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 - Strong SCD

Cyclerion Therapeutics, Inc.0 sites88 target enrollmentAugust 29, 2019

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Not specified
Sponsor: Cyclerion Therapeutics, Inc.
Enrollment: 88
Status: Active, not recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

August 29, 2019

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Cyclerion Therapeutics, Inc.

Eligibility Criteria

Inclusion Criteria

•1\. Patient has provided written consent before any study\-specific procedures are performed; for patients \<18 years of age, parental permission and child assent will be obtained.
•2\. Patient is ambulatory male or female 16 to 70 years of age at the Screening Visit.
•3\. Patient has sickle cell disease (SCD), including HbSS, HbSC, HbSß0\-thalassemia, or HbSß\+\-thalassemia, documented in their medical history by hemoglobin electrophoresis or genotyping.
•4\. If receiving hydroxyurea (HU), erythropoietin, L\-glutamine, or prophylactic oral antibiotics, patient has had no change in regimen(s) (ie, drug and dose) for at least 8 weeks before the Randomization Visit and has no plans to change regimen(s) during the study. If receiving HU, patient must have been prescribed HU for at least 6 months prior to the Randomization Visit. (Note: Patient is not required to be taking medication\[s] for SCD.)
•Should the patient begin any new chronic treatment/therapy for SCD during the study or alter any current treatment/therapy for SCD during the study, the Sponsor Medical Monitor must be informed.
•5\. If receiving chronic medication(s) for hypertension, patient has had no change in regimen(s) (ie, drug and dose) for at least 8 weeks before the Randomization Visit and has no plans to change regimen(s) during the study.
•6\. 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.
•For assessing study eligibility, an SCD\-related pain crisis is defined as an acute episode of new\-onset pain that lasts \=2 hours with no medically determined cause other than a vaso\-occlusive event and requires presentation to a medical facility (eg, acute care setting,
•Emergency Department, urgent care clinic) and treatment with oral or parenteral opioids, parenteral nonsteroidal anti\-inflammatory drugs, or other analgesics, prescribed by a healthcare provider.
•7\. Patient has clinically acceptable (per Investigator discretion) electrocardiogram (ECG) with QT interval corrected using Fridericia’s formula (QTcF interval) \<500 ms at the Screening Visit.

Exclusion Criteria

•1\. Patient requires a program of prescheduled, regularly administered chronic blood transfusion therapy, has received a transfusion in the 8 weeks before the Randomization Visit, and/or is scheduled to receive a transfusion during the study.
•2\. Patient has been hospitalized for an SCD\-related complication in the 4 weeks before the Randomization Visit.
•3\. Patient is planning to undergo major surgery during the trial or has undergone surgery within 4 weeks of the Screening Visit, other than minor dermatologic procedures.
•4\. Patient has used oral or parenteral corticosteroids in the 8 weeks before the Randomization visit. Note: Transient use for \=2 days may be acceptable; consult the Medical Monitor for confirmation.
•5\. Patient has taken opioid(s) \>200 morphine mg equivalent/day within the 4 weeks before the Randomization Visit.
•6\. Patient is taking aspirin \=325 mg daily, any P2Y12 inhibitor, any anticoagulant medication, specific inhibitors of phosphodiesterase 5 (PDE5\), nonspecific inhibitors of PDE5 (including dipyridamole and theophylline), any supplements for the treatment of erectile dysfunction, riociguat, or nitrates or nitric oxide donors in any form.
•Patient is taking moderate or strong cytochrome P450 3A (CYP3A) inhibitors or inducers.
•These medications are prohibited from the Run\-in Visit (which may occur from Day \-14 to Day \-17\) through the duration of the study.
•See Appendix 1 of the study protocol for a list of prohibited medications, supplements, and foods.
•7\. Patient uses or requires the use of fentanyl.