Long-term Safety and Efficacy of Momelotinib in Subjects With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Polycythemia Vera or Essential Thrombocythemia

Primary Myelofibrosis Clinical Trial

Official title:

Open-label Study to Assess the Long-term Safety and Efficacy of Momelotinib in Subjects With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis, Polycythemia Vera or Essential Thrombocythemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02124746
• Other study ID #: GS-US-352-1154
• Secondary ID: 2013-004476-36
• Status: Completed
• Phase: Phase 2
• Start date: April 30, 2014
• Est. completion date: December 6, 2018

Study information

• Verified date: June 2023
• Source: Sierra Oncology LLC - a GSK company
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This open-label study is to determine the long-term safety and tolerability of momelotinib in previously enrolled study participants with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), post-essential thrombocythemia myelofibrosis (post-ET MF), polycythemia vera (PV), or essential thrombocythemia (ET), who have tolerated and achieved stable disease or better with momelotinib treatment while enrolled in a previous clinical trial.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 87
• Est. completion date: December 6, 2018
• Est. primary completion date: December 6, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Key Inclusion Criteria:

• Currently enrolled in study CCL09101E, or YM387-II-02, or successfully completed 24 weeks of study GS-US-352-1672

• Able to comprehend and willing to sign informed consent form

Key Exclusion Criteria:

• Known hypersensitivity to momelotinib, its metabolites, or formulation excipients

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Essential Thrombocythemia
• Polycythemia
• Polycythemia Vera
• Post-Essential Thrombocythemia Myelofibrosis
• Post-Polycythemia Vera Myelofibrosis
• Primary Myelofibrosis
• Thrombocythemia, Essential
• Thrombocytosis

Intervention

Drug:
• Momelotinib
Momelotinib tablets administered orally once daily

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Sierra Oncology LLC - a GSK company

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Long Term Safety and Tolerability as Measured by the Incidence and Severity of Adverse Events and Clinical Laboratory Abnormalities	Long-term safety and tolerability profile of momelotinib based on safety data (adverse events and selected hematology and chemistry laboratory parameters) collected after the first dose of momelotinib in the parent study.	From the first dose of momelotinib in the parent study to 30 days following permanent discontinuation of momelotinib in Study GS-US-352-1154.
Secondary	Splenic Response Rate	The number of subjects achieving a spleen response, defined as a reduction of 50% or more in palpable splenomegaly of a spleen that was at least 10 cm below the LCM at baseline, or a spleen that was palpable at > 5 cm and < 10 cm below the LCM at baseline becoming not palpable for at least 56 days, using baseline of the parent study as the reference.	From baseline in the parent study until the last spleen assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Duration of Splenic Response	The interval from the first onset of splenic response (in the parent study or Study GS-US-352-1154) to the earliest date of loss of splenic response. Loss of response was defined as the reduction of splenomegaly by < 50% among responders (with splenomegaly = 10 cm below the LCM at baseline) that lasts = 56 days, or the recurrence of > 0 cm splenomegaly among responders (with splenomegaly > 5 and < 10 cm at baseline) that lasts = 56 days.; Duration of splenic response was measured by descriptive statistics. Data from responders who maintained their response was censored at the last assessment date.	From baseline in the parent study until the last spleen assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Transfusion Independence Response Rate	The number of transfusion dependent subjects at entry to a parent study who became transfusion-independent for = 12 weeks at any time from the first dose of momelotinib in the parent study until the end of Study GS-US-352-1154.	From baseline in the parent study until the last assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Duration of Transfusion Independence Response	The interval from the first onset date of transfusion independence (in the parent study or Study GS-US-352-1154) to the earliest date of loss of response for participants who are transfusion dependent at baseline in the parent study. Loss of TI response was defined as receiving an RBC transfusion after achieving a TI response.; Duration of transfusion independence response was measured by descriptive statistics. Data from responders who maintained their response was censored at the last assessment date.	From baseline in the parent study until the last assessment date in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Anemia Response Rate	The number of subjects achieving an anemia response, defined as: Achieving transfusion independence for = 12 weeks, for subjects who were transfusion-dependent at baseline in the parent study, or; Having = 2 g/dL increase in Hgb from baseline for = 12 weeks, for subjects with Hgb < 10 g/dL at baseline in the parent study who were not transfusion-dependent (Cohort 1) or who were transfusion-independent (Cohort 2).	From baseline in the parent study until the last assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Duration of Anemia Response	The interval from the first onset of anemia response (in the parent study or Study GS-US-352-1154) to the earliest date of loss of anemia response. Loss of anemia response was defined as having any RBC transfusion after achieving an anemia response.; Duration of anemia response was measured by descriptive statistics. Data from responders who maintained their response was censored at the last assessment date.	From baseline in the parent study until the last assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Rate of RBC Transfusion	The average number of RBC units per subject month during the parent study and/or Study GS-US-352-1154.	From the first dose of momelotinib in the parent study until the last dose of momelotinib in Study GS-US-352-1154.
Secondary	Overall Survival	The interval from the first dose of momelotinib in the parent study until death from any cause.; Overall survival was analyzed using the Kaplan-Meier method. Data from subjects who were lost to follow-up or remained alive until the end of the study were censored at the date of last contact or last response assessment.	From baseline in the parent study until the date of last contact or last response assessment, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Progression-Free Survival	The interval from the first dose of momelotinib in the parent study until the first documentation of definitive progressive disease as defined in 2006 IWG-MRT or death due to any cause.; Subjects who were free of progression were censored at the last assessment date.	From baseline in the parent study until the last response assessment, up to 30 days following permanent discontinuation of momelotinib.
Secondary	Leukemia-Free Survival	The interval from the first dose of momelotinib in the parent study until the first documented leukemic transformation or death from any cause. Leukemic transformation was documented in the adverse event electronic case report form.; Leukemia-free survival was analyzed using the Kaplan-Meier method. Subjects who were free of leukemia transformation were censored at the last assessment date.	From baseline in the parent study until the date of the last assessment, up to 30 days following permanent discontinuation of momelotinib.