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Anti-TGF-beta Therapy in Patients With Myelofibrosis

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ClinicalTrials.gov Identifier: NCT01291784
Recruitment Status : Terminated (Early termination when the drug was no longer made available by the pharmaceutical company due to unanticipated management and administrative decisions.)
First Posted : February 8, 2011
Results First Posted : August 28, 2014
Last Update Posted : December 8, 2014
Sponsor:
Information provided by (Responsible Party):
John Mascarenhas, Icahn School of Medicine at Mount Sinai

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Myelofibrosis
Primary Myelofibrosis
Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase
Post-essential Thrombocythemia Related Myelofibrosis
Intervention: Biological: monoclonal antibody to TGF-beta

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Recruitment period from Jan 2011 to Feb 2012 of known patients to the medical clinic.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Sub A Subject A
Sub B Subject B
Sub C Subject C

Participant Flow:   Overall Study
    Sub A   Sub B   Sub C
STARTED   1   1   1 
COMPLETED   1   0   1 
NOT COMPLETED   0   1   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
patients with intermediate -1 or higher primary myelofibrosis (PMF) or post-polycythemia vera/essential thrombocythemia myelofibrosis (Post-PV/ET MF). Patients were eligible if they had documented BMF of MF-2 or higher as assessed by the European consensus grading score and grade 3 or higher by modified Bauermeister scale.

Reporting Groups
  Description
Phase 1 MF Subjects 3 subjects were enrolled in the study. The three subjects were treated at a GC1008 dose level of 1 mg/kg given intravenously over approximately 60 minutes and then repeated every 28 days for a total of 6 cycles in the core study period and then an additional 6 cycles in an extension phase.

Baseline Measures
   Phase 1 MF Subjects 
Overall Participants Analyzed 
[Units: Participants]
 3 
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 68.67  (10.07) 
[1] Actual age
Gender 
[Units: Participants]
 
Female   2 
Male   1 
Region of Enrollment 
[Units: Participants]
 
United States   3 


  Outcome Measures

1.  Primary:   Safety and Tolerability   [ Time Frame: 28 days ]

2.  Secondary:   Bauermeister Scale   [ Time Frame: 6 months ]

3.  Secondary:   European Consensus Fibrosis Grade   [ Time Frame: 6 months ]

4.  Secondary:   Peripheral Blood CD34+   [ Time Frame: 6 months ]

5.  Secondary:   JAK2V617F Allele Burden   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data

Early termination when the drug was no longer made available by the pharmaceutical company due to unanticipated management and administrative decisions.

Limited patient data (2 evaluable patients), a dose accumulation ratio cannot be determined.



  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. John O. Mascarenhas
Organization: Icahn School of Medicine at Mount Sinai
phone: 212-241-6756
e-mail: john.mascarenhas@mssm.edu


Publications of Results:

Responsible Party: John Mascarenhas, Icahn School of Medicine at Mount Sinai
ClinicalTrials.gov Identifier: NCT01291784     History of Changes
Other Study ID Numbers: GCO 10-1450
GC1008-JM
First Submitted: February 7, 2011
First Posted: February 8, 2011
Results First Submitted: July 31, 2013
Results First Posted: August 28, 2014
Last Update Posted: December 8, 2014