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

Sponsor
Cyclerion Therapeutics (Industry)
Overall Status
Completed
CT.gov ID
NCT03285178
Collaborator
(none)
70
Enrollment
37
Locations
5
Arms
31
Actual Duration (Months)
1.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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
Phase 2

Study Design

Study Type:
Interventional
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 :
Dec 22, 2017
Actual Primary Completion Date :
Jul 22, 2020
Actual Study Completion Date :
Jul 22, 2020

Arms and Interventions

Arm Intervention/Treatment
Experimental: IW-1701 (Olinciguat) Low Dose

Drug: IW-1701
Oral Tablet
Other Names:
  • Olinciguat

    		•
    Experimental: IW-1701 (Olinciguat) Medium Dose

    Drug: IW-1701
    Oral Tablet
    Other Names:
  • Olinciguat

    		•
    Experimental: IW-1701 (Olinciguat) High Dose

    Drug: IW-1701
    Oral Tablet
    Other Names:
  • Olinciguat

    		•
    Experimental: IW-1701 (Olinciguat) Higher Dose

    Drug: IW-1701
    Oral Tablet
    Other Names:
  • Olinciguat

    		•
    Placebo Comparator: Placebo

    Drug: Placebo
    Oral Tablet

    Outcome Measures

    Primary Outcome Measures

    1. Incidence (number and percentage of patients) and frequency (number of events) of treatment-emergent adverse events (TEAE). [From first dose of study treatment through 7 days of last dose.]

    2. Incidence of treatment-emergent adverse events (TEAE) by severity [From first dose of treatment through 7 days of last dose.]

    3. Incidence of study drug related treatment-emergent adverse events (TEAE) [From first dose of treatment through 7 days of last dose.]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    16 Years to 70 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No

    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

    Locations

    Site City State Country Postal Code
    1 Children's Hospital of Orange County Orange California United States 92868
    2 MedStar Health Research Institute, MedStar Washington Hospital Center Washington District of Columbia United States 20010
    3 Howard University Center for Sickle Cell Disease Washington District of Columbia United States 20060
    4 Innovative Medical Research of South Florida, Inc. Aventura Florida United States 33180
    5 Century Clinical Research, Inc. Fort Lauderdale Florida United States 32117
    6 Foundation for Sickle Cell Disease Research Hollywood Florida United States 33021
    7 Omega Research Maitland, LLC Orlando Florida United States 32810
    8 Grady Memorial Hospital Atlanta Georgia United States 30303
    9 Atlanta Center for Medical Research Atlanta Georgia United States 30331
    10 University of Illinois at Chicago Chicago Illinois United States 60612
    11 Healthcare Research Network II, LLC Flossmoor Illinois United States 60422
    12 Clinical Trials of SWLA, LLC Lake Charles Louisiana United States 70601
    13 University of Maryland Medical Center Baltimore Maryland United States 21201
    14 Johns Hopkins School of Medicine Children's Center Baltimore Maryland United States 21205
    15 Boston Children's Hospital Boston Massachusetts United States 02115
    16 Children's Hospital of Michigan-Detroit Detroit Michigan United States 33021
    17 Healthcare Research Network Hazelwood Missouri United States 63042
    18 Hackensack University Medical Center, Pediatric Hematology and Oncology Hackensack New Jersey United States 07601
    19 Jacobi Medical Center Bronx New York United States 10461
    20 New York Medical College Valhalla New York United States 10595
    21 East Carolina University - Leo W. Jenkins Cancer Center Greenville North Carolina United States 27834
    22 East Carolina University Brody School of Medicine, Department of Pediatrics, Division of Pediatric Hematology Greenville North Carolina United States 27834
    23 Lynn Institute of Tulsa Tulsa Oklahoma United States 74105
    24 The Clinical Trial Center LLC Jenkintown Pennsylvania United States 19046
    25 University of Pittsburgh Medical Center Hillman Cancer Center Pittsburgh Pennsylvania United States 15232
    26 Accurate Clinical Research Baytown Texas United States 77521
    27 "UT Health Clinical Research Unit Center for Clinical and Translational Sciences Houston Texas United States 77030
    28 Mays Cancer Center UT Health San Antonio San Antonio Texas United States 78229
    29 Virginia Commonwealth University - Clinical Research Unit Richmond Virginia United States 23298
    30 Blood Center of Wisconsin (BCW) Wauwatosa Wisconsin United States 53226
    31 Hammoud Hospital University Medical Center Sidon Lebanon
    32 Nini Hospital Tripoli Lebanon
    33 Royal London Hospital London United Kingdom E1 2ES
    34 Whittington Hospital London United Kingdom N19 5NF
    35 Guys and St Thomas NHS Foundation Trust - Evelina London Childrens Hospital London United Kingdom SE1 7EH
    36 Guy's Hospital London United Kingdom SE1 9RT
    37 Hammersmith Hospital London United Kingdom W12 0NN

    Sponsors and Collaborators

    • Cyclerion Therapeutics

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Cyclerion Therapeutics
    ClinicalTrials.gov Identifier:
    NCT03285178
    Other Study ID Numbers:
    • C1701-202
    First Posted:
    Sep 15, 2017
    Last Update Posted:
    Jul 22, 2021
    Last Verified:
    Jul 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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:
    Anemia, Sickle Cell

    Study Results

    No Results Posted as of Jul 22, 2021