological, and hemostatic parameters, according to the consensus guidelines of aTTP diagnosis and as a part of routine diagnostic work-up:
- Age, sex, BMI, blood pressure at diagnosis
- Platelet count at diagnosis and at the follow up visits
- Hb level at diagnosis at the follow up visits
- White blood cell count at diagnosis at the follow up visits
- Creatinine at diagnosis at the follow up visits
- schistocytes count at diagnosis at the follow up visits
- LDH at diagnosis at the follow up visits
- Coombs' assay at diagnosis
- alanine-leucine-amino-transferase (ALT) at diagnosis at the follow up visits
- total bilirubin at diagnosis at the follow up visits
- Troponin above ULN at any point
- ADAMTS13 activity (where measured) at diagnosis at the follow up visits
- Anti-ADAMTS13 antibodies (where measured) at diagnosis at the follow up visits The clinical signs will be parametrized by the PLASMIC score and the Glasgow score, where calculated in the various centers. The PLASMIC score and the Glasgow score will be evaluated according to the obtained values (0-4, 5-6, and 7; <13 and 13-15, respectively). All the clinical and laboratory parameters will be taken in each center and reported in the CRF.
OBJECTIVES The primary objective in this study is the description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab , in addition to PEX and immunosuppression in the real-world setting. The Secondary objectives include: Number of exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of therapy; Rate of relapse, defined as a TTP event occurring more than 30 days after the end of daily plasma exchange;Refractoriness; defined by the lack of a doubling of platelet count after 4 days of treatment and a lactate dehydrogenase level that remained above the upper limit of the normal range, TTP-related mortality and evaluation of adverse events.
Data collection and management A customized eCRF (RedCap) will be created for the study and only the principal investigator and collaborating investigators of the center will have access to patient medical records during the study.
STATISTICAL CONSIDERATIONS Qualitative variables will be expressed by absolute and percentage frequency. Quantitative variables will be previously assessed for their distribution by the Shapiro-Wilk test. Normally distributed data will be described by mean and standard deviation (SD), whilst non-normally distributed variable by median and interquartile range (IQR).
INFORMED CONSENT Informed consent form will be collected for all except for all patients whom death is ascertained
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population consists of patients treated for acute aTTP episodes in the hematology, internal medicine, and critical care centers between Q4-2019 and Q1-2021 in Italy.
Key inclusion criteria (1 listed)
- The patients included in this study should have received caplacizumab for treatment in the period between Q4-2019 and the end of February 2021 while the end of follow up observation is scheduled for Q1-2021 (to observe at least one month of post treatment follow up). The diagnosis should be based on either clinical/laboratory parameters inclusive of measurement of ADAMTS13 level <10%) or the PLASMIC score (platelets, lysis, active cancer, stem cell or solid organ transplant, MCV, INR, and creatinine) with intermediate and high risk (sore>5) already computed or retrospectively calculated as previously detailed for centers that did not measure the ADAMTS13 level.
Key exclusion criteria (1 listed)
- Patients treated with uncertain aTTP diagnosis according to the above inclusion criteria and manifesting clinical signs like aTTP but characterized by a different pathogenesis (e.g. cancer, sepsis)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets
Time frame: 18 months
Platelet response was defined as recovery of platelets ≥ 150,000/µL.
1 secondary outcome
study of critical parameters
Time frame: 18 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2022.
- Results publication: PubMed 22624596Scully M, Hunt BJ, Benjamin S, Liesner R, Rose P, Peyvandi F, Cheung B, Machin SJ; British Committee for Standards in Haematology. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012 Aug;158(3):323-35. doi: 10.1111/j.1365-2141.2012.09167.x. Epub 2012 May 25. No abstract available.
- Results publication: PubMed 22790258Rizzo C, Rizzo S, Scire E, Di Bona D, Ingrassia C, Franco G, Bono R, Quintini G, Caruso C. Thrombotic thrombocytopenic purpura: a review of the literature in the light of our experience with plasma exchange. Blood Transfus. 2012 Oct;10(4):521-32. doi: 10.2450/2012.0122-11. Epub 2012 Jun 27. No abstract available.
- Results publication: PubMed 2062331Bell WR, Braine HG, Ness PM, Kickler TS. Improved survival in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Clinical experience in 108 patients. N Engl J Med. 1991 Aug 8;325(6):398-403. doi: 10.1056/NEJM199108083250605.
- Results publication: PubMed 18028271Nguyen L, Li X, Duvall D, Terrell DR, Vesely SK, George JN. Twice-daily plasma exchange for patients with refractory thrombotic thrombocytopenic purpura: the experience of the Oklahoma Registry, 1989 through 2006. Transfusion. 2008 Feb;48(2):349-57. doi: 10.1111/j.1537-2995.2007.01530.x. Epub 2007 Nov 19.
- Results publication: PubMed 20033409Balduini CL, Gugliotta L, Luppi M, Laurenti L, Klersy C, Pieresca C, Quintini G, Iuliano F, Re R, Spedini P, Vianelli N, Zaccaria A, Pogliani EM, Musso R, Bobbio Pallavicini E, Quarta G, Galieni P, Fragasso A, Casella G, Noris P, Ascari E; Italian TTP Study Group. High versus standard dose methylprednisolone in the acute phase of idiopathic thrombotic thrombocytopenic purpura: a randomized study. Ann Hematol. 2010 Jun;89(6):591-6. doi: 10.1007/s00277-009-0877-5. Epub 2009 Dec 23.
- Results publication: PubMed 15647017Ziman A, Mitri M, Klapper E, Pepkowitz SH, Goldfinger D. Combination vincristine and plasma exchange as initial therapy in patients with thrombotic thrombocytopenic purpura: one institution's experience and review of the literature. Transfusion. 2005 Jan;45(1):41-9. doi: 10.1111/j.1537-2995.2005.03146.x.
- Results publication: PubMed 22404639Beloncle F, Buffet M, Coindre JP, Munoz-Bongrand N, Malot S, Pene F, Mira JP, Galicier L, Guidet B, Baudel JL, Subra JF, Tanguy-Schmidt A, Pourrat J, Azoulay E, Veyradier A, Coppo P; Thrombotic Microangiopathies Reference Center. Splenectomy and/or cyclophosphamide as salvage therapies in thrombotic thrombocytopenic purpura: the French TMA Reference Center experience. Transfusion. 2012 Nov;52(11):2436-44. doi: 10.1111/j.1537-2995.2012.03578.x. Epub 2012 Mar 8.
- Results publication: PubMed 25784681Sayani FA, Abrams CS. How I treat refractory thrombotic thrombocytopenic purpura. Blood. 2015 Jun 18;125(25):3860-7. doi: 10.1182/blood-2014-11-551580. Epub 2015 Mar 17.
- Results publication: PubMed 25573992Lim W, Vesely SK, George JN. The role of rituximab in the management of patients with acquired thrombotic thrombocytopenic purpura. Blood. 2015 Mar 5;125(10):1526-31. doi: 10.1182/blood-2014-10-559211. Epub 2015 Jan 8. No abstract available.
- Results publication: PubMed 26907228Vazquez-Mellado A, Pequeno-Luevano M, Cantu-Rodriguez OG, Villarreal-Martinez L, Jaime-Perez JC, Gomez-De-Leon A, De La Garza-Salazar F, Gonzalez-Llano O, Colunga-Pedraza P, Sotomayor-Duque G, Gomez-Almaguer D. More about low-dose rituximab and plasma exchange as front-line therapy for patients with thrombotic thrombocytopenic purpura. Hematology. 2016 Jun;21(5):311-6. doi: 10.1080/10245332.2015.1133008. Epub 2016 Feb 24.
- Results publication: PubMed 21636861Scully M, McDonald V, Cavenagh J, Hunt BJ, Longair I, Cohen H, Machin SJ. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011 Aug 18;118(7):1746-53. doi: 10.1182/blood-2011-03-341131. Epub 2011 Jun 2.
- Results publication: PubMed 27060171Page EE, Kremer Hovinga JA, Terrell DR, Vesely SK, George JN. Rituximab reduces risk for relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2016 Jun 16;127(24):3092-4. doi: 10.1182/blood-2016-03-703827. Epub 2016 Apr 8. No abstract available.
- European-Medicines-Agency. Cablivi (caplacizumab): summary of product characteristics. Document of the European Medicines Agency. 2018;Link provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thrombotic Thrombocytopenic Purpura, Acquired, run by Catholic University of the Sacred Heart at 1 site in Italy.
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 1 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 2 Mar 2022. 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.
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?
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?
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets (time frame: 18 months). 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 NCT05262881, which also lists 18 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 2 Mar 2022.