Morrisville

Researchers are evaluating CRD-4730 in a Phase 2 clinical trial to study its safety, tolerability, pharmacokinetics, and pharmacodynamics in people with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). This condition is confirmed by genetic testing and clinical symptoms. The trial is multicenter, double-blind, and placebo-controlled, involving repeated doses of the study drug. Participants will join a 3-period, randomized, crossover study where each person receives two different doses of CRD-4730 and one matching placebo dose in varying sequences. CRD-4730 is given orally as tablets, and placebo tablets match the drug tablets in appearance. The study carefully compares the effects of these treatments over the course of the trial. During the study, participants will undergo exercise stress tests to monitor heart activity and frequent evaluations to assess safety and drug effects. Researchers will measure outcomes from the start of treatment to Day 101. Safety monitoring includes checking heart rhythms, blood pressure, liver function, and other health indicators. Participants will be followed closely to observe how they respond to the treatments throughout the study duration.

The trial investigates the safety, tolerability, effectiveness, and how the body processes and responds to osivelotor in people with sickle cell disease (SCD). This Phase 2/3, multicenter study involves adults and adolescents with SCD, aiming to find the best dose and compare osivelotor to a placebo. The study also includes a long-term extension to assess ongoing safety and blood-related effects. Osivelotor is given orally as tablets. The study has three parts: Part A focuses on determining a safe and effective dose in adults; Part B compares osivelotor to placebo in adults and adolescents over 48 weeks; and the Open Label Extension (OLE) offers continued treatment to those who completed Part B to monitor long-term safety and responses. Participants will be monitored throughout the study periods with assessments of safety, tolerability, and treatment effects. These include evaluations at 12 weeks (Part A), 48 weeks (Part B), and approximately 24 months after the last participant joins (OLE). Researchers will track blood values, side effects, and disease-related events to understand how the drug works and its impact on SCD over time.

Researchers are evaluating the efficacy and safety of HBS-301 in treating symptoms of idiopathic hypersomnia (IH), including excessive daytime sleepiness, sleep inertia, and fatigue. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study focuses on adults aged 18 years and older diagnosed with IH. The study aims to compare HBS-301 to a placebo in relieving these symptoms and to assess its overall safety profile. Participants will be randomly assigned to receive either HBS-301 tablets or matching placebo tablets during an 8-week double-blind treatment period. Before this, there is a screening and baseline period lasting up to 28 days. Following the double-blind phase, participants have the option to join a one-year open-label extension period where they may receive HBS-301. After completing treatment, a 30-day safety follow-up will monitor participants for any adverse effects. Throughout the study, participants will undergo various assessments including sleep studies and symptom evaluations to measure the effectiveness of HBS-301. Researchers will track changes in idiopathic hypersomnia symptoms from baseline through the end of the double-blind period. Safety will also be closely monitored during treatment and follow-up periods to ensure participant well-being over the course of the study, which may last over a year for those in the extension phase.

Researchers are evaluating the effectiveness and safety of zanidatamab combined with a physician's choice of chemotherapy compared to trastuzumab combined with chemotherapy in treating adults with metastatic HER2-positive breast cancer. This study focuses on participants whose cancer has progressed or who cannot tolerate previous treatment with trastuzumab deruxtecan (T-DXd). The study is a phase 3 randomized trial aiming to assess progression-free survival and other important outcomes such as patient-reported tolerability and physical functioning. Participants receive either zanidatamab or trastuzumab through intravenous infusion, alongside chemotherapy drugs chosen by their physician from eribulin, gemcitabine, vinorelbine (all intravenous), or oral capecitabine. The study includes detailed monitoring of drug safety and how the body processes zanidatamab. The treatments continue until disease progression or unacceptable side effects occur. During the study, participants undergo regular evaluations including scans to measure cancer progression according to RECIST guidelines. Researchers also monitor safety through laboratory tests and heart function assessments. Participants are followed for up to approximately 44 months to measure progression-free survival and overall treatment outcomes. Long-term follow-up and patient-reported outcomes help provide a complete understanding of the treatments' effects.

Researchers are conducting a global Phase 3 study to evaluate the safety and effectiveness of clemizole hydrochloride (EPX-100) as an additional treatment for children and adults with Dravet syndrome, a severe form of epilepsy. The study focuses on participants with seizures that are not fully controlled by anti-epileptic drugs and includes those with a confirmed SCN1A gene mutation and other specific clinical features of Dravet syndrome. This randomized, double-blind, placebo-controlled trial aims to provide important data on this investigational therapy. Participants will receive either clemizole hydrochloride or a placebo, both given as oral solutions. The study includes a 4-week observational period without treatment changes, followed by a 16-week double-blind treatment phase where participants receive either the study drug or placebo. After this, there is an open-label extension period where all participants can receive clemizole hydrochloride. The treatment periods are designed to assess the drug's effect on seizure control over time. During the study, participants will be closely monitored for changes in the frequency of countable motor seizures compared to their baseline levels. Evaluations will include seizure tracking over 28-day periods during the titration, maintenance, and baseline phases. Safety and tolerability will also be assessed throughout the trial. Overall, the trial lasts about 20 weeks, with additional follow-up during the extension phase to gather long-term information about the treatment's effects.

Researchers are evaluating pitolisant in a global, Phase 3 study involving patients with Prader-Willi syndrome who are 6 years of age or older. The study aims to assess whether pitolisant can reduce excessive daytime sleepiness (EDS) and also evaluate its effects on irritability, disruptive behaviors, hyperphagia, and other behavioral problems such as social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech. The study includes up to a 45-day screening and baseline period, followed by a double-blind treatment period where patients are randomly assigned to receive either pitolisant or a placebo once daily. In-person visits during this period occur on Day 29, Day 57, and Day 77. After this, patients may choose to enter an optional open-label extension period with visits on Day 113, Day 260, and Day 441. Follow-up visits occur 15 and 30 days after the final dose in both the double-blind and extension periods. Participants will have assessments to measure the severity of EDS using patient-reported outcomes at the start and end of the double-blind period. Caregivers will complete behavioral evaluations, and researchers will monitor safety and treatment effects throughout. Total participation may last over a year if the optional extension is chosen, with continued follow-up after treatment ends.

Researchers are evaluating the effectiveness and safety of empasiprubart compared to intravenous immunoglobulin (IVIg) in adults diagnosed with Multifocal Motor Neuropathy (MMN). This Phase 3 study includes a double-blinded phase where participants receive either empasiprubart or IVIg, followed by an open-label phase where all receive empasiprubart. The study aims to assess treatment impact over a maximum duration of up to 49 months. Participants receive intravenous infusions of either empasiprubart or IVIg during the double-blinded phase, with matching placebo infusions given to maintain blinding. In the open-label phase, all participants are given empasiprubart infusions. The study carefully monitors treatments and their effects throughout these periods. Participants undergo assessments including measuring changes in grip strength of their most affected hand, using a 3-day moving average at week 24 as the primary outcome. Safety and efficacy are tracked throughout the study duration, with detailed follow-up visits and evaluations to monitor participant response and well-being during the study period.

Researchers are evaluating the investigational drug volixibat to treat itching (pruritus) caused by Primary Biliary Cholangitis (PBC), a liver disease. This study aims to learn more about how volixibat affects itching symptoms and whether it has any impact on the progression of PBC. The trial is a Phase 2 randomized, double-blind, placebo-controlled study designed to assess the drug's safety and effectiveness. Participants will receive either volixibat oral capsules or placebo capsules that look identical but do not contain the active drug. Volixibat is taken twice daily and works as an Ileal Bile Acid Transporter (IBAT) inhibitor. The study compares volixibat to placebo over a treatment period to determine differences in itching severity and safety outcomes. During the study, participants will complete the Adult Itch Reported Outcome (Adult ItchRO) questionnaire to measure daily itch scores from the start of the trial to week 28. Researchers will monitor symptoms and adverse events throughout the study visits. Participants must comply with all scheduled visits and assessments until the study ends to provide data on the drug's effects and safety profile.

Researchers are evaluating whether giving XEMBIFY® every two weeks along with Standard Medical Treatment (SMT) over one year can reduce the rate of serious bacterial infections in adults with low antibody levels (hypogammaglobulinemia) who have B-cell Chronic Lymphocytic Leukemia, Multiple Myeloma, or Non-Hodgkin Lymphoma. This phase 3 clinical trial compares XEMBIFY® plus SMT to a placebo plus SMT to see which better prevents infections in this group. Participants receive either XEMBIFY® or placebo through a subcutaneous infusion pump every two weeks, combined with their regular medical treatments. The study is randomized, double-blinded, and placebo-controlled, ensuring that neither participants nor researchers know who receives which treatment during the trial. Throughout the study, researchers will monitor participants for infection rates, specifically tracking major bacterial infections over about 51 weeks. Participants will have regular assessments including safety monitoring, pharmacokinetics, and infection tracking. The total study duration for each participant includes one year of treatment and observation, with careful follow-up to evaluate the treatment’s impact and safety.

Researchers are evaluating the changes in symptoms and functional limitations in adults with symptomatic hypertrophic cardiomyopathy (HCM), including both obstructive and non-obstructive types. This Phase 3 trial aims to compare the effects of sotagliflozin, an oral medication, to a placebo in these participants. The study focuses on how these treatments affect heart-related quality of life as measured by the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS) over 26 weeks. Participants will be randomly assigned to receive either sotagliflozin tablets or matching placebo tablets once daily. The study includes participants with specific heart function criteria, including left ventricular outflow tract gradients for obstructive or non-obstructive HCM and stable background therapy. The treatment period lasts 26 weeks, during which participants take the assigned tablets daily. Throughout the study, participants will undergo assessments including symptom evaluations and functional status measurements. Researchers will monitor changes from the start of the study to week 26 using the KCCQ CSS to evaluate treatment effects on heart symptoms and quality of life. Safety and adherence to medication will also be observed during this time.