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

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:

Genetics Home Reference related topics: thrombotic thrombocytopenic purpura

U.S. FDA Resources

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

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):

Robak T, Windyga J, Trelinski J, von Depka Prondzinski M, Giagounidis A, Doyen C, Janssens A, Alvarez-Román MT, Jarque I, Loscertales J, Rus GP, Hellmann A, Jêdrzejczak WW, Kuliczkowski K, Golubovic LM, Celeketic D, Cucuianu A, Gheorghita E, Lazaroiu M, Shpilberg O, Attias D, Karyagina E, Svetlana K, Vilchevska K, Cooper N, Talks K, Prabhu M, Sripada P, Bharadwaj TP, Næsted H, Skartved NJ, Frandsen TP, Flensburg MF, Andersen PS, Petersen J. Rozrolimupab, a mixture of 25 recombinant human monoclonal RhD antibodies, in the treatment of primary immune thrombocytopenia. Blood. 2012 Nov 1;120(18):3670-6. doi: 10.1182/blood-2012-06-438804. Epub 2012 Aug 20.

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
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations

Signs and Symptoms
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases

ClinicalTrials.gov processed this record on May 19, 2013