Sickle Cell DiseasePhase 2NCT02633397
A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases
A Phase 2 study testing Riociguat in people with Sickle Cell Disease, run by Mark Gladwin at 20 sites in United States.
Trial summary
The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with sickle cell disease (SCD). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Riociguat
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 130 participants
- Where
- 20 sitesUnited States
- Study dates
- 11 Apr 2017to 4 May 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years
- Sickling disorder (HbSS, HbSC, HbSbeta-thalassemia, HbSD, HbSO-Arab documented by hemoglobin electrophoresis or HPLC fractionation)
- At least one of the following findings: a. Systolic blood pressure ≥ 130 mm Hg on at least two occasions at least 1 day apart (one of these may be by history), b. Macroalbuminuria as manifested by urine albumin to creatinine ratio > 300 mg/g, c. Tricuspid regurgitant velocity (TRV) > 2.9 m/sec measured by echocardiography d. NT-proBNP level ≥ 160 pg/mL e. Urinalysis protein 1 + or higher.
- Females of reproductive potential (FRP) must have a negative, pre-treatment pregnancy test. Post-menopausal women (defined as no menses for at least 1 year or post-surgical from bilateral oophorectomy) are not required to undergo a pregnancy test.
- Females of reproductive potential must agree to use reliable contraception when sexually active. Adequate contraception is defined as any combination of at least 2 effective methods of birth control, of which at least one is a physical barrier (e.g. condoms with hormonal contraception or implants or combined oral contraceptives, certain intrauterine devices). Adequate contraception is required beginning at the signing of the informed consent form until one month after the last dose of riociguat.
- Patients must be willing to provide a blood sample for DNA analysis.
Who cannot join
- Pregnant or breast feeding women
- Patients with severe hepatic impairment defined as Child Pugh C
- End stage renal disease requiring dialysis
- Patients with eGFR <30 mL/min/1.73m, where GFR is estimated based on CKD-epi equation
- Patients on phosphodiesterase type 5 inhibitors (PDE-5) (such as sildenafil, tadalafil, vardenafil) and nonspecific PDE inhibitors (such as dipyridamole or theophylline) or nitrates
- Patients on strong cytochrome P450 (CYP) and P-glycoprotein 1(P-gp)/BCRP inhibitors such as systemic azole antimycotics (eg: ketoconazole, itraconazole), or HIV protease inhibitors (such as ritonavir)
Show all 14Show fewer
- Patients on St. John's Wort
- If patients are taking antihypertensive drugs, hydroxyurea, L-glutamine, crizanlizumab, or voxelotor prior to enrollment, they are excluded until the dose level is stable for at least three months
- Systolic blood pressure <95 mm Hg at Screening Visit 1 or 2 or Week 0 before randomization
- Current enrollment in an investigational new drug trial. Patients are eligible for enrollment 30 days after the last dose of an investigational drug has been received
- Evidence of qualitative urine drug test at screening for cocaine, phencyclidine (PCP), heroin, or amphetamines within three months prior to enrollment
- Patients who have recently (last six months) experienced serious bleeding from the lung or have undergone a bronchial arterial embolization procedure.
- Pulmonary hypertension associated with Idiopathic Interstitial Pneumonias
- Medical disorder, condition, or history that in the investigator's judgment would impair the patient's ability to participate or complete this study or render the patient to be inappropriate for enrollment
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
United States20 sites
Emory University School of Medicine
Atlanta, Georgia, 30329
Johns Hopkins University
Baltimore, Maryland, 21205
Boston University Medical Center
Boston, Massachusetts, 02118
New York Presbyterian Brooklyn Methodist Hospital
Brooklyn, New York, 11225
UNC Comprehensive Sickle Cell Center
Chapel Hill, North Carolina, 27599-7305
Medical University of South Carolina
Charleston, South Carolina, 29425
University of Illinois, Chicago
Chicago, Illinois, 60612
Ohio State University
Columbus, Ohio, 43210
University of Texas Southwestern
Dallas, Texas, 75390
Duke University
Durham, North Carolina, 27710
East Carolina University
Greenville, North Carolina, 27834
Indiana University
Indianapolis, Indiana, 46202
University of Tennessee Health Science Center
Memphis, Tennessee, 38163
University of Miami
Miami, Florida, 33136
Tulane University
New Orleans, Louisiana, 70112
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
UPMC Division of Hematology and Oncology
Pittsburgh, Pennsylvania, 15233
Virginia Commonwealth University Medical Center
Richmond, Virginia, 23298
Albert Einstein University/ Montefiore Medical Center
The Bronx, New York, 10467
Howard University
Washington D.C., District of Columbia, 20060
About this study
Official title: A Phase II Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riociguat in Patients With Sickle Cell Diseases
The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with sickle cell disease (SCD).
Read the detailed description
This randomized study involves 12 weeks of treatment with riociguat pills or placebo pills, and a follow-up period of 30 days after treatment. The dose is adjusted every 2 weeks based on systolic blood pressure (SBP) and well-being assessed at that visit. Physical examinations, vital signs, blood tests and questionnaires will be performed at 2 week intervals during the double blinded study treatment. Echocardiogram, urine testing, six-minute walk distance and questionnaires will be assessed at the beginning and end of the treatment phase.
- Conditions
- Sickle Cell Disease
- Being tested
- Riociguat, Placebo
- Keywords
- SCD, Sickle Cell Disease, Riociguat, Adempas
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RiociguatDrug
Also called Adempas
Riociguat 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg three times a day for 12 weeks
PlaceboDrug
Matching placebo to riociguat 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg three times a day for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Riociguat | Experimental | Treatment Arm Receives: Drug: Riociguat |
| Placebo | Placebo comparator | Placebo Arm Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Incidence of Treatment Emergent Severe Adverse Events (SAE)
The primary endpoint was the proportion of participants who experienced at least 1 treatment-emergent serious adverse event (SAE), which was defined as any event occurring after the baseline visit (i.e., after initiation of study drug), adjudicated by a designated committee of protocol investigators and outside experts. SAEs were considered to be treatment-emergent if they started or worsened after the first dose of study medication and up to 7 days after end of study treatment.
Measured: Baseline through 7 days after discontinuation of treatment, up to 13 Weeks
Secondary outcomes (22)
Frequency of SAE Due to Sickle Cell Related Painful Crisis
The proportion of participants with treatment-emergent SAE for sickle-cell related crises (Vaso-occlusive crises)
Measured: Baseline through 7 days after discontinuation of treatment, up to 13 Weeks
Overall Incidences of Treatment-emergent Adverse Events (AEs)
Proportion of participants that experienced treatment-emergent AEs
Measured: Baseline to Week 12
Incidences of Sickle Cell Related Clinical Complications
Incidence of Vaso-occlusive Crisis (VOC) between baseline and week 12
Measured: Baseline to Week 12
Pain Intensity Using the Brief Pain Inventory
Brief Pain Inventory (BPI) short form - score ranges from 0 (no pain) to 10 (Pain as bad as you can imagine)
Mean at 12 weeks assessed using ANCOVA adjusting for baseline.
Measured: Baseline, 12 weeks
6-minute Walk Distance
6-minute walk distance was used to assess functional exercise capacity
Mean at 12 weeks assessed using ANCOVA adjusting for baseline.
Measured: Baseline, 12 weeks
Changes in the Dyspnea Borg Scale
Baseline to 12 weeks mean change of the Borg dyspnea scores, post 6MWT, using a linear regression model.
Dyspnea Borg score was used to measure the level of severity of breathlessness perceived by the patient at the end of the 6MWD Test. The severity is measured on a 10-point scale with 0= nothing at all and 10=maximum severity of breathlessness.
Measured: Baseline,12 Weeks
Fatigue Borg Scale
Fatigue Borg score was used to rank the participant's exertion at the end of the 6MWD Test. The rate of exertion was given according to a scale ranging from 0 (nothing at all) to 10=maximum severity of exertion
Mean at 12 weeks assessed using ANCOVA adjusting for baseline.
Measured: Baseline,12 weeks
Changes in Blood Pressure as the Main Pharmacodynamic (MAP)
Baseline to 12 weeks mean change of MAP, estimated with a repeated measures analysis (Baseline, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks) using a linear mixed model.
Measured: Baseline, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks
Tricuspid Regurgitant Velocity (TRV) Using Non-invasive Echocardiography
Mean TRV at 12 weeks assessed using ANCOVA, adjusting for baseline.
Measured: Baseline, 12 Weeks
End-systolic Volume Using Non-invasive Echocardiography
Mean end-systolic volume (biplane) at 12 weeks, assessed using ANCOVA adjusting for baseline.
Measured: Baseline,12 weeks
Ejection Fraction (Biplane) Using Non-invasive Echocardiography
Mean ejection fraction (biplane) at 12 weeks, assessed using ANCOVA adjusting for baseline.
Measured: Baseline, 12 weeks
Changes in the Levels of Plasma NT-proBNP
Mean Change in the levels of plasma NT-proBNP from baseline to 12 weeks using a linear regression model.
Measured: Baseline,12 weeks
Changes in Albumin/Creatinine Ratio
Albumin/Creatinine Ratio (ACR) mean change from baseline to 12 weeks using linear regression model.
Measured: Baseline,12 weeks
Microalbuminuria
Odds ratio per week of microalbuminuria estimated with a repeated measures analysis (baseline, 12 weeks) using a generalized linear mixed model without random slope
Measured: Baseline,12 weeks
Macroalbuminuria
Odds ratio per week of macroalbuminuria estimated with a repeated measures analysis (baseline, 12 weeks) using a generalized linear mixed model without random slope.
Measured: Baseline,12 weeks
Changes in Glomerular Filtration Rate
Mean change in GFR to 12 weeks, estimated with a repeated measures analysis (Baseline, 4 weeks, 8 weeks, 12 weeks) a using a linear mixed model.
Measured: Baseline, 4 weeks, 8 weeks, 12 weeks
Chronic Kidney Disease (CKD) Stage - Low Risk Versus at Least Moderately Increased Risk
Odds ratio (OR) per week of low-risk CKD stage versus at least moderately increased risk (includes moderately increased risk CKD, high increased risk CKD, and very high increased risk CKD) estimated with a repeated measures analysis (baseline, 12 weeks) using a generalized linear mixed model without random slope
Measured: Baseline,12 weeks
Changes in Hemoglobin
Mean change in hemoglobin to 12 weeks, estimated with a repeated measures analysis (Baseline, 4 weeks, 8 weeks, 12 weeks) a using a linear mixed model.
Measured: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in Reticulocyte Count
Mean change in reticulocyte count to 12 weeks, estimated with a repeated measures analysis (Baseline, 4 weeks, 8 weeks, 12 weeks) a using a linear mixed model.
Measured: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in White Blood Cell Count
Mean change in White Blood Cell count to 12 weeks estimated with a repeated measures analysis (Baseline, 4 weeks, 8 weeks, 12 weeks) a using a linear mixed model.
Measured: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in Lactate Dehydrogenase (LDH)
Mean change in LDH to 12 weeks estimated with a repeated measures analysis (Baseline, 4 weeks, 8 weeks, 12 weeks) a using a linear mixed model.
Measured: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in Fetal Hemoglobin
Mean change in fetal hemoglobin from baseline to 12 weeks using a linear mixed model
Measured: Baseline,12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2015 (estimated)
- Study start11 Apr 2017
- Primary completion4 May 2022
- Study completion4 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 130 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mark Gladwin
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Mark Gladwin, University of Pittsburgh
- Investigators
- Mark Gladwin, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02633397
- Sponsor's protocol ID
- PRO15110016
- Status last verified
- Jul 2023
- First submitted
- 7 Dec 2015
- Last updated
- 19 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Gladwin MT, Gordeuk VR, Desai PC, Minniti C, Novelli EM, Morris CR, Ataga KI, De Castro L, Curtis SA, El Rassi F, Ford HJ, Harrington T, Klings ES, Lanzkron S, Liles D, Little J, Nero A, Smith W, Taylor JG 6th, Baptiste A, Hagar W, Kanter J, Kinzie A, Martin T, Rafique A, Telen MJ, Lalama CM, Kato GJ, Abebe KZ. Riociguat in patients with sickle cell disease and hypertension or proteinuria (STERIO-SCD): a randomised, double-blind, placebo controlled, phase 1-2 trial. Lancet Haematol. 2024 May;11(5):e345-e357. doi: 10.1016/S2352-3026(24)00045-0. Epub 2024 Mar 27.
PubMed 38554715Azbell RCG, Desai PC. Treatment dilemmas: strategies for priapism, chronic leg ulcer disease, and pulmonary hypertension in sickle cell disease. Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):411-417. doi: 10.1182/hematology.2021000275.
PubMed 34889382
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Randomized, Double-Blind, Placebo-Controlled Multi-Center Study to Assess the Safety, Tolerability, and Efficacy of Riociguat in Patients With Sickle Cell Diseases
- ClinicalTrials.gov ID
- NCT02633397
- Acronym / sponsor ID
- PRO15110016
- Conditions
- Sickle Cell Disease
- Keywords
- SCD, Sickle Cell Disease, Riociguat, Adempas
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 130 participants (actual)
- Lead sponsor
- Mark GladwinUniversity, hospital or other
- Study start
- 11 Apr 2017 (actual)
- Primary completion
- 4 May 2022 (actual)
- Study completion
- 4 May 2022 (actual)
- First posted
- 17 Dec 2015 (estimated)
- Results
- Posted 19 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with sickle cell disease (SCD).
Detailed description
This randomized study involves 12 weeks of treatment with riociguat pills or placebo pills, and a follow-up period of 30 days after treatment. The dose is adjusted every 2 weeks based on systolic blood pressure (SBP) and well-being assessed at that visit. Physical examinations, vital signs, blood tests and questionnaires will be performed at 2 week intervals during the double blinded study treatment. Echocardiogram, urine testing, six-minute walk distance and questionnaires will be assessed at the beginning and end of the treatment phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Riociguat | Experimental | Drug: RiociguatTreatment Arm |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo Arm |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age ≥ 18 years
- Sickling disorder (HbSS, HbSC, HbSbeta-thalassemia, HbSD, HbSO-Arab documented by hemoglobin electrophoresis or HPLC fractionation)
- At least one of the following findings: a. Systolic blood pressure ≥ 130 mm Hg on at least two occasions at least 1 day apart (one of these may be by history), b. Macroalbuminuria as manifested by urine albumin to creatinine ratio > 300 mg/g, c. Tricuspid regurgitant velocity (TRV) > 2.9 m/sec measured by echocardiography d. NT-proBNP level ≥ 160 pg/mL e. Urinalysis protein 1 + or higher.
- Females of reproductive potential (FRP) must have a negative, pre-treatment pregnancy test. Post-menopausal women (defined as no menses for at least 1 year or post-surgical from bilateral oophorectomy) are not required to undergo a pregnancy test.
- Females of reproductive potential must agree to use reliable contraception when sexually active. Adequate contraception is defined as any combination of at least 2 effective methods of birth control, of which at least one is a physical barrier (e.g. condoms with hormonal contraception or implants or combined oral contraceptives, certain intrauterine devices). Adequate contraception is required beginning at the signing of the informed consent form until one month after the last dose of riociguat.
Key exclusion criteria (14 listed)
- Pregnant or breast feeding women
- Patients with severe hepatic impairment defined as Child Pugh C
- End stage renal disease requiring dialysis
- Patients with eGFR <30 mL/min/1.73m, where GFR is estimated based on CKD-epi equation
- Patients on phosphodiesterase type 5 inhibitors (PDE-5) (such as sildenafil, tadalafil, vardenafil) and nonspecific PDE inhibitors (such as dipyridamole or theophylline) or nitrates
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RiociguatAlso: AdempasRiociguat 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg three times a day for 12 weeks | Drug | Riociguat |
| PlaceboMatching placebo to riociguat 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg three times a day for 12 weeks | Drug | Placebo |
Primary outcome
Overall Incidence of Treatment Emergent Severe Adverse Events (SAE)
Time frame: Baseline through 7 days after discontinuation of treatment, up to 13 Weeks
The primary endpoint was the proportion of participants who experienced at least 1 treatment-emergent serious adverse event (SAE), which was defined as any event occurring after the baseline visit (i.e., after initiation of study drug), adjudicated by a designated committee of protocol investigators and outside experts. SAEs were considered to be treatment-emergent if they started or worsened after the first dose of study medication and up to 7 days after end of study treatment.
22 secondary outcomes
Frequency of SAE Due to Sickle Cell Related Painful Crisis
Time frame: Baseline through 7 days after discontinuation of treatment, up to 13 Weeks
Overall Incidences of Treatment-emergent Adverse Events (AEs)
Time frame: Baseline to Week 12
Incidences of Sickle Cell Related Clinical Complications
Time frame: Baseline to Week 12
Pain Intensity Using the Brief Pain Inventory
Time frame: Baseline, 12 weeks
6-minute Walk Distance
Time frame: Baseline, 12 weeks
Changes in the Dyspnea Borg Scale
Time frame: Baseline,12 Weeks
Fatigue Borg Scale
Time frame: Baseline,12 weeks
Changes in Blood Pressure as the Main Pharmacodynamic (MAP)
Time frame: Baseline, 2 weeks, 4 weeks, 6 weeks, 8 weeks, 10 weeks, 12 weeks
Tricuspid Regurgitant Velocity (TRV) Using Non-invasive Echocardiography
Time frame: Baseline, 12 Weeks
End-systolic Volume Using Non-invasive Echocardiography
Time frame: Baseline,12 weeks
Ejection Fraction (Biplane) Using Non-invasive Echocardiography
Time frame: Baseline, 12 weeks
Changes in the Levels of Plasma NT-proBNP
Time frame: Baseline,12 weeks
Changes in Albumin/Creatinine Ratio
Time frame: Baseline,12 weeks
Microalbuminuria
Time frame: Baseline,12 weeks
Macroalbuminuria
Time frame: Baseline,12 weeks
Changes in Glomerular Filtration Rate
Time frame: Baseline, 4 weeks, 8 weeks, 12 weeks
Chronic Kidney Disease (CKD) Stage - Low Risk Versus at Least Moderately Increased Risk
Time frame: Baseline,12 weeks
Changes in Hemoglobin
Time frame: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in Reticulocyte Count
Time frame: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in White Blood Cell Count
Time frame: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in Lactate Dehydrogenase (LDH)
Time frame: Baseline, 4 weeks, 8 weeks,12 weeks
Changes in Fetal Hemoglobin
Time frame: Baseline,12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38554715Gladwin MT, Gordeuk VR, Desai PC, Minniti C, Novelli EM, Morris CR, Ataga KI, De Castro L, Curtis SA, El Rassi F, Ford HJ, Harrington T, Klings ES, Lanzkron S, Liles D, Little J, Nero A, Smith W, Taylor JG 6th, Baptiste A, Hagar W, Kanter J, Kinzie A, Martin T, Rafique A, Telen MJ, Lalama CM, Kato GJ, Abebe KZ. Riociguat in patients with sickle cell disease and hypertension or proteinuria (STERIO-SCD): a randomised, double-blind, placebo controlled, phase 1-2 trial. Lancet Haematol. 2024 May;11(5):e345-e357. doi: 10.1016/S2352-3026(24)00045-0. Epub 2024 Mar 27.
- Derived from this study: PubMed 34889382Azbell RCG, Desai PC. Treatment dilemmas: strategies for priapism, chronic leg ulcer disease, and pulmonary hypertension in sickle cell disease. Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):411-417. doi: 10.1182/hematology.2021000275.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark Gladwin, MD principal investigator
University of Pittsburgh
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Riociguat in people with Sickle Cell Disease, run by Mark Gladwin at 20 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Incidence of Treatment Emergent Severe Adverse Events (SAE) (time frame: Baseline through 7 days after discontinuation of treatment, up to 13 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02633397, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jul 2023.