Nyandiwa

Researchers are evaluating the long-term safety and effects of etavopivat, a new oral medicine being developed for inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein responsible for carrying oxygen in the blood. This phase 3 study involves participants who have already completed treatment in a prior etavopivat study and aims to understand how etavopivat performs over an extended period of up to 264 weeks, though the study may end earlier if the drug gains approval locally. Participants will receive oral doses of etavopivat, with different forms A, B, or C given based on their age and condition. Those aged 12 years and older will receive etavopivat A or C, while children under 12 years will receive etavopivat B. The study includes several groups covering various sickle cell disease and thalassaemia categories, including transfusion-dependent and non-transfusion-dependent cases. Treatment is continuous during the study period. Throughout the study, participants will undergo regular monitoring for side effects and treatment responses, including tracking treatment emergent adverse events, hospitalizations, vaso-occlusive crises, hemoglobin concentration changes, and blood transfusion needs. These assessments will help evaluate the drugs safety and efficacy across age groups and disease types. The study is open-label and non-randomized, with follow-up lasting up to about six years.

Researchers are evaluating the effects of etavopivat in adolescents and adults with sickle cell disease. This study aims to confirm whether etavopivat reduces the number of painful vaso-occlusive crises caused by blood vessel blockages. It also examines if the medicine helps reduce organ damage, improve exercise tolerance, and decrease fatigue. The study is a phase 3, randomized, double-blind, placebo-controlled trial lasting about two years. Participants will be randomly assigned to receive either oral etavopivat or a placebo. The treatment is given by mouth, and neither the participants nor the researchers know which treatment is given during the study to ensure impartial results. The primary treatment period is 52 weeks, during which the number of vaso-occlusive crises with medical contact will be measured. Secondary assessments include changes in hemoglobin levels, fatigue scores, walking distance, and other blood markers related to sickle cell disease. During the trial, participants will undergo regular evaluations including blood tests, walking tests, and fatigue assessments from baseline to week 52. Researchers will monitor the safety and effectiveness of etavopivat throughout the study. The total participation time is about two years, with ongoing follow-ups to observe the treatment effects and any side effects. All procedures aim to provide detailed information on how etavopivat affects sickle cell disease symptoms and patient well-being.

Researchers are evaluating the pharmacokinetics and safety of etavopivat in children with sickle cell disease SCD. This phase 12 open-label study involves pediatric participants divided into four age groups, starting with the oldest and moving sequentially to younger cohorts after reviewing safety and pharmacokinetic data. The study aims to understand how the drug behaves in the body and its safety profile in this pediatric population. Participants will receive oral tablets or granules of etavopivat once daily. Each age group, ranging from 6 months to under 18 years, will be treated for a 24-week primary treatment period. Following this, participants will enter a 72-week extension treatment phase to further assess long-term safety and pharmacokinetics. The total duration for each participant is approximately 96 weeks. During the study, participants will undergo various assessments including measuring drug levels in the blood and monitoring for adverse events throughout the 24-week primary treatment and 72-week extension periods. Researchers will also evaluate hemoglobin response, changes in vaso-occlusive crisis frequency, fatigue levels, and blood flow velocity by ultrasound at multiple time points. Safety monitoring and dose adjustments will be tracked, and participants will be followed closely to understand the long-term effects of etavopivat.

Researchers are investigating the effects of crizanlizumab compared to a placebo in adolescents and adults with Sickle Cell Disease who experience frequent vaso-occlusive crises VOCs. This Phase III, randomized, double-blind study involves patients aged 12 years and older who have had 4 to 12 VOCs managed by healthcare professionals in the past year. The study evaluates the safety and effectiveness of crizanlizumab with or without standard hydroxyureahydroxycarbamide therapy. Participants are randomly assigned in a 21 ratio to receive either crizanlizumab at a dose of 5 mgkg or a placebo, both given alongside standard care. The treatment is administered intravenously as a concentrate for infusion. The study is stratified by hydroxyurea use and geographical region to ensure balanced groups. The main treatment period lasts for one year. During the study, participants will attend regular visits for treatment and monitoring. Researchers will assess the number of healthcare-managed VOCs, including those treated in person or via remote consultation, and measure various other outcomes such as time to first VOC, VOC duration, antibody development to crizanlizumab, adverse events, and changes in hemoglobin levels. Safety and efficacy will be observed over two years, with detailed documentation of VOC events and other health assessments throughout participation.

Access to safe drinking water remains a major challenge in rural households of developing countries, leading to millions of deaths annually due to unsafe water sources. This research evaluates a program that provides coupons for free dilute chlorine solution to improve child health by reducing diarrhea, fever, and cough incidents. The study is a large-scale randomized controlled trial conducted at health clinics, assessing the sustained health impacts and behavior related to chlorine use and health clinic visits. Participants are randomly assigned to receive coupons redeemable for a monthly supply of a 150ml bottle of dilute chlorine solution at registered health facilities for 12 months or to a control group that receives no coupons after enrollment. The intervention is delivered via SMS with an ID for coupon redemption after enrollment over a phone call. The study is conducted in collaboration with the Kenya Medical Research Institute at Health and Demographic Surveillance Systems sites. Participants are monitored for chlorine use in drinking water at 6-month intervals up to 36 months, along with assessments of child illness occurrences like diarrhea and fever. Data collection also includes child mortality and verbal autopsies for deceased children, providing valuable information for future studies on the mortality impact of chlorine distribution. The study involves ongoing data collection on self-reported health facility visits and objective measures of chlorine use over the study period.