A Randomized, Placebo-controlled, Phase 2 Study to Evaluate the Safety and Pharmacodynamics of Once-daily Oral IW-1701 in Patients with Stable Sickle Cell Disease
- Conditions
- Stable sickle cell diseaseSickle Cell Disease (SCD)
- Registration Number
- LBCTR2019091283
- Lead Sponsor
- Cyclerion Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 88
1. Patient is ambulatory male or female 16 to 70 years of age at the Screening Visit.
2. Patient has SCD, including HbSS, HbSC, HbSß0-thalassemia, or HbSß+-thalassemia, documented in their medical history
3. If patient is on medication(s) for SCD, such as hydroxyurea (HU), are on a stable regimen.
4. Per medical history and/or patient recall, patient has had at least 1 and no more than 10 sickle cell-related pain crises in the 12 months
before the Screening Visit and none occurring in the 4 weeks before the Randomization Visit.
5. Women of childbearing potential must have a negative pregnancy test prior to randomization and must agree to use protocol-specified
contraception from the Screening Visit through 90 days after the final dose of study drug.
6. Male patients must be surgically sterile by vasectomy (conducted =60 days before the Screening Visit or confirmed via sperm analysis) or must
agree to use protocol-specified contraception and agree to refrain from sperm donation from the Screening Visit through 90 days after the final
dose of study drug.
7. Patient completes daily eDiary entries for at least 10 days during the last 14 days of the Run in Period as assessed at the Randomization Visit.
1. Patient requires a program of prescheduled, regularly administered chronic blood transfusion therapy.
2. Patient has been hospitalized for an SCD-related complication in the 4 weeks before the
Randomization Visit.
3. Patient has taken opioid(s) >200 morphine mg equivalent/day within the 4 weeks before the
Randomization Visit.
4. Patient is taking aspirin =325 mg daily, P2Y12 inhibitors, any anticoagulant medication, specific inhibitors of phosphodiesterase 5
(PDE5), nonspecific inhibitors of PDE5, moderate or strong cytochrome P450 3A (CYP3A) inhibitors, any supplements for the treatment of
erectile dysfunction, riociguat, or nitrates or nitric oxide donors in any form.
5. Patient has major concurrent illness or medical condition that in the opinion of the Investigator would preclude participation in a clinical study.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ame: Safety and tolerability;Timepoints: 12 weeks;Measure: Incidence, frequency, and severity of TEAEs and study drug-related TEAEs
- Secondary Outcome Measures
Name Time Method ame: Hemodynamic Parameters;Timepoints: 12 weeks;Measure: blood pressure and pulse;Name: Pain Crisis Paramaters;Timepoints: 12 weeks;Measure: Time to first pain crisis, proportion and frequency of pain crisis;Name: Biomarkers;Timepoints: 12 weeks;Measure: Biomarker concentration changes;Name: Pharmacokinetic;Timepoints: 12 weeks;Measure: Plasma concentrations;Name: Patient-reported Outcomes;Timepoints: 12 weeks;Measure: Patient Questionnaires