Treatment for Anemia, Sickle Cell

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Children's National Hospital, Washington, United States
Anemia, Sickle Cell+1 More
Eligibility
< 65
All Sexes
What conditions do you have?
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Study Summary

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

See full description

Eligible Conditions

  • Anemia, Sickle Cell
  • Acute Vaso Occlusive Crisis (VOC)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Anemia, Sickle Cell

Study Objectives

This trial is evaluating whether Treatment will improve 6 primary outcomes and 4 secondary outcomes in patients with Anemia, Sickle Cell. Measurement will happen over the course of Collected from enrollment to end of study (2-Days).

Hour 7
To determine the how the study medication is processed by the body.
Collected from enrollment to end of study (2-Days)
Preliminary assessment of pain change measured
Day 30
Preliminary assessment of change of opioid use overall
Time to clinical resolution of VOC
Collected from enrollment to end of study (30-Days)
Duration or length of hospital stay
Month 18
Assessment of safety
Change from baseline in amount of overall opioid use
Change in Visual Analogue Scale (VAS) pain score
Discharge from ED/hospital within 7 hours
Dose Optimization (Part 1)

Trial Safety

Safety Progress

1 of 3

Other trials for Anemia, Sickle Cell

Trial Design

6 Treatment Groups

Arm 4 (L-citrulline)
1 of 6
Arm 1 (L-citrulline)
1 of 6
Arm 3 (L-citrulline)
1 of 6
Part 2 Arm 1 (L-citrulline)
1 of 6
Arm 2 (L-citrulline)
1 of 6
Part 2 Arm 2 (normal saline (0.9% NaCl) D5 ½)
1 of 6
Active Control
Non-Treatment Group

This trial requires 120 total participants across 6 different treatment groups

This trial involves 6 different treatments. Treatment 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 1 & 2 and have already been tested with other people.

Part 2 Arm 2 (normal saline (0.9% NaCl) D5 ½)
Other
Subjects will be randomized to 2 of the doses selected from Part 1 and placebo in a 1:1:1 ratio.
Arm 4 (L-citrulline)
Drug
100 mg/kg bolus + 11 mg/kg/hr continuous infusion of L-citrulline for up to 7 hours
Arm 1 (L-citrulline)
Drug
25 mg/kg bolus + 9 mg/kg/hr continuous infusion of L-citrulline for up to 7 hours
Arm 3 (L-citrulline)
Drug
100 mg/kg bolus + 9 mg/kg/hr continuous infusion of L-citrulline for up to 7 hours
Part 2 Arm 1 (L-citrulline)
Drug
Subjects will be randomized to 2 of the doses selected from Part 1 and placebo in a 1:1:1 ratio. L-citrulline will be administered to the active arm.
Arm 2 (L-citrulline)
Drug
50 mg/kg bolus + 9 mg/kg/hr continuous infusion of L-citrulline for up to 7 hours

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: collected from enrollment to end of study over 18 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly collected from enrollment to end of study over 18 months for reporting.

Closest Location

Children's National Hospital - Washington, United States

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 5 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
At study entry, the patient was not experiencing any acute complication other than a VOC due to sickle cell disease. show original
, are required For a woman of childbearing potential, a negative urine pregnancy test and using an adequate method of contraception, or denies sexual activity, are required. show original
People who are willing and able to sign and provide consent and assent (where appropriate for the age of the child). show original
Sickle cell anemia is a red blood cell disorder show original
are typically diagnosed with ADHD Children, adolescents and young adults are typically diagnosed with ADHD if they exhibit symptoms of inattention, hyperactivity or impulsiveness show original

Patient Q&A Section

What are common treatments for anemia, sickle cell?

"Although the common names do give this condition a reputation of being difficult, sickle cell anemia can resolve by itself after several months of supportive care." - Anonymous Online Contributor

Unverified Answer

What is anemia, sickle cell?

"Sickle cell disease is the most prominent disorder in African-Americans. People with sickle cell anaemia have a tendency to suffer from a common vascular disease called thrombosis. The pathophysiological mechanisms underlying this tendency are not clear. Other types of anaemias present only in certain groups of human populations, such as the people of sub-Saharan Africa, the Indian Ocean Islands, Southeast Asia and sub-saharan Africa. Sickle cell anaemia affects roughly 230 million people in the world and 40 million people in Africa. Sickle cell anaemia is more commonly seen in Subsidiary-Saharan Africa and Indian Ocean Islands." - Anonymous Online Contributor

Unverified Answer

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

"The prevalence of Hb in the United States is about 12%, with the majority of cases being mild. The overall prevalence of Hb in persons of African Caribbean and non-Hispanic black birth, is significantly higher (P < 0.00) than other ethnic groups." - Anonymous Online Contributor

Unverified Answer

What are the signs of anemia, sickle cell?

"Anemia may present with pallor, pale stool, dark urine, tachycardia and low blood pressure. It may also affect the red cell indices, hematocrit, hemoglobin and other red cell indices, thus making diagnosis difficult." - Anonymous Online Contributor

Unverified Answer

What causes anemia, sickle cell?

"Risk factors for alpha haemoglobin diseases, particularly malaria and sickle cell disease, overlap with risk factors for malaria, sickle cell pathology, and anaemia. The exact ways in which the two coexist is unclear, but there is a very strong chance that both are caused by the same set of risk factors.\n" - Anonymous Online Contributor

Unverified Answer

Can anemia, sickle cell be cured?

"The most effective and cost efficient cure for [sickle cell anemia (SCA)] is the blood transfusion of Hgb/Hct >8 g/dL. In clinical practice, SCA patients may be cured by blood transfusions if Hgb/Hct >8 g/dL and if their mean Hgb is between 6 and 9 g/dL. Hgb/Hct > 9 g/dL is achievable if they get transfusions every 4 to 8 weeks." - Anonymous Online Contributor

Unverified Answer

Is treatment safe for people?

"In people with sickle cell anemia, aspirin is well tolerated and does not contribute to significant reductions in either hemoglobin levels or morbidity. In people with sickle cell anemia and mild to moderate anemia a daily aspirin dose of >90 mg can be considered. Clinicians must consider the risk and benefit profile for all patients and tailor the aspirin dosing." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of anemia, sickle cell?

"Sickle cell anemia is caused by mutations in hemoglobin S and hemoglobin F. These mutations produce abnormal hemoglobosins that tend to aggregate and bind to deform red blood cells, which leads to micro-hemolysis, and eventually micro- and macro-hemolysis, and anemia. You can find recent clinical trials by searching for sickle cell anemia using [Power(https://www.withpower.com/clinical-trials/anemia-folic/)." - Anonymous Online Contributor

Unverified Answer

How serious can anemia, sickle cell be?

"[Anemia can be one of the first symptoms of sickle cell disease or stroke] and [sickle cell disease-related anemia and stroke are especially deadly diseases]. [You are unlikely to find out a diagnosis of sickle cell disease in your routine medical clinic, but if you notice anemia symptoms, such as the ones mentioned above, or have headaches with no obvious cause, or do not get on well with your doctor, give your doctor a copy of your medical records and ask for an Hgb test. And tell your doctor that you have sickle cell anemia. The CDC has several online screening tools that check hemoglobin levels against age and the symptoms in your diagnosis." - Anonymous Online Contributor

Unverified Answer

What does treatment usually treat?

"Hemoglobin levels usually remain in this range for children with sickle cell disease, so children are less likely to need frequent testing. This is a review of the current understanding of the treatment options available for anemia in children with sickle cell disease." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets anemia, sickle cell?

"Sickle cell anemia can occur at any age but tends to start early in infancy. This is especially true among African-Americans. Individuals with sickle cell anemia may be at higher risk for complications during fetal development." - Anonymous Online Contributor

Unverified Answer

Is treatment typically used in combination with any other treatments?

"There is an indication that most patients with sickle cell anemia are being treated in this region. It is important for patients to be properly informed about the therapy they are receiving so that they can understand the rationale for prescribing treatment. The role of medical practitioners must be seen as a key provider of information and advice to the patient about the treatment they are receiving." - Anonymous Online Contributor

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