A Randomised, Multi-centre, Double-Blind, Placebo-Controlled, Single/Multiple Dose Escalation Phase Ib/IIa Clinical Trial to Investigate the Safety and Efficacy of Recombinant Human Soluble Fc-gamma Receptor IIb (SM101) for Intravenous Application in the Treatment of Patients with Chronic Adult Idiopathic Thrombocytopenic Purpura (ITP).

• Register: EUCTR
• Last refreshed on: 8 August 2016
• Main ID: EUCTR2009-014842-28-BE
• Date of registration: 14/09/2009
• Prospective Registration: Yes
• Primary sponsor: SuppreMol GmbH
• Public title: A Randomised, Multi-centre, Double-Blind, Placebo-Controlled, Single/Multiple Dose Escalation Phase Ib/IIa Clinical Trial to Investigate the Safety and Efficacy of Recombinant Human Soluble Fc-gamma Receptor IIb (SM101) for Intravenous Application in the Treatment of Patients with Chronic Adult Idiopathic Thrombocytopenic Purpura (ITP).
• Scientific title: A Randomised, Multi-centre, Double-Blind, Placebo-Controlled, Single/Multiple Dose Escalation Phase Ib/IIa Clinical Trial to Investigate the Safety and Efficacy of Recombinant Human Soluble Fc-gamma Receptor IIb (SM101) for Intravenous Application in the Treatment of Patients with Chronic Adult Idiopathic Thrombocytopenic Purpura (ITP).
• Date of first enrolment: 06/01/2010
• Target sample size: 51
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-014842-28
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Germany, Poland

Key inclusion & exclusion criteria

Inclusion criteria:
1. Subject has provided written informed consent prior to any study-related procedure
2. Male or female subjects aged 18 to 75, with or without splenectomy
3. Diagnosis of chronic idiopathic thrombocytopenic purpura (ITP) based on subject’s history, physical examination, blood count and blood film examination according to the British Society for Haematology (BSH) and American Society of Hematology (ASH) guidelines for at least 6 months
4. Subject received previously at least one ITP therapy
5. Platelet count less than 30,000/µL from at least 2 measurements within 4 weeks prior to first investigational medicinal product (IMP) administration. The second measurement should not be older than 1 week. Both measurements should be at least 1 week apart.
6. Concurrent ITP corticosteroid dose, if any, has been stable for 4 weeks preceding the first dose of IMP, and is intended to remain stable during the IMP treatment period
7. Subjects > 60 years of age must have had a documented history of chronic ITP with a bone marrow report to confirm the diagnosis
8. Women of childbearing potential (WOCBP) must have a negative serum pregnancy test within 1 week prior to first IMP administration
9. Both WOCBP and men must use a medically acceptable method of contraception (Appendix III) prior to inclusion and throughout the study
10. Adequate liver and kidney function (as detailed in the protocol)
11. Eastern Cooperative Oncology Group (ECOG) Performance status of 0 or 1, with a life expectancy of at least 6 months

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Female subjects who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period
2. Secondary thrombocytopenia such as: acute/chronic leukaemia, myelodysplasia, megaloblastic anaemia, microangiopathic anaemia, inherited thrombocytopenia, pseudothrombocytopenia, HIV, drug induced thrombocytopenia, etc.
3. Subjects with confirmed HIV, hepatitis B or C infection.
4. Subjects with other acute infections within 4 weeks preceding the first dose of investigational medicinal product (IMP)
5. Subject received intravenous immunoglobulins (IVIGs) or anti-D antibody treatment within 4 weeks preceding the first dose of IMP
6. Subject received rituximab or any other B-cell depleting agent within 24 months preceding the first dose of IMP
7. All other previously completed ITP treatment must achieve at least 5 times their terminal half-life prior to first administration of IMP.
8. Subject receives concomitant ITP medication other than corticosteroids, except rescue medication during the clinical trial
9. Splenectomy within 4 weeks prior to screening
10. Subject received or is planning to receive a haematopoietic stem cell transplantation during the clinical trial
11. Any kind of disorder that compromises the ability of the subject to give written informed consent and/or to comply with all study procedures
12. Known hypersensitivity to any recombinant E. coli-derived product or IMP excipients (Tween, Mannitol)
13. Subjects participating in a concurrent clinical trial or treated with another investigational drug within 4 weeks or 5 terminal half-lives of the drug (whichever is longer) preceding the first dose of IMP
14. History of or current alcohol or drug abuse
15. Any condition which in the judgment of the investigator would place the subject at undue risk or interfere with the results of the study
16. Subjects with an active malignancy
17. Subjects with active, serious, life-threatening disease with a life expectancy of less than 6 months
18. Subject was previously included in the present clinical trial


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Idiopathic Thrombocytopenic Purpura
MedDRA version: 12.0 Level: LLT Classification code 10021245 Term: Idiopathic thrombocytopenic purpura

Intervention(s)

Product Name: SM101
Product Code: SM101
Pharmaceutical Form: Concentrate for solution for infusion
Current Sponsor code: SM101
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

Main Objective: To evaluate the safety and tolerability of i.v. administered SM101 at various dose levels in a single/multiple dosing manner in subjects with chronic ITP

Secondary Objective: To evaluate the efficacy of SM101 following single and multiple i.v. dosing in the treatment of subjects with chronic ITP
To evaluate an appropriate SM101 dose for the extension part
To evaluate various immunology parameters during and after SM101 treatment
To evaluate PK parameters of i.v. administered SM101

Primary end point(s): Safety based on incidence, severity, causality and seriousness of adverse events, laboratory results and adverse events of special interest. Adverse events will be graded using the NCI CTCAE, v 3.0

Secondary ID(s)

SM101-201-itp-09