Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With… (NCT01151423) | Clinical Trial Compass
CompletedPhase 2
Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
United States, Australia, Austria75 participantsStarted 2011-01
Plain-language summary
This study was a Phase II, single-blind, randomized, placebo-controlled trial to determine whether anti-vWF Nanobody is safe and effective as adjunctive treatment in patients with aTTP.
Patients received either placebo or anti-vWF Nanobody as adjunctive therapy to plasma exchange (PE).
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 18 years of age or older (adults) or aged 12 to \< 18 years (adolescents)
* Male or female subject, willing to accept an acceptable contraceptive regimen
* Subject with a clinical diagnosis of TTP
* Requiring PE (one single PE session prior to randomization into the study was allowed)
* Subject accessible to follow-up
* Subject able to provide signed and dated informed consent and assent (if applicable, for adolescents)
Exclusion Criteria:
* Platelet count ≥ 100,000/µL
* Severe active infection indicated by sepsis (requirement for pressors with or without positive blood cultures)
* Clinical evidence of enteric infection with Escherichia coli 0157 or related organism
* Anti-phospholipid syndrome
* Diagnosis of disseminated intravascular coagulation (DIC)
* Pregnancy or breast-feeding
* Hematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy
* Known with congenital TTP
* Active bleeding or high risk of bleeding
* Uncontrolled arterial hypertension
* Known chronic treatment with anticoagulant treatment that cannot be stopped safely, including but not limited to:
* vitamin K antagonists
* heparin or low molecular weight heparin (LMWH)
* non-acetyl salicylic acid non-steroidal anti-inflammatory molecules
* Severe or life threatening clinical condition other than TTP that would impair participation in the study
* Subjects with malignancies resulting in a life expectation of less than 3 months
* Subjects with known…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets ≥ 150,000/µL
Timeframe: From the day of first study drug administration up to 30 days after first study drug administration