Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP)

This study has been completed.
Sponsor:
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Symphogen A/S
ClinicalTrials.gov Identifier:
NCT00718692
First received: July 18, 2008
Last updated: September 5, 2012
Last verified: September 2012
  Purpose

This trial is designed as a multi-centre, single-dose, exploratory dose-finding, open label trial evaluating the safety and efficacy of Sym001 in 4-9 consecutive cohorts. Subjects will receive a single IV dose of Sym001.


Condition Intervention Phase
Immune Thrombocytopenic Purpura
Biological: Sym001
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multi-centre, Single Intravenous Dose, Exploratory Dose-finding, Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP) in RhD Positive, Non-splenectomized Adult Subjects.

Resource links provided by NLM:


Further study details as provided by Symphogen A/S:

Primary Outcome Measures:
  • Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs) [ Time Frame: 6 weeks post dosing ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Measurements of platelet counts [ Time Frame: From day 1 through week 6 ] [ Designated as safety issue: No ]

Enrollment: 61
Study Start Date: July 2008
Study Completion Date: October 2011
Primary Completion Date: August 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Biological: Sym001
Each cohort will receive one single dose of Sym001 according the assigned dose level.

  Eligibility

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

Inclusion Criteria:

  • Confirmed presence of thrombocytopenia with platelet count < 30,000/mm3 at the pre-dose visit.
  • History of isolated ITP
  • RhD-positive serology.
  • Previous treatment and response to first line therapy for ITP

Exclusion Criteria:

  • Known clinical picture suggestive of other causes of thrombocytopenia, especially systematic lupus erythematosus,antiphospholipid syndrome, Evans syndrome, immunodeficiency states, lymphoproliferative disorders, liver disease,ingestion of drugs such as quinidine/quinine, heparin and sulfonamides and hereditary thrombocytopenia confirmed by relevant laboratory findings.
  • Suspected infection with HIV, Hepatitis C, H. pylori.
  • Clinical splenomegaly
  • History of abnormal bone marrow examination.
  • Ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale.
  • Underlying haemolytic condition
  • History of splenectomy.
  • Subject is pregnant, breast feeding or intends to become pregnant.
  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: NCT00718692

  Show 59 Study Locations
Sponsors and Collaborators
Symphogen A/S
Swedish Orphan Biovitrum
Investigators
Principal Investigator: Mario Von Depka Prondzinski, PD Dr. Werlhoff Institut Hannover, Germany
Principal Investigator: Ann Janssens, Dr. ZU Gasthuisberg, Leuven, Belgium
Principal Investigator: Javier Loscertales Pueyo, Dra Hospital La Princesa, Madrid, Spain
Principal Investigator: Wieslaw Wiktor Jedrzrjczak, Prof. Katedra i klinika Hematologii, Warszawa, Poland
Principal Investigator: Andrei Cucucianu, Dr. Prof. Dr. Ion Chiricuta Oncology Institute, Cluj-Napoca, Romania
Principal Investigator: Marie Scully, Prof. Dr. MD University College London, UK
  More Information

No publications provided by Symphogen A/S

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Symphogen A/S
ClinicalTrials.gov Identifier: NCT00718692     History of Changes
Other Study ID Numbers: Sym001-03
Study First Received: July 18, 2008
Last Updated: September 5, 2012
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products
Germany: Paul-Ehrlich-Institut
Spain: Spanish Agency of Medicines
Poland: Ministry of Health
Serbia and Montenegro: Agency for Drugs and Medicinal Devices
Romania: National Medicines Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency
India: Ministry of Health
Israel: Ministry of Health
Russia: Ministry of Health of the Russian Federation
Ukraine: Ministry of Health
United States: Food and Drug Administration

Keywords provided by Symphogen A/S:
Idiopathic
ITP

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

ClinicalTrials.gov processed this record on September 16, 2014