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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ablynx
ClinicalTrials.gov Identifier:
NCT01151423
First received: June 25, 2010
Last updated: July 3, 2014
Last verified: July 2014

June 25, 2010
July 3, 2014
September 2010
April 2014   (final data collection date for primary outcome measure)
Reduction of time-to-recovery [ Time Frame: after completion of plasma exchange, followed by confirmation 48h later ] [ Designated as safety issue: No ]
Reduction of time-to-recovery, defined by the achievement of laboratory blood marker response (platelet count and LDH level), confirmed at 48 hours after the initial reporting of this response
Same as current
Complete list of historical versions of study NCT01151423 on ClinicalTrials.gov Archive Site
  • Reduction of number of relapses [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Number of patients relapsing of TTP (de novo event of TTP that occurs later than 30 days after the last daily plasma exchange) for a maximum of 1 year, and time to first relapse of TTP
  • Reduction of number of exacerbations [ Time Frame: 30 days after the last daily plasma exchange session ] [ Designated as safety issue: No ]
    Number of exacerbations of TTP, and time to first exacerbation of TTP
  • Reduction of number of relapses [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Number of patients relapsing of TTP (de novo event of TTP that occurs later than 30 days after the last plasma exchange) for a maximum of 1 year, and time to first relapse of TTP
  • Reduction of number of exacerbations [ Time Frame: 30 days after the last plasma exchange session ] [ Designated as safety issue: No ]
    Number of exacerbations of TTP, and time to first exacerbation of TTP
Not Provided
Not Provided
 
Study to Assess Efficacy and Safety of Anti-von Willebrand Factor Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (TTP)
A Phase II, Single-blind, Randomised, Placebo-controlled Trial to Study the Efficacy and Safety of Anti-von Willebrand Factor Nanobody Administered as Adjunctive Treatment to Patients With Acquired Thrombotic Thrombocytopenic Purpura

The purpose of this study is to determine whether anti-von Willebrand factor Nanobody is safe and effective as adjunctive treatment in patients with acquired thrombotic thrombocytopenic purpura (TTP). Patients will receive either placebo or anti-von Willebrand factor Nanobody as adjunctive therapy to plasma exchange.

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Thrombotic Thrombocytopenic Purpura
  • Biological: anti-vWF Nanobody
    10 mg i.v. bolus injection prior to plasma exchange, followed by 10 mg s.c. injection once or twice per day. Maximum treatment duration is limited to 90 days.
  • Biological: Placebo
    10 mg i.v. bolus injection prior to plasma exchange, followed by daily 10 mg s.c. injection. Maximum treatment duration is limited to 90 days.
  • Experimental: anti-vWF Nanobody
    Intervention: Biological: anti-vWF Nanobody
  • Placebo Comparator: Placebo
    Intervention: Biological: Placebo
Holz JB. The TITAN trial--assessing the efficacy and safety of an anti-von Willebrand factor Nanobody in patients with acquired thrombotic thrombocytopenic purpura. Transfus Apher Sci. 2012 Jun;46(3):343-6. doi: 10.1016/j.transci.2012.03.027. Epub 2012 Apr 3.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
75
Not Provided
April 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • 18 years of age or older
  • Men or women willing to accept an acceptable contraceptive regimen
  • Patients with clinical diagnosis of TTP
  • Necessitating plasma exchange (one, single PE session prior to randomisation into the study is allowed)
  • Patient accessible to follow-up
  • Obtained, signed and dated informed consent

Exclusion Criteria:

  • Platelet count greater or equal to 100,000/µL
  • Severe active infection indicated by sepsis (requirement for pressors with or without positive blood cultures)
  • Clinical evidence of enteric infection with E.coli 0157 or related organism
  • Anti-phospholipid syndrome
  • Diagnosis of disseminated intravascular coagulation (DIC)
  • Pregnancy or breast-feeding
  • Haematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy
  • Known congenital TTP
  • Active bleeding or high risk of bleeding
  • Uncontrolled arterial hypertension
  • Known chronic treatment with anticoagulant treatment that can not be stopped safely
  • Severe or life threatening clinical condition other than TTP that would impair participation in the trial
  • 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 (common terminology criteria for adverse events) scale
  • Severe chronic renal impairment
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Austria,   United States,   Australia,   United Kingdom,   Belgium,   Bulgaria,   France,   Germany,   Israel,   Italy,   Romania,   Spain,   Switzerland
 
NCT01151423
ALX-0681-2.1/10, 2010-019375-30
Yes
Ablynx
Ablynx
Not Provided
Study Director: Christian G Duby, MD Ablynx NV
Ablynx
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP