Thrombotic Thrombocytopenic PurpuraTTP registryNCT01257269
Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
An observational study of Thrombotic Thrombocytopenic Purpura and Congenital Thrombotic Thrombocytopenic Purpura, run by Insel Gruppe AG, University Hospital Bern at 7 sites in 7 countries.
Trial summary
Hereditary thrombotic thrombocytopenic purpura (Upshaw-Schulman syndrome) is a rare disorder characterized by thrombocytopenia as a result of platelet consumption, microangiopathic hemolytic anemia, occlusion of the microvasculature with von Willebrand factor-platelet-thrombic and ischemic end organ damage. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura, Familial Thrombotic Thrombocytopenic Purpura
- What is being tested?
- Observation
- How many people
- 450 participants
- Where
- 7 sitesin 7 countries
- Study dates
- Oct 2006to Oct 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with either confirmed or with suspected hereditary TTP and their family members are eligible for enrollment
Who may be able to join
- Severe ADAMTS13 deficiency ( ≤ 10% activity) and no ADAMTS 13 inhibitor on two or more occasions at least one month apart
- Being a family member of a confirmed or suspected patient
- Molecular analysis of ADAMTS13 gene with one or more mutations and/or positive infusion trial (full recovered ADAMTS13 activity after infused fresh frozen plasma (FFP) with a plasma half-life of 2-4 days)
Who cannot join
None listed.
Trial locations and contacts
7 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 7 countries
United States1 site, 1 recruiting
- Recruiting
University of Oklahoma Health Sciences Center, Department of Medicine, PO Box 26901
Oklahoma City, Oklahoma, 73126-0901
James N. George, M.D., 405-271-4222, james-george@ouhsc.edu
James N. George, M.D.
Austria1 site, 1 recruiting
- Recruiting
Medical University of Vienna, Department of Medicine 1, Div. Hematology and Hemostasis Waehringer Guertel 18-20
Vienna, A-1090
Paul N. Knoebl, M.D., +43 1 40400 4410, paul.knoebl@meduniwien.ac.at
Paul N. Knoebl, M.D.
Czechia1 site, 1 recruiting
- Recruiting
Institute of Hematology and Blood Transfusion, Coagulation Laboratory, U nemocnice 1
Prague, CZ-12820
Ingrid Hrachovinova, Ph.D., +420 2 2197 271, Ingrid.Hrachovinova@uhkt.cz
Ingrid Hrachovinova, Ph.D.
Germany1 site, 1 recruiting
- Not yet recruiting
University Medical Center Hamburg-Eppendorf, Department of Pediatric Hematology and Oncology, Martinistr 52
Hamburg, D-20246
Reinhard Schneppenheim, M.D., Ph.D., +49 40 7410 54270, schneppenheim@uke.de
Reinhard Schneppenheim, M.D., Ph.D.
Japan1 site, 1 recruiting
- Recruiting
Nara Medical University, Department of Blood Transfusion Medicine, Shijyo-cho 840
Kashihara, Nara, 634-8522
Yoshihiro Fujimura, M.D., +81 744 22 3051 ext. 3289, yoshifuji325@naramed-u.ac.jp
Yoshihiro Fujimura, M.D.
Norway1 site, 1 recruiting
- Recruiting
Trondheim University St Olavs Hospital, Department of Hematology, PO Box 3250 Sluppen
Trondheim, NO-7006
Petter Quist-Paulsen, M.D., Ph.D., +47 815 55 850, Petter.Quist-Paulsen@stolav.no
Anne-Sophie von Krogh, M.D., +47 815 55 850, Anne-Sophie.von.Krogh@stolav.no
Switzerland1 site, 1 recruiting
- Recruiting
University Clinic of Hematology and Central Hematology Laboratory, Bern University Hospital and the University of Bern, Inselspital
Bern, 3010
Johanna A Kremer Hovinga, MD, +41 31 632 02 65, johanna.kremer@insel.ch
Isabella Aebi, BMA, +41 31 632 77 16, isabella.aebi-huber@insel.ch
About this study
Official title: Thrombotic Thrombocytopenic Purpura Registry - A Prospective Observational Study for Patients Suffering From Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
Hereditary thrombotic thrombocytopenic purpura (Upshaw-Schulman syndrome) is a rare disorder characterized by thrombocytopenia as a result of platelet consumption, microangiopathic hemolytic anemia, occlusion of the microvasculature with von Willebrand factor-platelet-thrombic and ischemic end organ damage. The underlying patho-mechanism is a severe congenital ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, 13) deficiency which is the result of compound heterozygous or homozygous ADAMTS13 gene mutations.
Although considered a monogenic disorder the clinical presentation in Upshaw-Schulman syndrome patients varies considerably without an apparent genotype-phenotype correlation. In 2006 we have initiated a registry for patients with Upshaw-Schulman syndrome and their family members to identify possible triggers of acute bouts of TTP, to document individual clinical courses and treatment requirements as well as possible side effects of long standing plasma substitution, e.g. alloantibody formation or viral infections.
Read the detailed description
Background
Thrombocytopenia and microangiopathic hemolytic anemia together with a severely deficient ADAMTS13 activity confirm the diagnosis of acute thrombotic thrombocytopenic purpura (TTP). Today two forms of classical TTP are distinguished. The acquired form is caused by circulating auto-antibodies, mainly Immunoglobulin G (IgG), inhibiting ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, 13) activity. In contrast, hereditary TTP, also known as Upshaw-Schulman syndrome (USS); #274150 Online Mendelian Inheritance in Man (OMIM), is the result of severe constitutional deficiency of ADAMTS13 due to compound heterozygous or homozygous mutations in the ADAMTS13 gene.
The clinical course of USS is variable with rather mild courses in some of the patients requiring plasma infusions only in special situations (i.e. pregnancy), while in others severe courses with important sequelae and even death in early childhood occur. The reasons for the variable clinical presentation and treatment requirements have not been elucidated. It seems likely, that additional, hitherto unidentified factors besides severe ADAMTS13 deficiency modify the clinical course.
At present, the clinical symptoms and laboratory values on which to base treatment regimens for hereditary TTP are poorly understood. Furthermore, increasing awareness of hereditary TTP results in rising numbers of patients in need of treatment and/or prophylaxis. However, currently very little is known on side effects of long standing plasma substitution. Alloimmunization with the formation of antibodies acting as inhibitors of treatment are well known in other congenital coagulation factor deficiencies (e.g. hemophilia A), but so far no case of treated hereditary TTP with subsequent antibody formation has been reported. It is the aim of the hereditary TTP Registry to provide information on the clinical course of the disease in as many patients as possible and therefore help to establish recommendations on the necessity, modalities, and risks of prophylactic plasma therapy in patients with hereditary TTP. Furthermore, it will help to gain detailed insight into triggers and risk factors of acute bouts of TTP.
Moreover, the hereditary TTP Registry will provide information for family members on their risk to develop TTP-like or TTP-related disorders.
Objective
Primary objective: Collection of as much information as possible on the clinical presentation, disease course, disease-modifying factors, and treatment modalities of patients suffering from hereditary thrombotic thrombocytopenic purpura (TTP).
Secondary objective: To document potential adversary effects of (long-term) plasma treatment in patients with hereditary thrombotic thrombocytopenic purpura (TTP).
Methods
The TTP Registry is designed to collect both retrospective and prospective clinical, molecular, and observational data on patients with confirmed or suspected hereditary TTP. Additionally, the Registry will collect data from family members of TTP patients enrolled in the Registry. The Registry will enroll as many confirmed or suspected hereditary TTP patients and their family members as possible; there is no cap on enrollment. The Registry enrollment and follow-up periods are open-ended. The endpoints for patients with confirmed or suspected hereditary TTP and family members are death or withdrawal of consent.
- Conditions
- Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura, Familial Thrombotic Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Purpura, Congenital, Upshaw-Schulman Syndrome
- Being tested
- Observation
- Keywords
- Thrombotic thrombocytopenic purpura, ADAMTS13, Von Willebrand factor, Von Willebrand factor cleaving protease, Thrombocytopenia, Hemolytic anemia
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic, Thrombocytopenia, Anemia, Hemolytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ObservationOther
No interventions planned: treatment of patients at the discretion of the treating/responsible physician
| Study group | Role | What they receive |
|---|---|---|
| 1 | Patients with confirmed hereditary TTP due to congenital ADAMTS13 deficiency Receives: Other: Observation | |
| 2 | Family members of patients with confirmed hereditary TTP Receives: Other: Observation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical presentation and disease course in hereditary TTP
Measured: every year until death
Secondary outcomes (5)
Identification of disease-modifying factors, including genotype-phenotype correlation
Measured: every year until death
Treatment requirements in hereditary TTP patients
Measured: every year until death
Documentation of potential adversary effects of (long-term) plasma treatment
Measured: every year until death
Mortality of hereditary TTP
Measured: every year until death
Clinical course in family members
Measured: every year until death
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2010 (estimated)
- Study startOct 2006
- Primary completionOct 2030 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Thrombotic Thrombocytopenic PurpuraStudies 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
- Case only
- Time perspective
- Other
- Enrolment
- 450 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Insel Gruppe AG, University Hospital Bern
- Sponsor type
- University, hospital or other
- Collaborators
- Swiss National Science Foundation, Mach Gaensslen Foundation, Baxalta Innovations GmbH, Wien, Austria
- Responsible party
- Sponsor
- Investigators
- Johanna A Kremer Hovinga, MD, Study chair, University Clinic of Hematology and Central Hematology Laboratory, Bern University Hospital and the University of Bern, InselspitalBernhard Lämmle, M.D., Study chair, University Medical Center, Center for Thrombosis and Hemostasis, Mainz, GermanyYoshihiro Fujimura, M.D., Study chair, Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, JapanIngrid Hrachovinova, Ph.D., Study chair, Institute of Hematology and Blood Transfusion, Coagulation Laboratory, Prague, Czech RepublicPetter Quist-Paulsen, M.D., Ph.D., Study chair, Department of Hematology, St Olavs Hospital, 7006 Trondheim, NorwayReinhard Schneppenheim, M.D., Ph.D., Study chair, Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyJames N. George, MD, Study chair, University of Oklahoma Health Sciences Center, Department of Medicine, United States of AmericaPaul N Knoebl, MD, Study chair, Medical University of Vienna, Div. Hematology and Hemostasis, Austria
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01257269
- Sponsor's protocol ID
- 031/06
- Acronym
- TTP registry
- Status last verified
- Oct 2023
- First submitted
- 6 Dec 2010
- Last updated
- 11 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
Publications
Tarasco E, Butikofer L, Friedman KD, George JN, Hrachovinova I, Knobl PN, Matsumoto M, von Krogh AS, Aebi-Huber I, Cermakova Z, Gorska-Kosicka M, Jalowiec KA, Largiader CR, Prohaszka Z, Sinkovits G, Windyga J, Lammle B, Kremer Hovinga JA. Annual incidence and severity of acute episodes in hereditary thrombotic thrombocytopenic purpura. Blood. 2021 Jun 24;137(25):3563-3575. doi: 10.1182/blood.2020009801.
PubMed 33649760van Dorland HA, Taleghani MM, Sakai K, Friedman KD, George JN, Hrachovinova I, Knobl PN, von Krogh AS, Schneppenheim R, Aebi-Huber I, Butikofer L, Largiader CR, Cermakova Z, Kokame K, Miyata T, Yagi H, Terrell DR, Vesely SK, Matsumoto M, Lammle B, Fujimura Y, Kremer Hovinga JA; Hereditary TTP Registry. The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017. Haematologica. 2019 Oct;104(10):2107-2115. doi: 10.3324/haematol.2019.216796. Epub 2019 Feb 21.
PubMed 30792199Fan X, Kremer Hovinga JA, Shirotani-Ikejima H, Eura Y, Hirai H, Honda S, Kokame K, Taleghani MM, von Krogh AS, Yoshida Y, Fujimura Y, Lammle B, Miyata T. Genetic variations in complement factors in patients with congenital thrombotic thrombocytopenic purpura with renal insufficiency. Int J Hematol. 2016 Mar;103(3):283-91. doi: 10.1007/s12185-015-1933-7. Epub 2016 Feb 1.
PubMed 26830967Lammle B. VWF and complement. Blood. 2015 Feb 5;125(6):896-8. doi: 10.1182/blood-2014-12-612556. No abstract available.
PubMed 25655456Mansouri Taleghani M, von Krogh AS, Fujimura Y, George JN, Hrachovinova I, Knobl PN, Quist-Paulsen P, Schneppenheim R, Lammle B, Kremer Hovinga JA. Hereditary thrombotic thrombocytopenic purpura and the hereditary TTP registry. Hamostaseologie. 2013 May 29;33(2):138-43. doi: 10.5482/HAMO-13-04-0026.
PubMed 23715103
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thrombotic Thrombocytopenic Purpura Registry - A Prospective Observational Study for Patients Suffering From Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
- ClinicalTrials.gov ID
- NCT01257269
- Acronym / sponsor ID
- TTP registry / 031/06
- Conditions
- Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura, Familial Thrombotic Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Purpura, Congenital, Upshaw-Schulman Syndrome
- Keywords
- Thrombotic thrombocytopenic purpura, ADAMTS13, Von Willebrand factor, Von Willebrand factor cleaving protease, Thrombocytopenia, Hemolytic anemia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 450 participants (estimated)
- Lead sponsor
- Insel Gruppe AG, University Hospital BernUniversity, hospital or other
- Collaborators
- Swiss National Science Foundation, Mach Gaensslen Foundation, Baxalta Innovations GmbH, Wien, Austria
- Study start
- Oct 2006
- Primary completion
- Oct 2030 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 9 Dec 2010 (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.
Hereditary thrombotic thrombocytopenic purpura (Upshaw-Schulman syndrome) is a rare disorder characterized by thrombocytopenia as a result of platelet consumption, microangiopathic hemolytic anemia, occlusion of the microvasculature with von Willebrand factor-platelet-thrombic and ischemic end organ damage. The underlying patho-mechanism is a severe congenital ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, 13) deficiency which is the result of compound heterozygous or homozygous ADAMTS13 gene mutations.
Although considered a monogenic disorder the clinical presentation in Upshaw-Schulman syndrome patients varies considerably without an apparent genotype-phenotype correlation. In 2006 we have initiated a registry for patients with Upshaw-Schulman syndrome and their family members to identify possible triggers of acute bouts of TTP, to document individual clinical courses and treatment requirements as well as possible side effects of long standing plasma substitution, e.g. alloantibody formation or viral infections.
Detailed description
Background
Thrombocytopenia and microangiopathic hemolytic anemia together with a severely deficient ADAMTS13 activity confirm the diagnosis of acute thrombotic thrombocytopenic purpura (TTP). Today two forms of classical TTP are distinguished. The acquired form is caused by circulating auto-antibodies, mainly Immunoglobulin G (IgG), inhibiting ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, 13) activity. In contrast, hereditary TTP, also known as Upshaw-Schulman syndrome (USS); #274150 Online Mendelian Inheritance in Man (OMIM), is the result of severe constitutional deficiency of ADAMTS13 due to compound heterozygous or homozygous mutations in the ADAMTS13 gene.
The clinical course of USS is variable with rather mild courses in some of the patients requiring plasma infusions only in special situations (i.e. pregnancy), while in others severe courses with important sequelae and even death in early childhood occur. The reasons for the variable clinical presentation and treatment requirements have not been elucidated. It seems likely, that additional, hitherto unidentified factors besides severe ADAMTS13 deficiency modify the clinical course.
At present, the clinical symptoms and laboratory values on which to base treatment regimens for hereditary TTP are poorly understood. Furthermore, increasing awareness of hereditary TTP results in rising numbers of patients in need of treatment and/or prophylaxis. However, currently very little is known on side effects of long standing plasma substitution. Alloimmunization with the formation of antibodies acting as inhibitors of treatment are well known in other congenital coagulation factor deficiencies (e.g. hemophilia A), but so far no case of treated hereditary TTP with subsequent antibody formation has been reported. It is the aim of the hereditary TTP Registry to provide information on the clinical course of the disease in as many patients as possible and therefore help to establish recommendations on the necessity, modalities, and risks of prophylactic plasma therapy in patients with hereditary TTP. Furthermore, it will help to gain detailed insight into triggers and risk factors of acute bouts of TTP.
Moreover, the hereditary TTP Registry will provide information for family members on their risk to develop TTP-like or TTP-related disorders.
Objective
Primary objective: Collection of as much information as possible on the clinical presentation, disease course, disease-modifying factors, and treatment modalities of patients suffering from hereditary thrombotic thrombocytopenic purpura (TTP).
Secondary objective: To document potential adversary effects of (long-term) plasma treatment in patients with hereditary thrombotic thrombocytopenic purpura (TTP).
Methods
The TTP Registry is designed to collect both retrospective and prospective clinical, molecular, and observational data on patients with confirmed or suspected hereditary TTP. Additionally, the Registry will collect data from family members of TTP patients enrolled in the Registry. The Registry will enroll as many confirmed or suspected hereditary TTP patients and their family members as possible; there is no cap on enrollment. The Registry enrollment and follow-up periods are open-ended. The endpoints for patients with confirmed or suspected hereditary TTP and family members are death or withdrawal of consent.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | – | Other: ObservationPatients with confirmed hereditary TTP due to congenital ADAMTS13 deficiency |
| 2 | – | Other: ObservationFamily members of patients with confirmed hereditary TTP |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with either confirmed or with suspected hereditary TTP and their family members are eligible for enrollment
Key inclusion criteria (3 listed)
- Severe ADAMTS13 deficiency ( ≤ 10% activity) and no ADAMTS 13 inhibitor on two or more occasions at least one month apart
- Being a family member of a confirmed or suspected patient
- Molecular analysis of ADAMTS13 gene with one or more mutations and/or positive infusion trial (full recovered ADAMTS13 activity after infused fresh frozen plasma (FFP) with a plasma half-life of 2-4 days)
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 |
|---|---|---|
| ObservationNo interventions planned: treatment of patients at the discretion of the treating/responsible physician | Other | 1, 2 |
Primary outcome
Clinical presentation and disease course in hereditary TTP
Time frame: every year until death
5 secondary outcomes
Identification of disease-modifying factors, including genotype-phenotype correlation
Time frame: every year until death
Treatment requirements in hereditary TTP patients
Time frame: every year until death
Documentation of potential adversary effects of (long-term) plasma treatment
Time frame: every year until death
Mortality of hereditary TTP
Time frame: every year until death
Clinical course in family members
Time frame: every year until death
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Oct 2023.
- Derived from this study: PubMed 33649760Tarasco E, Butikofer L, Friedman KD, George JN, Hrachovinova I, Knobl PN, Matsumoto M, von Krogh AS, Aebi-Huber I, Cermakova Z, Gorska-Kosicka M, Jalowiec KA, Largiader CR, Prohaszka Z, Sinkovits G, Windyga J, Lammle B, Kremer Hovinga JA. Annual incidence and severity of acute episodes in hereditary thrombotic thrombocytopenic purpura. Blood. 2021 Jun 24;137(25):3563-3575. doi: 10.1182/blood.2020009801.
- Derived from this study: PubMed 30792199van Dorland HA, Taleghani MM, Sakai K, Friedman KD, George JN, Hrachovinova I, Knobl PN, von Krogh AS, Schneppenheim R, Aebi-Huber I, Butikofer L, Largiader CR, Cermakova Z, Kokame K, Miyata T, Yagi H, Terrell DR, Vesely SK, Matsumoto M, Lammle B, Fujimura Y, Kremer Hovinga JA; Hereditary TTP Registry. The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017. Haematologica. 2019 Oct;104(10):2107-2115. doi: 10.3324/haematol.2019.216796. Epub 2019 Feb 21.
- Derived from this study: PubMed 26830967Fan X, Kremer Hovinga JA, Shirotani-Ikejima H, Eura Y, Hirai H, Honda S, Kokame K, Taleghani MM, von Krogh AS, Yoshida Y, Fujimura Y, Lammle B, Miyata T. Genetic variations in complement factors in patients with congenital thrombotic thrombocytopenic purpura with renal insufficiency. Int J Hematol. 2016 Mar;103(3):283-91. doi: 10.1007/s12185-015-1933-7. Epub 2016 Feb 1.
- Derived from this study: PubMed 25655456Lammle B. VWF and complement. Blood. 2015 Feb 5;125(6):896-8. doi: 10.1182/blood-2014-12-612556. No abstract available.
- Derived from this study: PubMed 23715103Mansouri Taleghani M, von Krogh AS, Fujimura Y, George JN, Hrachovinova I, Knobl PN, Quist-Paulsen P, Schneppenheim R, Lammle B, Kremer Hovinga JA. Hereditary thrombotic thrombocytopenic purpura and the hereditary TTP registry. Hamostaseologie. 2013 May 29;33(2):138-43. doi: 10.5482/HAMO-13-04-0026.
- Additional informationLink provided by the sponsor.
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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.
Johanna A Kremer Hovinga, MD study chair
University Clinic of Hematology and Central Hematology Laboratory, Bern University Hospital and the University of Bern, Inselspital
Bernhard Lämmle, M.D. study chair
University Medical Center, Center for Thrombosis and Hemostasis, Mainz, Germany
Yoshihiro Fujimura, M.D. study chair
Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan
7 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thrombotic Thrombocytopenic Purpura and Congenital Thrombotic Thrombocytopenic Purpura, run by Insel Gruppe AG, University Hospital Bern at 7 sites in 7 countries.
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 3 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: looking for participants now, as of the sponsor's last update on 11 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Austria, Czechia, Germany, Japan and 2 more countries. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Johanna A Kremer Hovinga, MD (+41 31 632 02 65, johanna.kremer@insel.ch), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical presentation and disease course in hereditary TTP (time frame: every year until death). 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 NCT01257269, which also lists 5 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 11 Oct 2023.