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Trial details
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
Current status:
Approved
|
Date registered:
23/10/2019
Trial version(s)
History: 12/09/2019
History: 12/09/2019
Current: 12/09/2019
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2019091283
Protocol number
C1701-202
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
Primary sponsor
Cyclerion Therapeutics, Inc.
Primary sponsor: Country of origin
United States of America
Public 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
Acronym
STRONG SCD
Scientific 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
Acronym
STRONG SCD
Brief summary of the study: English
The primary objective of the C1701-202 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 sickle cell disease (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.
Brief summary of the study: Arabic
الهدف الأساسي من دراسة هو تقييم سلامة وتحمل مستويات جرعة مختلفة من C1701-202 STRONG SCD مقارنةً مع الدواء الارضائي عند تناوله يومياً لمدة ١٢ أسبوعاً تقريباً لدى مرضى داء الخلايا المنجلية المستقر. تشمل الأهداف الاستكشافية تقييم الحرائك الدوائية IW-1701 وكذلك تقييم تأثير (PK) على أعراض داء الخلايا المنجلية المستقر ونوعية الحياة المتعلقة بالصحة والمؤشرات الحيوية للنشاط الديناميكي الدوائي IW-1701 .(PD)
Health conditions/problem studied: Specify
Stable sickle cell disease
Interventions: Specify
Eligible patients will be stratified by hydroxyurea (HU) use (yes or no) and randomly assigned in a 3:1 ratio to receive IW-1701 once daily or placebo. Arm 1: IW-1701 (Olinciguat) -uptitration possible for patients who meet the conditions to begin taking the applicable higher dose. Arm 2: placebo.
Key inclusion and exclusion 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. 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.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
16
Key inclusion and exclusion criteria: Age maximum
70
Key inclusion and exclusion criteria: 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.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Safety
Trial scope: Specify scope
Study design: Allocation
Randomized controlled trial
Study design: Masking
Blinded (masking used)
Study design: Control
Placebo
Study phase
2
Study design: Purpose
Treatment
Study design: Assignment
Parallel
IMP has market authorization
No
Name of IMP
IW1701/olinciguat
Type of IMP
Cell therapy
Pharmaceutical class
soluble guanylate cyclase (sgc) stimulator
Therapeutic indication
Stable sickle cell disease
Therapeutic benefit
There remains considerable unmet medical need in SCD, not only for treatments that prevent painful crises and other acute complications, but also for treatments that address the daily symptoms of the disease, including chronic pain.
Biospecimen retention
Samples with DNA**
Biospecimen description
Optional genotyping testing. If patient agrees, a blood sample of 4 mL will be collected and stored. The test may help to better understand how the disease and related diseases work, the effet of IW-1701 and/or other medications on the body, how IW-1701 is processed by the body, who might benefit from IW-1701 and why some people have side effects from taking the drug but other people don't.
Target sample size
88
Actual enrollment target size
88
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
18/11/2019
Date of study closure: Type
Anticipated
Date of study closure: Date
31/07/2020
Recruitment status
Pending
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
Not applicable
Additional data URL
https://www.clinicaltrials.gov/ct2/show/NCT03285178
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
ClinicalTrials.gov
NCT03285178
Sources of Monetary or Material Support
Name
Cyclerion Therapeutics, Inc.
Secondary Sponsors
Name
None
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Dr. Gino
Girardi
100 Brandywine Boulevard
United States of America
+1 919 418 5164
gino.girardi@syneoshealth.com
Syneos Health (previsouly INC Research)
Scientific
Dr. Wissam
Houhou
Ghassan Hammoud Street, Saida
Lebanon
+961 70 874 770
dr_houhou_wissam@hotmail.com
Hammoud Hospital University Medical Center
Scientific
Dr. Adlette
Inati
El Maarad Street, Tripoli
Lebanon
+961 3 228 033
adlette.inati@lau.edu.lb
Nini Hospital
Scientific
Dr. Ali
Taher
Cairo Street, Beirut
Lebanon
+961 3 755 669
ataher@aub.edu.lb
American University of Beirut Medical Center
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hammoud Hospital University Medical Center
Dr. Wissam Houhou
Hematology and Oncology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hammoud Hospital University Medical Center
16/09/2019
Ghada Aoun
medical@hammoudhospital.org
+961 7 723 111 Ext 1956
Countries of Recruitment
Name
Lebanon
United Kingdom
United States of America
Health Conditions or Problems Studied
Condition
Code
Keyword
sickle cell disease
Sickle-cell disorders (D57)
Sickle Cell Disease (SCD)
Interventions
Intervention
Description
Keyword
Arm 1
Placebo
Placebo
Arm 2
IW-1701
olinciguat
Primary Outcomes
Name
Time points
Measure
Safety and tolerability
12 weeks
Incidence, frequency, and severity of TEAEs and study drug-related TEAEs
Key Secondary Outcomes
Name
Time points
Measure
Hemodynamic Parameters
12 weeks
blood pressure and pulse
Pain Crisis Paramaters
12 weeks
Time to first pain crisis, proportion and frequency of pain crisis
Biomarkers
12 weeks
Biomarker concentration changes
Pharmacokinetic
12 weeks
Plasma concentrations
Patient-reported Outcomes
12 weeks
Patient Questionnaires
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial