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4SC-201 (Resminostat) and Sorafenib in Advanced Hepatocellular Carcinoma (Shelter)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00943449
Recruitment Status : Completed
First Posted : July 22, 2009
Last Update Posted : January 16, 2014
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Tracking Information
First Submitted Date  ICMJE July 21, 2009
First Posted Date  ICMJE July 22, 2009
Last Update Posted Date January 16, 2014
Study Start Date  ICMJE July 2009
Actual Primary Completion Date June 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 21, 2009)
Progression Free Survival Rate (PFSR) of repeated oral doses of 4SC-201 and of the treatment combination of Sorafenib plus 4SC-201 [ Time Frame: 12 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 21, 2009)
  • To establish the MTD of 4SC-201 in combination with Sorafenib [ Time Frame: 12 weeks ]
  • To investigate the safety and tolerability of repeated oral doses of 4SC-201 and of the treatment combination of ascending repeated oral doses of 4SC-201 and Sorafenib [ Time Frame: 12 weeks ]
  • To investigate biomarkers [ Time Frame: 12 weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE 4SC-201 (Resminostat) and Sorafenib in Advanced Hepatocellular Carcinoma
Official Title  ICMJE A Proof-of-concept Phase II Study to Evaluate Efficacy, Safety and Pharmacokinetics of 4SC-201 and the Treatment Combination of Sorafenib Plus 4SC-201 in Patients With Hepatocellular Carcinoma Exhibiting Progressive Disease Under Sorafenib Treatment
Brief Summary The purpose of the study is to determine whether 4SC-201 alone or in combination with Sorafenib is effective and safe in the treatment of hepatocellular carcinoma in patients refractory to Sorafenib monotherapy.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hepatocellular Carcinoma
Intervention  ICMJE
  • Drug: 4SC-201
    oral administration
  • Drug: Sorafenib
    oral administration
Study Arms  ICMJE
  • Experimental: 4SC-201
    Intervention: Drug: 4SC-201
  • Experimental: 4SC-201 + Sorafenib
    • Drug: 4SC-201
    • Drug: Sorafenib
Publications * Bitzer M, Horger M, Giannini EG, Ganten TM, Wörns MA, Siveke JT, Dollinger MM, Gerken G, Scheulen ME, Wege H, Zagonel V, Cillo U, Trevisani F, Santoro A, Montesarchio V, Malek NP, Holzapfel J, Herz T, Ammendola AS, Pegoraro S, Hauns B, Mais A, Lauer UM, Henning SW, Hentsch B. Resminostat plus sorafenib as second-line therapy of advanced hepatocellular carcinoma - The SHELTER study. J Hepatol. 2016 Aug;65(2):280-8. doi: 10.1016/j.jhep.2016.02.043. Epub 2016 Mar 4.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 15, 2014)
Original Estimated Enrollment  ICMJE
 (submitted: July 21, 2009)
Actual Study Completion Date  ICMJE September 2013
Actual Primary Completion Date June 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  • Advanced stage hepatocellular carcinoma
  • Patients exhibiting progressive disease under Sorafenib treatment
  • Child-Pugh class A and B. Only patients with Child-Pugh index class B of not more than 7 will be included
  • ECOG performance status 0, 1 or 2
  • Precedent first-line treatment with Sorafenib minimum continuous dosing of 400 mg per day for at least 8 weeks

Main Exclusion Criteria:

  • Previous or concurrent cancer that is distinct in primary site or histology from HCC, EXCEPT cervical carcinoma in situ, treated basal cell carcinoma, superficial bladder tumors (Ta, Tis and T1) - any cancer curatively treated > 3 years prior to entry is permitted
  • Renal failure requiring hemo- or peritoneal dialysis
  • Known central nervous system (CNS) tumors including symptomatic brain metastasis
  • Child-Pugh index class B in combination with more than slight ascites or hepatic encephalopathy > Grade I
  • Pregnant or breastfeeding women
  • Sorafenib intolerance
  • Major surgery within the last 4 weeks
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany,   Italy
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00943449
Other Study ID Numbers  ICMJE 4SC-201-1-2009
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE 4SC AG
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Michael Bitzer, Prof. MD Medizinische Universitäts-Klinik Tübingen
PRS Account 4SC AG
Verification Date January 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP