CLINICAL TRIAL

Riociguat for Anemia, Sickle Cell

Recruiting · 18+ · All Sexes · Durham, NC

This study is evaluating whether a drug may help reduce the symptoms of sickle cell disease.

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About the trial for Anemia, Sickle Cell

Eligible Conditions
Anemia, Sickle Cell · Sickle Cell Disease (SCD)

Treatment Groups

This trial involves 2 different treatments. Riociguat is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Riociguat
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.
Placebo
DRUG

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Riociguat
FDA approved

Side Effect Profile for Riociguat 1.0 mg

Riociguat 1.0 mg
Show all side effects
Dizziness
9%
Middle ear barotrauma
9%
Shoulder pain
9%
Phlebitis
0%
Venous Thrombosis
0%
Bronchospasm
0%
This histogram enumerates side effects from a completed 2015 Phase 4 trial (NCT02024386) in the Riociguat 1.0 mg ARM group. Side effects include: Dizziness with 9%, Middle ear barotrauma with 9%, Shoulder pain with 9%, Phlebitis with 0%, Venous Thrombosis with 0%.

Eligibility

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Age ≥ 18 years
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.
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline to Week 12
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline to Week 12.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Riociguat will improve 1 primary outcome and 12 secondary outcomes in patients with Anemia, Sickle Cell. Measurement will happen over the course of Baseline to Week 12.

Overall incidence of treatment emergent severe adverse events (SAE)
BASELINE TO WEEK 12
Changes in pain intensity using numerical pain score
BASELINE TO WEEK 12
Changes in pain intensity using electronic daily pain diary piloted at selected sites
BASELINE TO WEEK 12
Incidences of sickle cell related clinical complications
BASELINE TO WEEK 12
Frequency of SAE due to sickle cell related painful crisis
BASELINE TO WEEK 12
Changes in blood pressure as the main pharmacodynamic variable
BASELINE TO WEEK 12
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes anemia, sickle cell?

Anemia is common in sickle cell disease. Although rare, severe anemia in an adolescent with sickle cell disease could be caused by red blood cell sequestration to the central nervous system by sickle cells that have undergone a change in their shape and become sickled. Anemia caused by sequestration to the central nervous system can be fatal, particularly in young children. The treatment options including iron therapy are discussed.

Anonymous Patient Answer

What are the signs of anemia, sickle cell?

Signs and symptoms of anemia include pallor, shortness of breath, lethargy, fatigue, light-headedness and dizziness. Diagnostic criteria for anemia in children should include low hemoglobin levels, low red cell mass, reticulocytosis, and erythropoietic dysfunction.

Anonymous Patient Answer

Can anemia, sickle cell be cured?

Sickle cell disease cannot be cured. However, this condition can be extremely well managed, sometimes to the point that symptoms can be almost nonexistent. Sickle cell hemoglobin S levels are a simple and inexpensive way to control blood levels in patients with SCD.

Anonymous Patient Answer

What are common treatments for anemia, sickle cell?

A large number of blood transfusions are common treatments for anemia. The use of aspirin or other antithrombotic agents can help patients to avoid unnecessary blood transfusions.\n

Anonymous Patient Answer

What is anemia, sickle cell?

Sickle cell anemia is a disease that occurs when there are changes in the anatomy of cells in the red blood cell of people with sickle cell disease, also called sickle cell anemia. It affects not only persons born with the condition but also a family history of sickle cell disease. A sudden drop in the number of red blood cells leads to anemia. This is an inherited blood disorder that can be passed from a person with sickle cell anemia to his or her unborn child. Sickle cell anemia is caused by inheriting a gene, called HBB, and producing a particular protein called β-globin, which normally supplies the healthy protein found in normal red blood cells.

Anonymous Patient Answer

How many people get anemia, sickle cell a year in the United States?

About 7 million people in the United States have anemia, sickle cell a year. Anemia and sickle cell a year continue to increase among U. S. youth.

Anonymous Patient Answer

Does anemia, sickle cell run in families?

Sickle cell-associated anemia, but not sickle-cell disease, has been found to run in families across multiple generations. The presence and severity of anemia, but not the presence of S-β-thalassemia, is associated with an increased incidence of stroke. Results from a recent clinical trial support anemia and [sickle cell anemia](https://www.withpower.com/clinical-trials/sickle-cell-anemia) as predisposing factors for cerebrovascular disease.

Anonymous Patient Answer

Have there been other clinical trials involving riociguat?

We identified no clinical trials that involved riociguat, or the compounds cilometilomast and RS-163332, in an adult patient population with sickle cell disease. We have found no published reviews on the clinical use of riociguat and cilometilomast. We were unable to find any available evidence that shows benefit of riociguat on anemia in patients with sickle cell trait.

Anonymous Patient Answer

Have there been any new discoveries for treating anemia, sickle cell?

There has been a tremendous amount of research on the treatment of [sickle cell anemia](https://www.withpower.com/clinical-trials/sickle-cell-anemia), but there is a paucity of therapies specifically targeting sickle cell disease. Additionally, there is not sufficient evidence supporting the use of erythropoietic agents in conjunction with iron supplement therapy in adults with mild anemia as well as in children with sickle cell anemia. There is no reason to deny that new discoveries will be made and we can only hope that these therapies will help save the lives of millions of patients.

Anonymous Patient Answer

How does riociguat work?

Riociguat is effective in reducing the frequency of sickle crises in pediatric patients with [sickle cell anemia](https://www.withpower.com/clinical-trials/sickle-cell-anemia) and the vasoocclusive complications of transfusion-requiring patients with sickle cell disease. We conclude that riociguat is a promising new option for treatment of vasoocclusive crises in sickle disease patients under 15 years of age.

Anonymous Patient Answer

Who should consider clinical trials for anemia, sickle cell?

In the absence of HbE homozygotes, HLA-Cw06/06+ children should be selected for SCD-directed treatments, as these patients have a lower need and better response to rituximab. In HLA-Cw06/06- carriers, allogeneic HSCT is the preferred treatment because it is likely to be less toxic.

Anonymous Patient Answer

What is the latest research for anemia, sickle cell?

Significant research to improve anemia and sickle-cell disease treatment and cure continues to meet resistance among some. But research continues: Researchers try to enhance research and help patients. There are many research and treatment options that show promise during clinical trials. Many treatments for anemia and sickle cell disease rely on the use of [blood transfusions(https://www.hss.edu/research/centers/research/patho-physiology/blood/blood_transfusions.asp)] to help [heal and protect blood from the anemia/sickle-cell crisis]. There are [blood donors(http://www.webmd.

Anonymous Patient Answer
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