MDS (Myelodysplastic Syndrome)Phase 1/2NCT02093429
Randomized Open-Label Study of INCB047986 in Subjects With Primary Myelodysplastic Syndrome (MDS)
A Phase 1/2 study testing INCB047986 in people with MDS (Myelodysplastic Syndrome), run by Incyte Corporation at 7 sites in United States.
Trial summary
The study design includes a 3-dose randomization phase to determine effective doses of INCB047986 in patients with myelodysplastic syndrome (MDS) who are refractory or unlikely to respond to erythropoiesis-stimulating agents (ESAs) followed by an extension phase. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- MDS (Myelodysplastic Syndrome)
- What is being tested?
- INCB047986
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 7 sitesUnited States
- Study dates
- Jan 2014to Sep 2014
- Phase
- Phase 1/2
- 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
- Subjects 18 years of age or older.
- Subjects must be diagnosed with MDS according to the World Health Organization (WHO) classification for de novo or primary MDS (Vardiman et al 2009).
- Subjects who require RBC transfusions or are either refractory to or unlikely to respond to ESA therapy should meet one of the following criteria:
- ESA failure as defined by no improvement in Hgb of at least 1.5 g/dL after 8 weeks of at least 40,000 IU per week of EPO (or equivalent).
- Have a serum erythropoietin (EPO) of ≥ 500 IU and Hgb level < 10.0 g/dL.
- Transfusion dependence defined as requiring at least 4 units of packed red blood cells (RBCs) for a Hgb of < 9 g/dL over the 8 weeks prior to screening.
- Subjects may not have received hypomethylating agents or immunosuppressive therapy for their MDS prior to this study.
Who cannot join
- Subjects at high risk for transformation to acute leukemia as evidenced by poor karyotype or peripheral blood blasts > 10%.
- Subjects with severely compromised bone marrow function as evidenced by trilineage cytopenias with anemia (Hgb < 10 g/L, platelets < 100 × 109/L, and absolute neutrophil count (ANC) < 1.8 × 109/L).
- Subjects who harbor the 5q deletion chromosomal aberration.
- Subjects with chronic myelomonocytic leukemia (CMML).
- Women who are pregnant or breastfeeding, and men and women who cannot comply with requirements to avoid fathering a child or becoming pregnant, respectively.
- Subjects with impaired liver function, end stage renal disease on dialysis, or clinically significant concurrent infections requiring therapy.
- Subjects with unstable cardiac function.
- Invasive malignancies over the previous 2 years except treated basal or squamous carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix, Stage 1 or 2 treated prostate
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Study site
Burlington, Vermont
Study site
Germantown, Tennessee
Study site
Highland, California
Study site
Houston, Texas
Study site
Indianapolis, Indiana
Study site
Morristown, New Jersey
Study site
Somerville, New Jersey
About this study
Official title: A Randomized, Open-Label, 2-Stage Study of INCB047986 Administered Orally to Subjects With Primary Myelodysplastic Syndrome (MDS) Refractory to or Unlikely to Respond to Erythropoiesis-Stimulating Agents (ESAs)
The study design includes a 3-dose randomization phase to determine effective doses of INCB047986 in patients with myelodysplastic syndrome (MDS) who are refractory or unlikely to respond to erythropoiesis-stimulating agents (ESAs) followed by an extension phase.
Read the detailed description
After 50% of the subjects have completed 16 weeks of treatment (10 subjects in each dose group), a planned interim analysis will be conducted to determine which, if any, dose levels warrant further investigation based on observing at least 3 responses for hematologic improvement in erythrocytes (HI-E) and adequate safety and tolerability.
Subjects who do not meet the response criteria for erythroid improvement may be treated with ESA in combination with INCB047986 for an additional 16 weeks.
The study comprises:
Screening: up to 4 weeks. Treatment Phase 1: at least 16 weeks of INCB047986. Treatment Phase 2: at least 8 and up to 16 weeks of INCB047986 in combination with ESA if failed treatment in Phase 1.
Extension Phase: Subjects receiving benefit from either therapy may continue indefinitely or until the study is terminated.
Follow-Up: 30 (± 7) days after the last dose of INCB047986 is taken.
- Conditions
- MDS (Myelodysplastic Syndrome)
- Being tested
- INCB047986
- Medical subject headings
- Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INCB047986Drug
INCB047986 will be supplied as tablets.
| Study group | Role | What they receive |
|---|---|---|
| INCB047986 4 mg | Experimental | Participants will receive INCB047986 4 mg once daily for at least 16 weeks. Receives: Drug: INCB047986 |
| INCB047986 6 mg | Experimental | Participants will receive INCB047986 6 mg once daily for at least 16 weeks. Receives: Drug: INCB047986 |
| INCB047986 10 mg | Experimental | Participants will receive INCB047986 10 mg once daily for at least 16 weeks. Receives: Drug: INCB047986 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects who achieve a response for Hematologic Improvement in Erythrocytes (HI-E) during any 8-week period within the first 16-week treatment period with INCB047986 Monotherapy.
Measured: Baseline to Week 16
Safety and tolerability of INCB047986 as assessed by summary of clinical laboratory assessments and summary of Adverse Events (AEs).
Measured: Up to 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2014 (estimated)
- Study startJan 2014
- Primary completionSep 2014
- Study completionSep 2014
Why it stopped: Business decision.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MDS (Myelodysplastic Syndrome)Studies of the same condition, open and under way.
- More trials run by Incyte CorporationThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Incyte Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- William V. Williams, M.D., Study director, Incyte Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02093429
- Sponsor's protocol ID
- INCB 47986-201
- Status last verified
- Jan 2018
- First submitted
- 3 Jan 2014
- Last updated
- 17 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-Label, 2-Stage Study of INCB047986 Administered Orally to Subjects With Primary Myelodysplastic Syndrome (MDS) Refractory to or Unlikely to Respond to Erythropoiesis-Stimulating Agents (ESAs)
- ClinicalTrials.gov ID
- NCT02093429
- Acronym / sponsor ID
- INCB 47986-201
- Conditions
- MDS (Myelodysplastic Syndrome)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business decision.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Incyte CorporationIndustry (pharma, biotech, devices)
- Study start
- Jan 2014
- Primary completion
- Sep 2014 (actual)
- Study completion
- Sep 2014 (actual)
- First posted
- 21 Mar 2014 (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 study design includes a 3-dose randomization phase to determine effective doses of INCB047986 in patients with myelodysplastic syndrome (MDS) who are refractory or unlikely to respond to erythropoiesis-stimulating agents (ESAs) followed by an extension phase.
Detailed description
After 50% of the subjects have completed 16 weeks of treatment (10 subjects in each dose group), a planned interim analysis will be conducted to determine which, if any, dose levels warrant further investigation based on observing at least 3 responses for hematologic improvement in erythrocytes (HI-E) and adequate safety and tolerability.
Subjects who do not meet the response criteria for erythroid improvement may be treated with ESA in combination with INCB047986 for an additional 16 weeks.
The study comprises:
Screening: up to 4 weeks. Treatment Phase 1: at least 16 weeks of INCB047986. Treatment Phase 2: at least 8 and up to 16 weeks of INCB047986 in combination with ESA if failed treatment in Phase 1.
Extension Phase: Subjects receiving benefit from either therapy may continue indefinitely or until the study is terminated.
Follow-Up: 30 (± 7) days after the last dose of INCB047986 is taken.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| INCB047986 4 mg | Experimental | Drug: INCB047986Participants will receive INCB047986 4 mg once daily for at least 16 weeks. |
| INCB047986 6 mg | Experimental | Drug: INCB047986Participants will receive INCB047986 6 mg once daily for at least 16 weeks. |
| INCB047986 10 mg | Experimental | Drug: INCB047986Participants will receive INCB047986 10 mg once daily for at least 16 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Subjects 18 years of age or older.
- Subjects must be diagnosed with MDS according to the World Health Organization (WHO) classification for de novo or primary MDS (Vardiman et al 2009).
- Subjects who require RBC transfusions or are either refractory to or unlikely to respond to ESA therapy should meet one of the following criteria:
- Subjects may not have received hypomethylating agents or immunosuppressive therapy for their MDS prior to this study.
Key exclusion criteria (8 listed)
- Subjects at high risk for transformation to acute leukemia as evidenced by poor karyotype or peripheral blood blasts > 10%.
- Subjects with severely compromised bone marrow function as evidenced by trilineage cytopenias with anemia (Hgb < 10 g/L, platelets < 100 × 109/L, and absolute neutrophil count (ANC) < 1.8 × 109/L).
- Subjects who harbor the 5q deletion chromosomal aberration.
- Subjects with chronic myelomonocytic leukemia (CMML).
- Women who are pregnant or breastfeeding, and men and women who cannot comply with requirements to avoid fathering a child or becoming pregnant, respectively.
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 |
|---|---|---|
| INCB047986INCB047986 will be supplied as tablets. | Drug | INCB047986 10 mg, INCB047986 4 mg, INCB047986 6 mg |
Primary outcome
Proportion of subjects who achieve a response for Hematologic Improvement in Erythrocytes (HI-E) during any 8-week period within the first 16-week treatment period with INCB047986 Monotherapy.
Time frame: Baseline to Week 16
Safety and tolerability of INCB047986 as assessed by summary of clinical laboratory assessments and summary of Adverse Events (AEs).
Time frame: Up to 16 weeks
Results and safety
The study was stopped early (reason given: Business decision.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2018.
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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.
William V. Williams, M.D. study director
Incyte Corporation
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing INCB047986 in people with MDS (Myelodysplastic Syndrome), run by Incyte Corporation at 7 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 7 inclusion and 8 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: stopped early, as of the sponsor's last update on 17 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: Business decision.). Results are not available in the source record.
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?
Proportion of subjects who achieve a response for Hematologic Improvement in Erythrocytes (HI-E) during any 8-week period within the first 16-week treatment period with INCB047986 Monotherapy. (time frame: Baseline to Week 16). 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?
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 Jan 2018.