This trial is evaluating whether Mitapivat will improve 4 primary outcomes and 36 secondary outcomes in patients with Anemia, Sickle Cell. Measurement will happen over the course of Up to Week 12.
This trial requires 267 total participants across 7 different treatment groups
This trial involves 7 different treatments. Mitapivat is the primary treatment being studied. Participants will be divided into 5 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 & 3 and have had some early promising results.
"A wide variety of medications are used to treat children and adults with SCD. The use of hydroxycarbamide, splenectomy, bone marrow transplantation, and regular medical exam and evaluation are common. Physicians must be aware of this variety of treatments for SCD." - Anonymous Online Contributor
"It is a genetic trait with different probabilities of developing sickle cell syndromes that is often unexposed to environmental hazards or drugs of abuse. It is caused by a mutation (a change of one or more nucleotides in the DNA of a cell) in one of the genes of sickle hematopoietic cells leading to their production." - Anonymous Online Contributor
"About 40,000 children under 15 years of age have SCD at some time, of which 24,000 will have an affected parent. The number of children in the United States with SCD will diminish over the next decade, probably due to increased surveillance of SCD and improved treatment of newborns by universal screening." - Anonymous Online Contributor
"In infants and children, SCD is marked by anemia, thrombocytopenia and vascular and organ damage due to sickling. In adults, SCD is a serious disease caused mainly by the progressive destruction and disability of the vasculature, heart, brain, liver, kidneys and joints. The average life expectancy in the developed world for people with a single SCD allele is now more than half that of the general population." - Anonymous Online Contributor
"In this population, symptoms of SCD are likely to have been present during childhood, and they include a history of painful crises, splenomegaly, and hepatomegaly; or a previous history of one, two, or three episodes during childhood. It is possible to diagnose SCD based on examination of a peripheral blood smear." - Anonymous Online Contributor
"SCD is a chronic autoimmune disorder and therefore it is impossible to alter the number of blood cells in the peripheral blood stream or to totally remove all cells affected by autoimmunity." - Anonymous Online Contributor
"SCD is inherited in families. The likelihood of developing SCD in a sibling of a parent with SCD is significantly higher than previously accepted. Results from a recent paper of this study suggest that there are at least two genetic factors that play a role in SCD. Such factors should be identified as molecular markers of SCD to enable the prediction of a genetic predisposition for future development of SCD." - Anonymous Online Contributor
"Mitapivat was well tolerated, and the side effects profile generally mirrored that of a non-cancer drug of this class. However, the occurrence of mild and transient reductions in red blood cell counts has been observed in some studies and warrants further investigation in clinical trial cohorts." - Anonymous Online Contributor
"Mitapivat showed high blood pressure and high levels of creatinine and a low haematocrit, which may interfere with the clinical effects of mitapivat. The safety of mitapivat has not been established for people with SCD, and mitapivat should be used with care in this group. More evidence of safety in people with SCD is also warranted to establish mitapivat as a reasonable first-line treatment in this group." - Anonymous Online Contributor
"Mitapivat significantly reduced the perceived burden and impairment in quality of life for both children with SCD and their caregivers. Parents of patients with SCD are also highly supportive of this medication." - Anonymous Online Contributor
"There is a substantial mortality during the course of the disease after the acute event is gone and the disease is stable even in the absence of treatments. This highlights the potential of this disease to become a burden for developing countries, especially in people of African derivation." - Anonymous Online Contributor
"Recent findings of the present trial show that MPA was effective in decreasing the frequency of adverse clinical events, a decrease in sickle cell crisis, and a higher percentage of patients who achieved a 50% reduction in the frequency of painful crises. Recent findings show that MPA can be effective in controlling thalassemia pain." - Anonymous Online Contributor