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A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD) (STRONG SCD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03285178
Recruitment Status : Completed
First Posted : September 15, 2017
Last Update Posted : July 22, 2021
Sponsor:
Information provided by (Responsible Party):
Cyclerion Therapeutics

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: IW-1701 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : December 22, 2017
Actual Primary Completion Date : July 22, 2020
Actual Study Completion Date : July 22, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: IW-1701 (Olinciguat) Low Dose Drug: IW-1701
Oral Tablet
Other Name: Olinciguat
Experimental: IW-1701 (Olinciguat) Medium Dose Drug: IW-1701
Oral Tablet
Other Name: Olinciguat
Experimental: IW-1701 (Olinciguat) High Dose Drug: IW-1701
Oral Tablet
Other Name: Olinciguat
Experimental: IW-1701 (Olinciguat) Higher Dose Drug: IW-1701
Oral Tablet
Other Name: Olinciguat
Placebo Comparator: Placebo Drug: Placebo
Oral Tablet


Outcome Measures
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Primary Outcome Measures :
  1. Incidence (number and percentage of patients) and frequency (number of events) of treatment-emergent adverse events (TEAE). [ Time Frame: From first dose of study treatment through 7 days of last dose. ]
  2. Incidence of treatment-emergent adverse events (TEAE) by severity [ Time Frame: From first dose of treatment through 7 days of last dose. ]
  3. Incidence of study drug related treatment-emergent adverse events (TEAE) [ Time Frame: From first dose of treatment through 7 days of last dose. ]

Eligibility Criteria
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Ages Eligible for Study:   16 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 HbSS, HbSC, HbSβ0-thalassemia, or 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 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

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03285178


Locations
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Sponsors and Collaborators
Cyclerion Therapeutics
More Information
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Responsible Party: Cyclerion Therapeutics
ClinicalTrials.gov Identifier: NCT03285178    
Other Study ID Numbers: C1701-202
First Posted: September 15, 2017    Key Record Dates
Last Update Posted: July 22, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cyclerion Therapeutics:
Sickle Cell Disease
SCD
Olinciguat
IW-1701
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn