Clinical trial NCT00718692: Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP)
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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Immune Thrombocytopenic Purpura
Keywords: Idiopathic
ITP
Lead sponsor: Symphogen A/S
Sponsor class: INDUSTRY
Collaborators: name: Swedish Orphan Biovitrum
First posted: 2008-07-21
Enrollment: 61
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility 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.

Primary outcomes:
measure: Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs)
timeFrame: 6 weeks post dosing
Secondary outcomes:
measure: Measurements of platelet counts
timeFrame: From day 1 through week 6
Other outcomes:
Locations:
city: Omaha
country: United States
facility: Univ Nebraska Med Ctr. UNMC, 1005
state: Nebraska
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania, Site 1002
state: Pennsylvania
city: Charleston
country: United States
facility: 2085 Henry Tecklenburg Drive, 1001
state: South Carolina
city: Brugge
country: Belgium
facility: Research Site 3203
city: Leuven
country: Belgium
facility: Research Site 3202
city: Yvoir
country: Belgium
facility: Research Site 3201
city: Berlin
country: Germany
facility: Research Site 4906, 4907
city: Duisburg
country: Germany
facility: Research Site 4903
city: Essen
country: Germany
facility: Research Site 4905
city: Halle
country: Germany
facility: Reseach Site 4908
city: Hannover
country: Germany
facility: Research Site 4901
city: Köln
country: Germany
facility: Research Site 4902
city: Regensburg
country: Germany
facility: Research Site 4904
city: Bangalore
country: India
facility: St. John's Medical College Hospital, 9106
city: Bangalore
country: India
facility: MS Ramaiah Memorial Hospital, 9108
city: Bangalore
country: India
facility: Narayana Hrudayalaya Hospitals, 9105
city: Bangalore
country: India
facility: Columbia Asia Referral Hospital-Yeshwanthpur, 9101
city: Chennai
country: India
facility: Aysha Hospital Pvt. Ltd., 9109
city: Hyderabad
country: India
facility: Apollo Hospital, 9104
city: Udupi
country: India
facility: Kasturba Medical College Hospital, 9103
city: Haifa
country: Israel
facility: Bnai-Zion Medical Center, 9721
city: Haifa
country: Israel
facility: Rambam Medical Center, 9723
city: Holon
country: Israel
facility: The Edith Wolfson Medical Center, 9725
city: Nahariya
country: Israel
facility: Western Galilee - Nahariya Hospital, 9722
city: Petach Tikva
country: Israel
facility: Rabin Medical Center, 9724
city: Tel Aviv
country: Israel
facility: Tel-Aviv Sourasky Medical Center, 9726
city: Gdansk
country: Poland
facility: Research site 4801
city: Krakow
country: Poland
facility: Research Site 4806
city: Lodz
country: Poland
facility: Research Site 4805
city: Warszava
country: Poland
facility: Research Site 4803
city: Warszawa
country: Poland
facility: Instytut Hematologii i Transfuzjologii, 4807
city: Warszawa
country: Poland
facility: Research Site 4802, 4808
city: Wroclaw
country: Poland
facility: Research Site 4804
city: Brasov
country: Romania
facility: Brasov Country Hospital, 4002
city: Bucuresti
country: Romania
facility: Institutul Clinic Fundeni, 4003
city: Cluj-Napoca
country: Romania
facility: "Prof. Dr. Ion Chiricuta" Oncolgy Institute, 4001
city: Iasi
country: Romania
facility: Spitalul Clinic de Urgente Sfantu Spiridon, 4004
city: Nizhny Novgorod
country: Russian Federation
facility: Regional Clinical Hospital of Nizhny Novgorod, 7003
city: Samara
country: Russian Federation
facility: Regional Clinical Hospital na MI Kalinin, 7005
city: St. Petersburg
country: Russian Federation
facility: Saint Petersburg State Institution Of Healthcare, 7001
city: Tula
country: Russian Federation
facility: State Healthcare Institution Tula Regional Clin. Hosp., 7002
city: Nis
country: Serbia
facility: Clinical Center Nis 3802
city: Novi Sad
country: Serbia
facility: Clinical center Vojvodina 3801
city: Zemun
country: Serbia
facility: Clinical Center Zemun 3803
city: Barcelona
country: Spain
facility: Reseach Site 3403
city: Madrid
country: Spain
facility: Hospital Gregorio Maranon, 3405
city: Madrid
country: Spain
facility: Hospital La Princesa, 3404
city: Madrid
country: Spain
facility: Research Site 3401
city: Valencia
country: Spain
facility: Research Site 3402
city: Cherkassy
country: Ukraine
facility: Cherkassy regional oncology center, 3906
city: Donetsk
country: Ukraine
facility: Gusak Academy of AMS Ukraine, 3905
city: Khmelnytskyi
country: Ukraine
facility: Khmelnytskyi Regional Hospital, 3903
city: Kiev
country: Ukraine
facility: City Hospital #9 Kyiv Clinical Hospital, 3904
city: Vinnytsya
country: Ukraine
facility: Vinnytsya Regional Clinical Hospital, 3902
city: Leeds
country: United Kingdom
facility: St. James's University Hospital, 4403
city: London
country: United Kingdom
facility: Hammersmith Hosptial, 4402
city: London
country: United Kingdom
facility: University College, 4401
city: Newcastle Upon Tyne
country: United Kingdom
facility: Royal Victoria Infirmery, 4404
city: Swansea
country: United Kingdom
facility: Singleton Hospital, 4405
Study officials:
affiliation: Werlhoff Institut Hannover, Germany
name: Mario Von Depka Prondzinski, PD Dr.
role: PRINCIPAL_INVESTIGATOR
affiliation: ZU Gasthuisberg, Leuven, Belgium
name: Ann Janssens, Dr.
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital La Princesa, Madrid, Spain
name: Javier Loscertales Pueyo, Dra
role: PRINCIPAL_INVESTIGATOR
affiliation: Katedra i klinika Hematologii, Warszawa, Poland
name: Wieslaw Wiktor Jedrzrjczak, Prof.
role: PRINCIPAL_INVESTIGATOR
affiliation: Prof. Dr. Ion Chiricuta Oncology Institute, Cluj-Napoca, Romania
name: Andrei Cucucianu, Dr.
role: PRINCIPAL_INVESTIGATOR
affiliation: University College London Hospitals
name: Marie Scully, Prof. Dr. MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Robak T, Windyga J, Trelinski J, von Depka Prondzinski M, Giagounidis A, Doyen C, Janssens A, Alvarez-Roman MT, Jarque I, Loscertales J, Rus GP, Hellmann A, Jedrzejczak 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, Naested 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.