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NCT01257269 · ClinicalTrials.gov registry record
Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
A clinical trial of Thrombotic Thrombocytopenic Purpura and Congenital Thrombotic Thrombocytopenic Purpura, sponsored by Insel Gruppe AG, University Hospital Bern.
- Recruiting
- Registry status
- 450
- Enrollment target
- 7
- Study locations
NCT01257269: Recruiting study of Thrombotic Thrombocytopenic Purpura and Congenital Thrombotic Thrombocytopenic Purpura, sponsored by Insel Gruppe AG, University Hospital Bern.
NCT01257269 is a study of Thrombotic Thrombocytopenic Purpura and Congenital Thrombotic Thrombocytopenic Purpura that is actively recruiting participants, run by Insel Gruppe AG, University Hospital Bern. The registered enrollment target is 450 participants. The trial reports 7 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01257269, a study of Thrombotic Thrombocytopenic Purpura and Congenital Thrombotic Thrombocytopenic Purpura, is actively recruiting participants, sponsored by Insel Gruppe AG, University Hospital Bern.
- RECRUITING
- Registry status
- 450 participants
- Enrollment target
- 7
- Study locations
Study 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. 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.
Conditions Studied
Interventions
- OTHER Observation
Study Locations (7)
Other
- Medical University of Vienna, Department of Medicine 1, Div. Hematology and Hemostasis Waehringer Guertel 18-20 - Vienna
- Institute of Hematology and Blood Transfusion, Coagulation Laboratory, U nemocnice 1 - Prague
- University Medical Center Hamburg-Eppendorf, Department of Pediatric Hematology and Oncology, Martinistr 52 - Hamburg
- Trondheim University St Olavs Hospital, Department of Hematology, PO Box 3250 Sluppen - Trondheim
- University Clinic of Hematology and Central Hematology Laboratory, Bern University Hospital and the University of Bern, Inselspital - Bern
Oklahoma
- University of Oklahoma Health Sciences Center, Department of Medicine, PO Box 26901 - Oklahoma City
Nara
- Nara Medical University, Department of Blood Transfusion Medicine, Shijyo-cho 840 - Kashihara
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 450 participants |
| Start Date | 2006-10 |
| Est. Completion | 2030-10 |
What NCT01257269 shows while recruiting
NCT01257269 is an observational study that tracks outcomes without assigning an intervention. The registered 450 participants enrollment target is mid-sized for trials with a published cap.
The record links to 5 conditions, with Thrombotic Thrombocytopenic Purpura appearing as the primary indexed condition, and to 1 intervention - of which Observation is the first listed.
NCT01257269 lists 7 locations in 3 states (Other, Oklahoma, Nara).
Frequently Asked Questions
What is clinical trial NCT01257269 about?
NCT01257269 is a clinical study titled "Genotype and Phenotype Correlation in 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...
What is the current status of trial NCT01257269?
This trial is currently recruiting. The enrollment target is 450 participants. The study started on 2006-10. Estimated completion is 2030-10.
What conditions does trial NCT01257269 study?
This clinical trial studies the following conditions: Thrombotic Thrombocytopenic Purpura, Congenital Thrombotic Thrombocytopenic Purpura, Familial Thrombotic Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Purpura, Congenital, Upshaw-Schulman Syndrome.
What interventions are being tested in trial NCT01257269?
The interventions under investigation include: Observation (OTHER).
Who is sponsoring clinical trial NCT01257269?
This trial is sponsored by Insel Gruppe AG, University Hospital Bern, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01257269 being conducted?
This trial has 7 study locations across Oklahoma, Nara. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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