Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Completed · Phase 2 · Has a placebo group

Conditions studied: Acquired Thrombotic Thrombocytopenic Purpura

In brief

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).

Key facts

Study ID

NCT01151423

Run by

Ablynx, a Sanofi company

People needed

75

Starts

2011-01-01

Expected to finish

2014-03-01

Last updated by the study team

2023-04-04

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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 or suspected bone marrow carcinosis
• Subjects who cannot comply with study protocol requirements and procedures
• Known hypersensitivity to the active substance or to excipients of the study drug
• Severe liver impairment, corresponding to grade 3 toxicity defined by the CTCAE scale. For the key liver parameters, this is defined as follows:
• bilirubin > 3 x upper limit of normal (ULN) (needed to differentiate isolated increase in indirect bilirubin due to hemolysis, this was not an exclusion parameter but disease-related)
• alanine transaminase (ALT)/ aspartate transaminase (AST) > 5 x ULN
• alkaline phosphatase (ALP) > 5 x ULN
• gamma-glutamyl transpeptidase (GGT) > 5 x ULN
• Severe chronic renal impairment, as defined by glomerular filtration rate < 30 mL/min

Where it is running

• Investigator Site — Washington D.C., District of Columbia, United States
• Investigator Site — Atlanta, Georgia, United States
• Investigator Site — St Louis, Missouri, United States
• Investigator Site — New York, New York, United States
• Investigator Site — Rochester, New York, United States
• Investigator Site — Valhalla, New York, United States
• Investigator Site — Durham, North Carolina, United States
• Investigator Site — Winston-Salem, North Carolina, United States
• Investigator Site — Columbus, Ohio, United States
• Investigator Site — Pittsburgh, Pennsylvania, United States
• Investigator Site — Dallas, Texas, United States
• Investigator Site — Salt Lake City, Utah, United States
• Investigator Site — Garran, Australian Capital Territory, Australia
• Investigator Site — Liverpool, Australia
• Investigator Site — Melbourne, Australia
• Investigator Site — Woolloongabba, Australia
• Investigator Site — Graz, Austria
• Investigator Site — Vienna, Austria
• Investigator Site — Antwerp, Belgium
• Investigator Site — Brussels, Belgium
• Investigator Site — Leuven, Belgium
• Investigator Site — Namur, Belgium
• Investigator Site — Sofia, Bulgaria
• Investigator Site — Caen, France
• Investigator Site — Los Angeles, California, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.