Trial Outcomes & Findings for Anti-TGF-beta Therapy in Patients With Myelofibrosis (NCT NCT01291784)
NCT ID: NCT01291784
Last Updated: 2014-12-08
Results Overview
To assess the safety and tolerability of GC1008 in patients with primary myelofibrosis (PMF) or post-polycythemia vera/essential thrombocythemia myelofibrosis (Post-PV/ET MF). A total of 9 AEs determined by the investigator to be at least possibly related to GC1008 occurred during the study.
TERMINATED
PHASE1
3 participants
28 days
2014-12-08
Participant Flow
Recruitment period from Jan 2011 to Feb 2012 of known patients to the medical clinic.
Participant milestones
| Measure |
Sub A
Subject A
|
Sub B
Subject B
|
Sub C
Subject C
|
|---|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
1
|
|
Overall Study
COMPLETED
|
1
|
0
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Anti-TGF-beta Therapy in Patients With Myelofibrosis
Baseline characteristics by cohort
| Measure |
Phase 1 MF Subjects
n=3 Participants
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.
|
|---|---|
|
Age, Continuous
|
68.67 years
STANDARD_DEVIATION 10.07 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 28 daysTo assess the safety and tolerability of GC1008 in patients with primary myelofibrosis (PMF) or post-polycythemia vera/essential thrombocythemia myelofibrosis (Post-PV/ET MF). A total of 9 AEs determined by the investigator to be at least possibly related to GC1008 occurred during the study.
Outcome measures
| Measure |
Sub A
n=1 Participants
Subject A
|
Sub B
n=1 Participants
Subject B
|
Sub C
n=1 Participants
Subject C
|
|---|---|---|---|
|
Safety and Tolerability
|
4 events
|
4 events
|
1 events
|
SECONDARY outcome
Timeframe: 6 monthsTo assess the clinical response to therapy with GC1008 by International Working Group (IWG) criteria and measure the change in degree of bone marrow fibrosis (BMF) assessed by Bauermeister scale. Bauermeister scale: 0, no demonstrable reticulin fibers; 1, occasional fine individual fibers and foci of a fine-fiber network; 2, fine fiber network throughout most of the section, but no coarse fibers; 3, diffuse fiber network with scattered thick coarse fibers, but no mature collagen; and 4, diffuse, often coarse fiber network with areas of collagen.
Outcome measures
| Measure |
Sub A
n=1 Participants
Subject A
|
Sub B
n=1 Participants
Subject B
|
Sub C
n=1 Participants
Subject C
|
|---|---|---|---|
|
Bauermeister Scale
|
4 units on a scale
|
3 units on a scale
|
4 units on a scale
|
SECONDARY outcome
Timeframe: 6 monthsTo assess the clinical response to therapy with GC1008 by International Working Group (IWG) criteria and measure the change in degree of bone marrow fibrosis (BMF) assessed by European consensus grading system. This scheme consists of a qualitative (reticulin or collagen) and quantitative evaluation of bone marrow fibrosis and distinguishes four increasing categories, ranging from MF-0, which corresponds to normal bone marrow, to MF-3, in which coarse bundles of collagen fibrosis are identifiable with significant osteosclerosis.
Outcome measures
| Measure |
Sub A
n=1 Participants
Subject A
|
Sub B
n=1 Participants
Subject B
|
Sub C
n=1 Participants
Subject C
|
|---|---|---|---|
|
European Consensus Fibrosis Grade
|
3 units on a scale
|
2 units on a scale
|
2 units on a scale
|
SECONDARY outcome
Timeframe: 6 monthsInvestigate exploratory markers for their ability to predict responsiveness to treatment with GC1008.
Outcome measures
| Measure |
Sub A
n=1 Participants
Subject A
|
Sub B
n=1 Participants
Subject B
|
Sub C
n=1 Participants
Subject C
|
|---|---|---|---|
|
Peripheral Blood CD34+
|
3 percentage of hematopoietic stem cells
|
2 percentage of hematopoietic stem cells
|
0 percentage of hematopoietic stem cells
|
SECONDARY outcome
Timeframe: 6 monthsInvestigate exploratory markers, hematopoietic cells, for their ability to predict responsiveness to treatment with GC1008. Analysis of percentage of mutant alleles in hematopoietic stem cells.
Outcome measures
| Measure |
Sub A
n=1 Participants
Subject A
|
Sub B
n=1 Participants
Subject B
|
Sub C
n=1 Participants
Subject C
|
|---|---|---|---|
|
JAK2V617F Allele Burden
|
48 percentage of mutant alleles
|
95 percentage of mutant alleles
|
14 percentage of mutant alleles
|
Adverse Events
Sub A
Sub B
Sub C
Serious adverse events
| Measure |
Sub A
n=1 participants at risk
Subject A
|
Sub B
n=1 participants at risk
Subject B
|
Sub C
n=1 participants at risk
Subject C
|
|---|---|---|---|
|
Cardiac disorders
Asystole
|
0.00%
0/1
|
100.0%
1/1 • Number of events 1
|
0.00%
0/1
|
Other adverse events
| Measure |
Sub A
n=1 participants at risk
Subject A
|
Sub B
n=1 participants at risk
Subject B
|
Sub C
n=1 participants at risk
Subject C
|
|---|---|---|---|
|
Blood and lymphatic system disorders
leukocytosis
|
0.00%
0/1
|
100.0%
1/1 • Number of events 1
|
0.00%
0/1
|
|
General disorders
fatigue
|
0.00%
0/1
|
100.0%
1/1 • Number of events 1
|
0.00%
0/1
|
|
General disorders
hypoxia
|
0.00%
0/1
|
100.0%
1/1 • Number of events 1
|
0.00%
0/1
|
|
Blood and lymphatic system disorders
leukopenia
|
100.0%
1/1 • Number of events 1
|
0.00%
0/1
|
0.00%
0/1
|
|
Skin and subcutaneous tissue disorders
skin lesions
|
100.0%
1/1 • Number of events 3
|
0.00%
0/1
|
0.00%
0/1
|
|
Immune system disorders
herpes zoster
|
0.00%
0/1
|
0.00%
0/1
|
100.0%
1/1 • Number of events 1
|
Additional Information
Dr. John O. Mascarenhas
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place