Kiveton Park

Researchers are evaluating whether ambroxol hydrochloride, a drug commonly used for respiratory conditions, can slow down the progression of Parkinson's disease (PD). This Phase IIIa clinical trial is conducted in the UK and aims to assess the safety and tolerability of ambroxol in patients diagnosed with PD. The study includes patients diagnosed within the last seven years and uses a double-blind, placebo-controlled design to compare ambroxol with a matching placebo. Participants are randomly assigned to receive either 420 mg of ambroxol hydrochloride or a placebo three times a day for 104 weeks, beginning with a 2-week dose escalation period. After this blinded treatment phase, all participants enter a 26-week open-label extension where they receive ambroxol three times daily, again including a 2-week dose escalation period. Throughout the trial, clinical staff, investigators, and participants remain unaware of the assigned treatments. An optional sub-study involves lumbar punctures to analyze cerebrospinal fluid for drug levels and biomarkers related to PD. During the study, participants attend trial visits for screening, treatment, and assessments. Researchers measure changes in Parkinson's symptoms using the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) from baseline to week 104. Safety and tolerability are monitored continuously. The optional sub-study collects cerebrospinal fluid samples twice to evaluate drug penetration into the central nervous system and biochemical effects. The total participation spans over two years, including the blinded phase and open-label extension.

This research is focused on patients diagnosed with Atypical Hemolytic-Uremic Syndrome (aHUS). It aims to gather post-marketing safety data for patients treated with the drugs eculizumab or ravulizumab, as well as to monitor the progression of the disease in all participating patients regardless of treatment status. The study is observational and non-interventional, conducted across multiple centers and countries. No specific treatments or interventions are administered as part of this study; rather, it observes patients who have already been treated or untreated with eculizumab or ravulizumab. The study collects data from these patients over time to understand safety outcomes and disease progression. Participants will be followed to record the proportion experiencing certain events over a period of 10 years and the timing of first and subsequent occurrences of these events over 5 years. The study involves gathering clinical information and safety data through routine monitoring. Patients or their guardians provide informed consent and may be asked for assent if applicable. The study tracks long-term outcomes and safety in aHUS patients.

Researchers are evaluating the safety and effectiveness of whole-body hypothermia treatment in newborn babies diagnosed with mild hypoxic ischaemic encephalopathy (HIE). This phase III randomized controlled trial aims to determine whether cooling babies to 33.57b0C within six hours of birth for 72 hours improves cognitive development at two years of age compared to maintaining normal body temperature (normothermia). The study also assesses the cost-effectiveness of cooling therapy to help guide national and international treatment guidelines and standardize care across the NHS. Babies born at or after 36 weeks with specific signs of birth asphyxia or acidosis are randomly assigned to either whole-body hypothermia or targeted normothermia groups. The hypothermia group will have their body temperature lowered and maintained at 33.57b0C using a cooling machine for 72 hours in a neonatal intensive care unit. The normothermia group will have their temperature maintained at 377b0C with treatment for any fever using standard protocols. If babies in the normothermia group develop seizures and worsen to moderate HIE, they may receive cooling treatment as part of clinical care. Conventional MRI scans will be performed before discharge. Participants will be followed up at 24 months of age (7 months) using the Bayley Scales of Infant and Toddler Development IV to measure cognitive, language, and motor skills. Additional neurological exams, including assessments for cerebral palsy, vision, and hearing, will be conducted. Parents will complete questionnaires about their child's development. Researchers will collect detailed clinical data from birth through follow-up to evaluate safety and developmental outcomes. Babies who die or cannot complete assessments due to severe disability will be assigned specific scores to reflect outcomes.

Researchers are evaluating whether stopping enteral feeds around the time of blood transfusions in very premature infants born before 30 weeks of gestation can reduce the risk of Necrotizing Enterocolitis (NEC). NEC is a serious intestinal disease that affects mostly preterm infants and can cause severe complications or death. This international trial compares two common care practices in Canada and the UK to see if either approach leads to better outcomes for these vulnerable infants. The trial includes two groups: one group will have all enteral feeds stopped starting 4 hours before a packed red cell transfusion, continuing during the transfusion, and until 4 hours after it ends. The other group will continue receiving feeds during the transfusion as they were before. Infants will stay on their assigned feeding strategy until they reach 34 weeks and 6 days of gestational age. Throughout the study, researchers will monitor infants for the development of NEC up to 40 weeks postmenstrual age. They will review medical records and track feeding and transfusion details to measure outcomes. This trial aims to provide clear evidence on whether withholding feeds around transfusions can help prevent NEC and improve health in very premature infants.