Hawthorne

Researchers are evaluating the safety, tolerability, and effectiveness of two drugs, inebilizumab and blinatumomab, in adults with active and refractory autoimmune diseases such as systemic lupus erythematosus SLE with nephritis and rheumatoid arthritis RA. This phase 2, open-label, multicenter trial aims to better understand how these drugs work in these conditions and their impact on disease activity and kidney health. Participants will receive inebilizumab through intravenous IV infusions in different dosing schedules, or blinatumomab through subcutaneous SC injections at varying doses depending on the subprotocol group. The study includes several parts focusing on different autoimmune conditions and treatment regimens, with some parts no longer recruiting new participants. During the trial, participants will undergo regular assessments including monitoring for adverse events, evaluation of disease activity using specific clinical scores, kidney function tests, and antibody levels. Researchers will track safety and treatment responses over a period of up to 52 weeks, with visits occurring at scheduled intervals to collect data and monitor participant health.

Researchers are evaluating the safety and tolerability of different doses of a nebulized treatment called KB407 in adults with cystic fibrosis, a genetic condition affecting the lungs. This is a Phase 1 study sponsored by Krystal Biotech, Inc., focusing on how well adults with cystic fibrosis tolerate this new inhaled therapy designed to deliver a functional version of the CFTR protein. Participants will receive KB407 through inhalation in one of several dosing schedules a single dose, two doses, four doses, or four consecutive doses followed by weekly doses up to six months. The study includes four open-label groups, each receiving different administration frequencies to assess safety and potential effects over time. During the study, participants will undergo evaluations of adverse events, physical exams, vital signs, ECGs, and laboratory tests over six months to monitor safety and tolerability. Researchers will also measure lung function changes using forced expiratory volume in one second FEV1. Participants will be closely monitored to track any side effects and changes in lung health throughout the treatment period and follow-up.

Researchers are evaluating the clinical benefit of combining Navlimetostat BMS-986504, a selective MTA-cooperative inhibitor of PRMT5, with pembrolizumab and chemotherapy compared to placebo plus pembrolizumab and chemotherapy. This study focuses on participants with first-line metastatic non-small cell lung cancer NSCLC who have a homozygous MTAP deletion. The trial is a randomized Phase 23 study aimed at advancing treatment options for this specific lung cancer group. Participants will receive one of several combinations Navlimetostat plus pembrolizumab and chemotherapy, or placebo plus pembrolizumab and chemotherapy. Chemotherapy drugs involved may include cisplatin, carboplatin, pemetrexed, paclitaxel, or nab-paclitaxel, given at specified doses on certain days. The study uses a quadruple-masked, parallel design with multiple treatment arms to compare these regimens. During the study, participants will be monitored for progression-free survival and overall survival up to five years. Researchers will assess tumor response, disease control, duration and time to response, and safety through adverse event reporting and laboratory tests. The study includes detailed follow-up to evaluate efficacy and safety outcomes over the long term, with a primary completion date in 2031.

Researchers are comparing the effectiveness of two different combinations of immunotherapy drugs with chemotherapy for adults with stage IV or recurrent non-squamous non-small cell lung cancer that has PD-L1 expression of 1% or higher. This phase 3, randomized study focuses on participants who have not previously received systemic treatment for advanced disease. The goal is to determine which combination better improves overall survival and other outcomes in this patient group. Participants will be randomly assigned to receive either Nivolumab plus Relatlimab combined with chemotherapy or Pembrolizumab combined with chemotherapy. The chemotherapy drugs used include Carboplatin, Pemetrexed, or Cisplatin, given in specified doses on scheduled days. Treatment is given as first-line therapy for their cancer, with dosing details managed throughout the study period. During the study, participants will undergo imaging scans like CT or MRI to measure disease status, and blood tests to monitor safety and side effects. The main outcome measured is overall survival over up to five years, along with progression-free survival, response rates, duration of response, and adverse events. Researchers will also assess symptoms related to lung cancer over two years. Participants are monitored regularly to track these outcomes and ensure safety throughout the study duration, which may last several years.

Researchers are evaluating BHV-7000 as a treatment for adults with refractory focal onset epilepsy, a form of epilepsy that does not respond to standard anti-seizure medications. The study aims to determine if BHV-7000 can reduce seizure frequency and assess its safety and tolerability. This Phase 23 clinical trial is sponsored by Biohaven Therapeutics Ltd. and involves participants aged 18 to 75 years with a diagnosis of focal epilepsy lasting at least one year and resistant to previous treatments. The trial consists of two parts. In Part A, participants are randomly assigned to receive either 25 mg or 50 mg of BHV-7000 once daily or a matching placebo. After completing Part A, participants may enter Part B, which involves randomization to either 75 mg of BHV-7000 once daily or placebo. Both parts are blinded, meaning neither participants nor researchers know who receives the active drug or placebo during the treatment periods. Participants will keep accurate seizure diaries throughout the study to track seizure frequency. Researchers will monitor safety by recording adverse events and laboratory abnormalities from Week 8 to Week 20 in both parts. The main outcome measured in Part B is the change in average seizure frequency over 28 days compared to baseline. Secondary outcomes include the percentage of participants with significant seizure reduction and seizure freedom during the study. The total participation duration includes treatment and follow-up assessments over several weeks.

Researchers are evaluating cannabidiol oral solution CBD-OS in participants aged 12 to 75 years who have focal-onset seizures FOS. This study aims to assess the effectiveness and safety of CBD-OS as an additional treatment to reduce seizure frequency compared to baseline. It also explores health outcomes in early line and refractory participants, along with pharmacokinetics, safety, and potential predictors of treatment response using functional magnetic resonance imaging fMRI and neuropsychological testing in a substudy. Participants will receive open-label CBD-OS starting with doses based on the approved local product label. The study is a single-group, open-label trial where all participants are treated with CBD-OS. It involves continuous administration of the drug with monitoring of effects and safety across the study period. During the study, participants will undergo evaluations including seizure frequency monitoring, health outcome assessments, pharmacokinetic sampling, fMRI scans, and neuropsychological testing for those in the substudy. Researchers will measure the percent change in countable focal seizure frequency compared to baseline, tracked for up to 16 weeks. Safety and treatment response predictors will also be monitored throughout the trial, which lasts until November 2027.

Researchers are evaluating the safety and effectiveness of tezepelumab in children aged 5 to under 12 years with severe uncontrolled asthma. These children are already on medium to high doses of inhaled corticosteroids and at least one other asthma controller medication. The study is a phase 3, randomized, double-blind, placebo-controlled trial aimed at assessing tezepelumabs impact on asthma control and safety in this pediatric population. Participants will be randomly assigned in a 21 ratio to receive either subcutaneous injections of tezepelumab or a matching placebo over a 52-week double-blind treatment period. Before this, there is a 4 to 6 week screening and run-in period. After the treatment phase, there is a 12-week off-treatment follow-up for those not continuing. Additionally, an optional 104-week open-label extension allows eligible participants to receive tezepelumab, followed by another 12-week post-treatment follow-up. Throughout the study, participants will undergo regular assessments including lung function tests, asthma control questionnaires, symptom diaries, and blood tests to measure inflammation and immune response. Researchers will monitor asthma exacerbations, medication use, quality of life, and any side effects. Safety will be tracked during treatment and follow-up periods, with total study involvement potentially lasting over three years for those in the extension phase.

Researchers are evaluating the safety and tolerability of a new multivalent pneumococcal vaccine called PG4 compared to the currently used 20-valent pneumococcal conjugate vaccine 20vPnC in healthy infants about 2 months old. The study aims to see if the new vaccine is as safe as the existing one and whether it can provide additional protection against infections caused by the Streptococcus pneumoniae germ, including pneumonia, meningitis, and ear infections. This is a phase 3, randomized, double-blinded trial conducted by Pfizer focused on prevention of pneumococcal disease. About 2400 infants will be randomly assigned to receive either the PG4 vaccine or the 20vPnC vaccine. Vaccinations are given by injection into the left thigh muscle at 2, 4, 6, and between 12 to 15 months of age. The study consists of two groups receiving these vaccines according to this schedule. The treatment period spans these vaccination times, with a total participation duration of about 16 to 19 months. During the study, infants will have six clinic visits and one phone call for monitoring. Parents or legal guardians will report any side effects or adverse events their infant experiences after vaccinations. Researchers will evaluate local and systemic reactions within 7 days of each dose, adverse events from the first dose through one month after the third and fourth doses, and serious adverse events up to six months after the last dose. Safety and tolerability data will be collected throughout the study period.

This research aims to evaluate the long-term safety and tolerability of brivaracetam in children and adolescents with epilepsy, including those who participated in earlier studies or are newly enrolled in Japan with partial-onset seizures. The study also seeks to assess pharmacokinetic data in Japanese participants. The study is an open-label, single-arm, multicenter trial focusing on pediatric epilepsy treatment with brivaracetam. Participants will receive brivaracetam tablets or oral solution administered twice daily in two equal doses. Dosage varies based on weight up to 5 mgkgday for those weighing 11 to less than 20 kg, up to 4 mgkgday for those weighing 20 to less than 50 kg, and no more than 200 mgday. Directly enrolled participants in Japan will receive 1 to 4 mgkgday, not exceeding 200 mgday. The study includes long-term follow-up for up to 5 years. During the study, participants will be monitored for treatment-emergent adverse events, serious adverse events, and adverse events leading to discontinuation of the drug. Evaluations occur from Day 1 through safety visits over the 5-year period. Researchers will assess safety, tolerability, and pharmacokinetics. The total participation duration extends up to 5 years, with ongoing safety evaluations throughout this time.

Researchers are conducting the American Lung Association ALA Lung Health Cohort, a multi-center, long-term observational study focusing on young adults aged 25 to 35 without severe lung disease. The study aims to understand lung health by examining various factors such as environmental exposures, physical activity, biomarkers, and respiratory cell activity to identify early markers and targets to prevent chronic lung diseases. Participants will undergo an initial baseline assessment to measure lung function, including pre-bronchodilator forced expiratory volume in one second FEV1 and forced vital capacity FVC. After this, regular remote contacts through text, email, mail, or phone will be used to follow up with participants for up to 4.5 years. Pending additional funding, the study may be extended beyond 5 years to explore long-term changes in lung health. During the study, participants will provide contact information and undergo lung function tests. Follow-up data will be collected remotely to monitor lung health over time. Researchers will track lung function measures and gather information on environmental and lifestyle factors. Safety and participant status will be monitored regularly, with the total study duration potentially extending beyond 5 years depending on funding.