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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01236638




Registration number
NCT01236638
Ethics application status
Date submitted
5/11/2010
Date registered
8/11/2010
Date last updated
4/02/2019

Titles & IDs
Public title
Extension Study Evaluating the Long Term Safety and Efficacy Study of CYT387 in Primary Myelofibrosis (PMF) or Post-polycythemia Vera (PV) or Post-essential Thrombocythemia (ET)
Scientific title
A Phase II, Open-Label Extension Study Evaluating the Long Term Safety, Tolerability & Efficacy of Orally-Administered CYT387 in Primary Myelofibrosis or Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis
Secondary ID [1] 0 0
CCL09101E
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Myelofibrosis 0 0
Post-Polycythemia Vera Myelofibrosis 0 0
Post-Essential Thrombocythemia Myelofibrosis 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Momelotinib

Experimental: Momelotinib -


Treatment: Drugs: Momelotinib
Patients will continue receiving the same doses as assigned during the CCL09101 protocol; up to 400 mg once per day (QD). CYT387 will be administered orally as a single daily dose (at least 20 and no more than 28 hours apart, preferably in a fasted state at least two hours before and one hour after a meal), except for patients on the twice daily (BID) dosing regime (150 mg BID).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To determine the long term safety and tolerability of orally-administered CYT387 in patients with PMF or post-ET/PV MF following completion of core study CCL09101
Timepoint [1] 0 0
Safety monitoring will be undertaken for all patients every 3 months
Primary outcome [2] 0 0
To obtain information on the long term effectiveness of orally-administered CYT387 in patients with PMF or post-ET/PV MF
Timepoint [2] 0 0
Every three months

Eligibility
Key inclusion criteria
* Patients must have completed at least 9 cycles of treatment on the core study 'A Phase I/II, Open-Label, Dose-Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Orally-Administered CYT387 in Primary Myelofibrosis or Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis (CCL09101)' and achieved stable disease (SD), clinical improvement (CI), partial remission (PR) or complete remission (CR) using the International Working Group consensus criteria for treatment responses in myelofibrosis with myeloid metaplasia (IWG-MRT; Tefferi et al., 2006)
* Must be able to provide informed consent and be willing to sign an informed consent form.
* Must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
* Must have evidence of acceptable organ function within 7 days of initiating study drug as evidenced by the following:

* SGOT (AST) or SGPT (ALT) <= 2.5 x upper limit of normal (ULN) (or <= 5 x ULN if in the investigator's opinion the elevation is due to extramedullary hematopoiesis)
* Bilirubin <= 2.0 x ULN or direct bilirubin < 1.0
* Serum creatinine <= 2.5 x ULN
* Absolute neutrophil count >= 500/µL
* Platelet count >= to 20,000/µL
* Females of childbearing potential must have a negative pregnancy test within 4 days of entering the extension protocol.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* A delay of 4 weeks or more since the last preceding dose of CYT387 on the CCL09101 core study.
* Any chemotherapy (e.g., hydroxyurea), immunomodulatory drug therapy (e.g., thalidomide), immunosuppressive therapy, corticosteroids > 10 mg/day prednisone or equivalent, or growth factor treatment (e.g., erythropoietin) within 14 days prior to initiation of study drug.
* Incomplete recovery from major surgery within four weeks of study entry.
* Radiation therapy within four weeks of study entry.
* Women of childbearing potential, unless surgically sterile for at least 3 months (i.e., hysterectomy), OR postmenopausal for at least 12 months (FSH > 30 U/mL), OR unless they agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through end of study. Permitted methods for preventing pregnancy must be communicated to study subjects and their understanding confirmed.
* Men who partner with a woman of childbearing potential, unless they agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through to the end of study. Permitted methods for preventing pregnancy must be communicated to study subjects and their understanding confirmed.
* Females who are pregnant or are currently breastfeeding.
* Known positive status for HIV.
* Clinically active hepatitis B or C.
* Diagnosis of another malignancy unless free of disease for at least three years following therapy with curative intent. Patients with early-stage basal cell or squamous cell skin cancer, cervical intraepithelial neoplasia, cervical carcinoma in situ or superficial bladder cancer may be eligible to participate at the Investigator's discretion.
* Any acute active infection.
* Cardiac dysrhythmias requiring treatment, or prolongation of the QTc (Fridericia) interval to >450 msec for males or >470 msec for females at pre-study screening, unless attributable to pre-existing bundle branch block.
* Presence of >= Grade 2 peripheral neuropathy.
* Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), uncontrolled or unstable angina, myocardial infarction, cerebrovascular accident, or pulmonary embolism within 3 months prior to initiation of study drug.
* Uncontrolled inter current illness or any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/11/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/06/2014
Sample size
Target
Accrual to date
Final
120
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Minnesota
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sierra Oncology LLC - a GSK company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This extension protocol to the core study CCL09101 allows patients who have tolerated the drug and derived a clinical benefit, to continue to receive treatment beyond the 9 cycles of the core protocol. Long term safety and efficacy of CYT387 (momelotinib) will be evaluated.
Trial website
https://clinicaltrials.gov/study/NCT01236638
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ayalew Tefferi, MD
Address 0 0
Mayo Clinic
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01236638