A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)
Condition: Sickle Cell Disease
MeSH terms: Anemia, Sickle Cell
Intervention: IW-1701; Placebo
Intervention description: Oral Tablet; Oral Tablet
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 88.0
Sex: All
Minimum age: 16 Years
Maximum age: 70 Years
Healthy volunteers: No
Keywords: Sickle Cell Disease; SCD; Olinciguat; IW-1701

Summary:

      The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and
      tolerability of different dose levels of IW-1701 compared with placebo when administered
      daily for approximately 12 weeks to patients with stable SCD. Exploratory objectives include
      evaluation of pharmacokinetic (PK) as well as evaluation of the effect of IW-1701 on symptoms
      of SCD, health-related quality of life, and biomarkers of pharmacodynamic (PD) activity.
    

Eligibility criteria:

        INCLUSION CRITERIA

          1. Patient is ambulatory male or female 16 to 70 years of age at the Screening Visit.

          2. Patient has SCD, including homozygous hemoglobin S (HbSS), hemoglobin SC disease
             (HbSC), heterozygous hemoglobin S-beta zero (HbSβ0)-thalassemia, or heterozygous
             hemoglobin S-beta plus (HbSβ+)-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. 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.

          6. 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.

          7. 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.

        EXCLUSION CRITERIA

          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 phosphodiesterase 5 (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.

        NOTE: Other inclusion and exclusion criteria apply, per protocol