Myeloproliferative DisordersPhase 1NCT01134120
A Study in Myeloproliferative Disorders
A Phase 1 study testing LY2784544 in people with Myeloproliferative Disorders and Thrombocythemia, Essential, run by Eli Lilly and Company at 2 sites in United States.
Trial summary
The purpose of this study is to find out the safe dose range of the study drug in patients with myeloproliferative disorders. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myeloproliferative Disorders, Thrombocythemia, Essential, Polycythemia Vera
- What is being tested?
- LY2784544
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2010to 22 Feb 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have a diagnosis of polycythemia vera (PV), essential thrombocythemia (ET), or myelofibrosis (MF) as defined by the World Health Organization (WHO) diagnostic criteria for myeloproliferative neoplasms and meet the following additional sub-type specific criteria:
- PV: has failed or is intolerant of standard therapies or refuses to take standard medications
- ET: has failed or is intolerant of standard therapies or refuses to take standard medications
- MF (patients with MF must meet at least one of the following):
- has intermediate or high-risk MF according to the Lille scoring system; or
- has symptomatic MF with spleen greater than 10 cm below left costal margin; or
Show all 10Show fewer
- has post-polycythemic MF; or
- has post-ET MF
- Have a quantifiable JAK2 V617F mutation
- Have discontinued all previous approved therapies for myeloproliferative disorders, including any chemotherapy, immunomodulating therapy (for example, thalidomide, interferon-alpha), immunosuppressive therapy (for example, corticosteroids greater than 10 mg/day prednisone or equivalent), radiotherapy, and erythropoietin, thrombopoietin, or granulocyte colony stimulating factor for at least 14 days and recovered from the acute effects of therapy. Hydroxyurea used to control blood cell counts is permitted at study entry if the subject has been maintained on a stable dose for at least 4 weeks. Low-dose acetylsalicylic acid (aspirin) is permitted as well
Who cannot join
- Have received treatment within 14 days of the initial dose of study drug with an experimental agent that has not received regulatory approval for any indication
- Are currently being treated with agents that are metabolized by CYP3A4 with a narrow therapeutic margin (for example, alfentanil, cyclosporine, diergotamine, ergotamine, fentanyl, pimozide, quinidine, sirolimus, and tacrolimus) or CYP2B6 (for example, cyclophosphamide, ifosfamide, tamoxifen, efavirenz, propofol, methadone, and bupropion)
- Are currently being treated with warfarin or one of its derivatives which is known to alter levels of protein C or protein S. An exception to this criterion will be allowed for patients with a prior history of Budd-Chiari Syndrome who are being treated with warfarin or one of its derivatives
Trial locations and contacts
2 sites in 1 country.
United States2 sites
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
Houston, Texas, 77030
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
Salt Lake City, Utah, 84132
About this study
Official title: A Phase 1 Study of LY2784544 in Patients With JAK2 V617F-Positive Myeloproliferative Disorders
The purpose of this study is to find out the safe dose range of the study drug in patients with myeloproliferative disorders.
Read the detailed description
The purpose of the study is to learn:
- How much and how often LY2784544 should be given to patients
- What is the safety profile of LY2784544 and any side effects that might be associated with it
- How LY2784544 is taken up, distributed, broken down, and passed out of your body
- Whether LY2784544 can help patients with myeloproliferative disorders
- If any markers in the blood (biomarkers) can identify patients who will respond better to the study drug.
The planned duration of the study is not fixed. The length of time patients participate in the study will be determined by the investigator/study doctor.
Part A of the study is to determine the dose of the study drug. Part A is divided into two sections, A1 and A2. In Part A1, patients will be given study drug without a lead-in period. In Parts A2 and B, patients will have a lead-in period of 2 or 4 weeks with a low dose of LY2784544 prior to taking a higher dose of LY2784544. Part B of the study is to confirm the safety of the dose and schedule.
- Conditions
- Myeloproliferative Disorders, Thrombocythemia, Essential, Polycythemia Vera, Primary Myelofibrosis
- Being tested
- LY2784544
- Medical subject headings
- Myeloproliferative Disorders, Thrombocythemia, Essential, Polycythemia Vera, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY2784544Drug
LY2784544 will be supplied as a capsule. The study drug will be taken by mouth daily while the patient is on study. In this study, different patients will be treated at different doses of LY2784544 until reaching the highest dose that patients can tolerate.
| Study group | Role | What they receive |
|---|---|---|
| LY2784544 | Experimental | Not described Receives: Drug: LY2784544 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of a recommended Phase 2 dosing regimen
Measured: Time of first dose until last dose
Number of participants with clinical significant effects
Measured: Time of first dose until last dose
Secondary outcomes (3)
Preliminary pharmacokinetics of LY2784544 (Cmax)
Measured: Part A1: Day 1,2,15, and 29; Part A2: Day 7, 14, 21, 28, 29, 56, and 57; Part B: Day 1, 29, 57, and 113
Preliminary pharmacokinetics of LY2784544 (AUC)
Measured: Part A1: Day 1,2,15, and 29; Part A2: Day 7, 14, 21, 28, 29, 56, and 57; Part B: Day 1, 29, 57, and 113
Malignant clone burden
Measured: Part A1: Baseline (2 times), Weeks 13, 21 and every 6 months while patient is on study; Parts A2 and B: Baseline (2 times), Weeks 5, 8, 17, 25 and every 6 months while patient is on study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2010 (estimated)
- Study startApr 2010
- Primary completionApr 2014
- Study completion22 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myeloproliferative DisordersStudies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01134120
- Sponsor's protocol ID
- 13201
- Other IDs
- I3X-MC-JHTA
- Status last verified
- Apr 2018
- First submitted
- 27 May 2010
- Last updated
- 17 Apr 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Verstovsek S, Mesa RA, Salama ME, Li L, Pitou C, Nunes FP, Price GL, Giles JL, D'Souza DN, Walgren RA, Prchal JT. A phase 1 study of the Janus kinase 2 (JAK2)V617F inhibitor, gandotinib (LY2784544), in patients with primary myelofibrosis, polycythemia vera, and essential thrombocythemia. Leuk Res. 2017 Oct;61:89-95. doi: 10.1016/j.leukres.2017.08.010. Epub 2017 Aug 31.
PubMed 28934680
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of LY2784544 in Patients With JAK2 V617F-Positive Myeloproliferative Disorders
- ClinicalTrials.gov ID
- NCT01134120
- Acronym / sponsor ID
- 13201
- Conditions
- Myeloproliferative Disorders, Thrombocythemia, Essential, Polycythemia Vera, Primary Myelofibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Eli Lilly and CompanyIndustry (pharma, biotech, devices)
- Study start
- Apr 2010
- Primary completion
- Apr 2014 (actual)
- Study completion
- 22 Feb 2018 (actual)
- First posted
- 31 May 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to find out the safe dose range of the study drug in patients with myeloproliferative disorders.
Detailed description
The purpose of the study is to learn:
- How much and how often LY2784544 should be given to patients
- What is the safety profile of LY2784544 and any side effects that might be associated with it
- How LY2784544 is taken up, distributed, broken down, and passed out of your body
- Whether LY2784544 can help patients with myeloproliferative disorders
- If any markers in the blood (biomarkers) can identify patients who will respond better to the study drug.
The planned duration of the study is not fixed. The length of time patients participate in the study will be determined by the investigator/study doctor.
Part A of the study is to determine the dose of the study drug. Part A is divided into two sections, A1 and A2. In Part A1, patients will be given study drug without a lead-in period. In Parts A2 and B, patients will have a lead-in period of 2 or 4 weeks with a low dose of LY2784544 prior to taking a higher dose of LY2784544. Part B of the study is to confirm the safety of the dose and schedule.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LY2784544 | Experimental | Drug: LY2784544 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Have a diagnosis of polycythemia vera (PV), essential thrombocythemia (ET), or myelofibrosis (MF) as defined by the World Health Organization (WHO) diagnostic criteria for myeloproliferative neoplasms and meet the following additional sub-type specific criteria:
- PV: has failed or is intolerant of standard therapies or refuses to take standard medications
- ET: has failed or is intolerant of standard therapies or refuses to take standard medications
- MF (patients with MF must meet at least one of the following):
- has intermediate or high-risk MF according to the Lille scoring system; or
Key exclusion criteria (3 listed)
- Have received treatment within 14 days of the initial dose of study drug with an experimental agent that has not received regulatory approval for any indication
- Are currently being treated with agents that are metabolized by CYP3A4 with a narrow therapeutic margin (for example, alfentanil, cyclosporine, diergotamine, ergotamine, fentanyl, pimozide, quinidine, sirolimus, and tacrolimus) or CYP2B6 (for example, cyclophosphamide, ifosfamide, tamoxifen, efavirenz, propofol, methadone, and bupropion)
- Are currently being treated with warfarin or one of its derivatives which is known to alter levels of protein C or protein S. An exception to this criterion will be allowed for patients with a prior history of Budd-Chiari Syndrome who are being treated with warfarin or one of its derivatives
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LY2784544LY2784544 will be supplied as a capsule. The study drug will be taken by mouth daily while the patient is on study. In this study, different patients will be treated at different doses of LY2784544 until reaching the highest dose that patients can tolerate. | Drug | LY2784544 |
Primary outcome
Determination of a recommended Phase 2 dosing regimen
Time frame: Time of first dose until last dose
Number of participants with clinical significant effects
Time frame: Time of first dose until last dose
3 secondary outcomes
Preliminary pharmacokinetics of LY2784544 (Cmax)
Time frame: Part A1: Day 1,2,15, and 29; Part A2: Day 7, 14, 21, 28, 29, 56, and 57; Part B: Day 1, 29, 57, and 113
Preliminary pharmacokinetics of LY2784544 (AUC)
Time frame: Part A1: Day 1,2,15, and 29; Part A2: Day 7, 14, 21, 28, 29, 56, and 57; Part B: Day 1, 29, 57, and 113
Malignant clone burden
Time frame: Part A1: Baseline (2 times), Weeks 13, 21 and every 6 months while patient is on study; Parts A2 and B: Baseline (2 times), Weeks 5, 8, 17, 25 and every 6 months while patient is on study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2018.
- Derived from this study: PubMed 28934680Verstovsek S, Mesa RA, Salama ME, Li L, Pitou C, Nunes FP, Price GL, Giles JL, D'Souza DN, Walgren RA, Prchal JT. A phase 1 study of the Janus kinase 2 (JAK2)V617F inhibitor, gandotinib (LY2784544), in patients with primary myelofibrosis, polycythemia vera, and essential thrombocythemia. Leuk Res. 2017 Oct;61:89-95. doi: 10.1016/j.leukres.2017.08.010. Epub 2017 Aug 31.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) study director
Eli Lilly and Company
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LY2784544 in people with Myeloproliferative Disorders and Thrombocythemia, Essential, run by Eli Lilly and Company at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determination of a recommended Phase 2 dosing regimen (time frame: Time of first dose until last dose). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01134120, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2018; AzTrials last refreshed its copy on 27 Oct 2026.