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
  Purpose

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.


Condition Intervention Phase
Thrombotic Thrombocytopenic Purpura
Biological: anti-vWF Nanobody
Biological: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Ablynx:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • 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


Enrollment: 75
Study Start Date: September 2010
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: anti-vWF Nanobody 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.
Placebo Comparator: Placebo 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01151423

  Show 51 Study Locations
Sponsors and Collaborators
Ablynx
Investigators
Study Director: Christian G Duby, MD Ablynx NV
  More Information

No publications provided by Ablynx

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Ablynx
ClinicalTrials.gov Identifier: NCT01151423     History of Changes
Other Study ID Numbers: ALX-0681-2.1/10, 2010-019375-30
Study First Received: June 25, 2010
Last Updated: July 3, 2014
Health Authority: United States: Food and Drug Administration
Belgium: Federal Agency for Medicinal Products and Health Products
Germany: Paul-Ehrlich-Institut
Austria: Agency for Health and Food Safety
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Italy: The Italian Medicines Agency
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Switzerland: Swissmedic
Israel: Israeli Health Ministry Pharmaceutical Administration
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Bulgaria: Bulgarian Drug Agency
Romania: National Medicines Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration

Additional relevant MeSH terms:
Purpura, Thrombocytopenic
Purpura
Purpura, Thrombotic Thrombocytopenic
Blood Coagulation Disorders
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombophilia

ClinicalTrials.gov processed this record on September 16, 2014