Sickle Cell DiseasePhase 1NCT03132324
A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
A Phase 1 study testing INCB059872 in people with Sickle Cell Disease, run by Incyte Corporation at 7 sites in United States.
Trial summary
The purpose of this study was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- INCB059872
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 7 sitesUnited States
- Study dates
- 20 Apr 2017to 3 Oct 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
- Diagnosis of SCD (sickle cell SS) confirmed through hemoglobin electrophoresis.
- Must be red blood cell (RBC) transfusion-independent (not currently on regularly scheduled transfusions) for ≥ 3 months from the time of first dose of study drug.
- No RBC transfusion within 30 days of first dose of study drug.
- Hydroxyurea (HU) refractory
- -Must not have received HU therapy during the 3 months before receiving study drug.
- Creatinine clearance ≥ 60 mL/min based on the institutional formula.
- Willingness to avoid pregnancy or fathering children.
Who cannot join
- Any unresolved toxicity ≥ Grade 2 from previous therapy except for stable chronic toxicities not expected to resolve.
- Pregnant or nursing women or participants expecting to conceive or father children within the projected duration of the study, starting with screening visit through completion of safety follow-up.
- Received an investigational study drug within 28 days or 5 half-lives (whichever is longer) before receiving the first dose of study drug (requirement may be waived with medical monitor approval).
- Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment.
- Prior receipt of LSD1 inhibitor therapy for any indication.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Boston University
Boston, Massachusetts, 02215
University of Illinois at Chicago
Chicago, Illinois, 60607
Acevedo Clinical Research Associates
Miami, Florida, 33142
Advanced Pharma
Miami, Florida, 33147
Blood Centers of Wisconsin
Milwaukee, Wisconsin, 53226
Virginia Commonwealth University
Richmond, Virginia, 23298
Vita Health and Medical Center
Tamarac, Florida, 33319
About this study
Official title: A Phase 1 Open-Label, Dose-Escalation Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
The purpose of this study was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease.
- Conditions
- Sickle Cell Disease
- Being tested
- INCB059872
- Keywords
- Sickle cell disease (SCD), sickle cell SS, lysine demethylase 1 (LSD1) inhibition
- Medical subject headings
- Anemia, Sickle Cell, Inhibition, Psychological
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INCB059872Drug
INCB059872 tablets
| Study group | Role | What they receive |
|---|---|---|
| INCB059872 0.5 mg | Experimental | INCB059872 0.5 mg tablet administered orally every other day (QOD) for 28 days on an empty stomach. If dose was well tolerated, once daily (QD) administration was evaluated independently and in parallel with QOD administration. Receives: Drug: INCB059872 |
| INCB059872 1 mg | Experimental | INCB059872 1 mg tablet administered orally QOD for 28 days on an empty stomach. If dose was well tolerated, QD administration was evaluated independently and in parallel with QOD administration. Receives: Drug: INCB059872 |
| INCB059872 2 mg | Experimental | INCB059872 2 mg tablet administered orally QOD for 28 days on an empty stomach. If dose was well tolerated, QD administration was evaluated independently and in parallel with QOD administration. Receives: Drug: INCB059872 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of INCB059872 assessed by monitoring frequency, duration, and severity of adverse events
An adverse event is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurs after a participant provides informed consent.
Measured: Screening through 35 days after end of treatment, up to approximately 3 months per participant.
Change in fetal hemoglobin (HbF) from baseline
Pharmacodynamic activity assessed by measuring changes of HbF from baseline and their correlation to INCB059872 treatment. The HbF (F cells) in human whole blood will be characterized using flow cytometry.
Measured: Baseline through 2 weeks after end of treatment, up to approximately 2.5 months per participant.
Secondary outcomes (2)
Cmax of INCB059872
Defined as maximum observed plasma concentration.
Measured: Baseline to Day 28.
AUC0-t of INCB059872
Defined as area under the single-dose plasma concentration-time curve from Hour 0 to the last quantifiable measurable plasma concentration.
Measured: Baseline to Day 28.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2017
- Study start20 Apr 2017
- Primary completion3 Oct 2018
- Study completion3 Oct 2018
Why it stopped: This study is terminated due to a business decision not to pursue INCB059782 in Sickle Cell Disease indication.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Incyte Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Fitzroy Dawkins, MD, Study director, Incyte Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03132324
- Sponsor's protocol ID
- INCB 59872-102
- Status last verified
- Oct 2019
- First submitted
- 24 Apr 2017
- Last updated
- 28 Oct 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-Label, Dose-Escalation Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT03132324
- Acronym / sponsor ID
- INCB 59872-102
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle cell disease (SCD), sickle cell SS, lysine demethylase 1 (LSD1) inhibition
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study is terminated due to a business decision not to pursue INCB059782 in Sickle Cell Disease indication.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Incyte CorporationIndustry (pharma, biotech, devices)
- Study start
- 20 Apr 2017 (actual)
- Primary completion
- 3 Oct 2018 (actual)
- Study completion
- 3 Oct 2018 (actual)
- First posted
- 27 Apr 2017 (actual)
- 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 was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| INCB059872 0.5 mg | Experimental | Drug: INCB059872INCB059872 0.5 mg tablet administered orally every other day (QOD) for 28 days on an empty stomach. If dose was well tolerated, once daily (QD) administration was evaluated independently and in parallel with QOD administration. |
| INCB059872 1 mg | Experimental | Drug: INCB059872INCB059872 1 mg tablet administered orally QOD for 28 days on an empty stomach. If dose was well tolerated, QD administration was evaluated independently and in parallel with QOD administration. |
| INCB059872 2 mg | Experimental | Drug: INCB059872INCB059872 2 mg tablet administered orally QOD for 28 days on an empty stomach. If dose was well tolerated, QD administration was evaluated independently and in parallel with QOD administration. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosis of SCD (sickle cell SS) confirmed through hemoglobin electrophoresis.
- Must be red blood cell (RBC) transfusion-independent (not currently on regularly scheduled transfusions) for ≥ 3 months from the time of first dose of study drug.
- No RBC transfusion within 30 days of first dose of study drug.
- Hydroxyurea (HU) refractory
- -Must not have received HU therapy during the 3 months before receiving study drug.
Key exclusion criteria (5 listed)
- Any unresolved toxicity ≥ Grade 2 from previous therapy except for stable chronic toxicities not expected to resolve.
- Pregnant or nursing women or participants expecting to conceive or father children within the projected duration of the study, starting with screening visit through completion of safety follow-up.
- Received an investigational study drug within 28 days or 5 half-lives (whichever is longer) before receiving the first dose of study drug (requirement may be waived with medical monitor approval).
- Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment.
- Prior receipt of LSD1 inhibitor therapy for any indication.
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 |
|---|---|---|
| INCB059872INCB059872 tablets | Drug | INCB059872 0.5 mg, INCB059872 1 mg, INCB059872 2 mg |
Primary outcome
Safety and tolerability of INCB059872 assessed by monitoring frequency, duration, and severity of adverse events
Time frame: Screening through 35 days after end of treatment, up to approximately 3 months per participant.
An adverse event is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurs after a participant provides informed consent.
Change in fetal hemoglobin (HbF) from baseline
Time frame: Baseline through 2 weeks after end of treatment, up to approximately 2.5 months per participant.
Pharmacodynamic activity assessed by measuring changes of HbF from baseline and their correlation to INCB059872 treatment. The HbF (F cells) in human whole blood will be characterized using flow cytometry.
2 secondary outcomes
Cmax of INCB059872
Time frame: Baseline to Day 28.
AUC0-t of INCB059872
Time frame: Baseline to Day 28.
Results and safety
The study was stopped early (reason given: This study is terminated due to a business decision not to pursue INCB059782 in Sickle Cell Disease indication.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2019.
- Individual participant dataSharing planned: No
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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.
Fitzroy Dawkins, MD 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 study testing INCB059872 in people with Sickle Cell Disease, 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 5 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 28 Oct 2019. 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 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 was stopped early (reason given: This study is terminated due to a business decision not to pursue INCB059782 in Sickle Cell Disease indication.). 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?
Safety and tolerability of INCB059872 assessed by monitoring frequency, duration, and severity of adverse events (time frame: Screening through 35 days after end of treatment, up to approximately 3 months per participant.). 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 28 Oct 2019.