CLINICAL TRIAL

Treatment for Anemia, Sickle Cell

Waitlist Available · Any Age · All Sexes · Pittsburgh, PA

This study is evaluating whether a supplement of L-citrulline can improve blood flow in people with 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 a single treatment. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

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

Eligibility

This trial is for patients born any sex of any age. There are 4 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
are required for the first 28 days of therapy and then continued for 4 weeks after the last dose Females of child-bearing potential must have a negative serum pregnancy test and use an adequate method of contraception for the first 28 days of therapy and then continue for 4 weeks after the last dose. show original
The subject has read and understands the information in the informed consent form, agrees to participate in the study, and will follow the study protocol. show original
This study will include males and females 10 years of age or older who are generally healthy, as determined by a medical history, physical examination, and laboratory test values. show original
Diagnosis of sickle cell anemia (Hb SS)
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Odds of Eligibility
High>50%
You meet most of the criteria! It's probably a good idea to apply to 1 other trial just in case this doesn't work out.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: Up to 5 years
Screening: ~3 weeks
Treatment: Varies
Reporting: Up to 5 years
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to 5 years.
View detailed reporting requirements
Trial Expert
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- 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 Treatment will improve 2 primary outcomes and 1 secondary outcome in patients with Anemia, Sickle Cell. Measurement will happen over the course of .

PK and PD responses to L-citrulline
Assessment of safety and tolerability of L-citrulline
Endpoints will be determined as change from baseline

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 are the common side effects of treatment?

Side effects of treatment for sickle cell anemia are common and range from minor to severe and must always be taken into account when making treatment decisions. Patients may also experience the following side effects to various degrees: fatigue, shortness of breath, chest pain, leg pain that can be alleviated by rest, cough, dizziness, headaches, and other dizziness-related symptoms.

Anonymous Patient Answer

What are common treatments for anemia, sickle cell?

Anemia, sickle cell and thalassemia require treatment. Liver or pancreas transplantation and chelation are two surgical options. Hemolytic crises may be effectively avoided with prophylactic calcium and vitamin supplements.

Anonymous Patient Answer

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

These numbers should be seen as the total number of people affected, not the whole population, because only adults are currently being tested for [sickle cell anemia](https://www.withpower.com/clinical-trials/sickle-cell-anemia). The number of people with sickle cell disease is more likely to be underestimated.

Anonymous Patient Answer

What is anemia, sickle cell?

Sickle cell anemia is a blood disorder in which hemoglobin S, a less sturdy, sicklier variant, precipitates into red blood cells, damaging and damaging them. Typically, people of African heritage, especially in sub-Saharan Africa, are affected. Sickle cell does not go away by itself; it continues to grow until, for many, the effects of sickling become painful. But there is a common, effective treatment. It has eliminated thousands of deaths in Africa from blood clots at the valves of the heart and other serious complications.

Anonymous Patient Answer

What are the signs of anemia, sickle cell?

Signs of anemia, sickle cell include shortness of breath, tingling or pain in one area of the body (hands) and numbness or tingling around the arms or legs. Symptoms such as short breath, chest pain and ankle swelling is due to a complication from sickle cell disease, not anemia itself. These signs usually improve after delivery.\n

Anonymous Patient Answer

What causes anemia, sickle cell?

[Sickle cell disease (SCD) is a genetic, hereditary condition that causes red blood cells to be abnormally shaped. Anemia is a condition in which the patient's red blood cells are no longer able to carry oxygen efficiently.

Anonymous Patient Answer

Can anemia, sickle cell be cured?

Based on current knowledge, it cannot be recommended to try to cure a person's sickle cell illness: many of the existing approaches are only supportive treatments. However, it is likely that with the development of better therapies and treatment approaches the ability to cure sickle cell will be increased over the coming years.

Anonymous Patient Answer

Has treatment proven to be more effective than a placebo?

Clinical trial subjects in the first group had lower sickle cell hemoglobin levels and better socioeconomic and health conditions than control subjects, so it would be inappropriate to conclude that the drug trial produced an effect that would not otherwise have occurred even in individuals with sickle cell disease otherwise. A well-controlled trial of aspirin for the prevention of PDA is warranted.

Anonymous Patient Answer

What does treatment usually treat?

The effectiveness of the treatment is in many ways dependent on the underlying cause of the disease and also can vary within the same disease depending on the individual. These effects mean that treatment effectiveness can be affected by the underlying diagnosis of the individual, which is why any treatment must be specifically tailored towards the disease of the individual.

Anonymous Patient Answer

How serious can anemia, sickle cell be?

Most adults with a mean Hgb level under 12.0 g/dL will obtain a satisfactory Hgb response to sickle cell treatment. Some adults with Hgb levels>16.0 g/dL will obtain a partial Hgb improvement following sickle cell treatment. To ensure a full return to normal Hgb levels, sickle cell patients with Hgb levels>18.0 g/dL will be required to be treated indefinitely. Sickle cell patients who achieve a Hgb level of>16.0 g/dL may require a prolonged duration of chronic sickle cell treatment to prevent severe morbidity with the potential to develop severe sequelae, particularly in patients with anemia.

Anonymous Patient Answer

What is the latest research for anemia, sickle cell?

Sickle cell disease is a hereditary genetic disorder. There were advancements in the medical field that contributed to a better understanding of the disease. Sickling has not only been discovered to be a cause of the disease but has also been shown to be more pronounced after surgery. It has also been discovered that there is a relation between sickling and the blood vessels in the kidneys which can also be a source of complications. In addition, erythrocyte deformability (which determines transfusion of a patient's blood) could be the target of future treatments.

Anonymous Patient Answer

Does treatment improve quality of life for those with anemia, sickle cell?

For a small cohort studied, it appears that anemia and sickle cell affect self-reported quality of life in a manner that reflects not only functional impairment, but psychometric distress. Although intervention has an immediate clinical impact in this population, it is difficult to say if this was due to the improvement in hematological parameters or the reduction in psychometric distress.

Anonymous Patient Answer
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