Sanford

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a genetic condition causing fluid-filled cysts in the kidneys, leading to kidney disease. This Phase 2 study is investigating the safety and effectiveness of ABBV-CLS-628, an investigational drug, in adults with ADPKD. The study aims to evaluate how well ABBV-CLS-628 works and monitor any side effects in this population. Participants will be assigned to one of four groups, with each group receiving a different treatment. There is a 1 in 4 chance of receiving a placebo. Treatments, either ABBV-CLS-628 or placebo, will be given by intravenous infusion every 4 weeks for 92 weeks. After this treatment period, participants will be followed for up to an additional 15 weeks. During the study, participants will attend regular visits at hospitals or clinics where their health will be monitored through medical exams, blood tests, and questionnaires. Researchers will measure changes in total kidney volume over 96 weeks and track any adverse events up to approximately 118 weeks. The study involves around 240 adults aged 18 to 55 years with specific stages of ADPKD.

Researchers are evaluating the safety, effectiveness, and tolerability of upadacitinib in adolescents and adults with severe alopecia areata (AA), a condition where the immune system attacks hair follicles causing hair loss on the head, face, or other body parts. This phase 3 study involves about 1500 participants worldwide and compares upadacitinib to a placebo to assess treatment impact on severe AA. Participants are randomly assigned to one of three groups receiving either upadacitinib or placebo oral tablets once daily for up to 160 weeks. There is a chance for re-randomization at weeks 24 and 52 based on Severity of Alopecia Tool (SALT) scores. Those completing initial studies may join an extension study to receive upadacitinib for up to an additional 108 weeks. Follow-up occurs for 30 days after the last dose. Throughout the study, participants attend regular visits at hospitals or clinics for medical assessments, blood tests, side effect monitoring, and questionnaires. Researchers measure the percentage of participants achieving a SALT score of 20 or less at week 24 and track adverse events up to 164 weeks. The study may involve a higher treatment burden compared to usual care due to frequent visits and evaluations.

Researchers are conducting a two-part, phase 2b/3 study to evaluate CSL300 (Clazakizumab) in adults with end stage kidney disease (ESKD) undergoing dialysis who have systemic inflammation and either atherosclerotic cardiovascular disease (ASCVD) or diabetes. The study aims to determine the best dose of CSL300 and assess its effects on cardiovascular outcomes and safety in this population. This multicenter, randomized, double-blind, placebo-controlled trial targets patients with elevated inflammation markers and significant health risks due to their conditions. In the first part (phase 2b), the study focuses on finding the appropriate dose of CSL300 compared to placebo. CSL300 is given through intravenous (IV) administration. The second part (phase 3) evaluates the impact of CSL300 on cardiovascular events such as heart attack or cardiovascular death over approximately 5 years, continuing to compare CSL300 to placebo for safety and efficacy. The placebo matches CSL300's excipient content but lacks the active drug. Participants will undergo baseline and regular assessments for inflammation markers like high-sensitivity C-reactive protein (hs-CRP) up to 12 weeks in phase 2b, and long-term monitoring for cardiovascular outcomes in phase 3. The study involves ongoing safety evaluations and efficacy measurements during the entire follow-up period. This comprehensive approach helps researchers understand how CSL300 affects inflammation and cardiovascular health in patients with ESKD on dialysis.

Researchers are evaluating VX-147 for its effectiveness, safety, tolerability, and how the body processes it in adults and children aged 10 to 65 who have apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease. This study is a Phase 2/3 trial designed to better understand treatment options for this specific kidney condition. Participants will receive either VX-147 or a placebo, both given as oral tablets. The study is double-blind and placebo-controlled, meaning neither participants nor researchers know who receives the active drug or placebo during the treatment period. The trial consists of two parts: Part A focuses on treatment effects over at least 48 weeks, while Part B involves continued safety and tolerability observation for approximately four years after the last participant enrolls. Throughout the study, participants will undergo regular assessments including measurements of urine protein to creatinine ratio and kidney function via estimated glomerular filtration rate (eGFR). Safety is monitored by tracking adverse events and serious adverse events. Data will be collected during the treatment period and followed long-term to evaluate both efficacy and safety outcomes, with some measures assessed at interim and final analyses over at least two years.

Researchers are evaluating the safety and effectiveness of up to two injections of REACT/rilparencel in adults with type 2 diabetes mellitus and chronic kidney disease. This phase 3 randomized controlled study divides participants into two groups to compare the effects of the actual treatment versus sham procedures mimicking kidney biopsy and injections. The goal is to monitor kidney function and clinical outcomes over time to understand the impact of this therapy on disease progression. Participants are randomly assigned before a kidney biopsy to either receive sham procedures or the real treatment involving a kidney biopsy followed by two rilparencel injections about 12 weeks apart, each into different kidneys. Those receiving sham procedures will undergo similar-sounding and looking activities without actual tissue removal or injection. All participants will be followed until the study's global end date, ensuring consistent long-term observation. During the study, participants will undergo kidney biopsies or sham procedures, followed by injections or sham injections. Researchers will assess kidney function by measuring the slope of estimated glomerular filtration rate (eGFR) over 18 months after the 135th participant's first injection or sham procedure. They will also track clinical events such as significant kidney function decline, need for dialysis or transplant, or renal and cardiovascular deaths for up to 94 months. Safety and efficacy will be monitored throughout the study to evaluate treatment impact.

Researchers are evaluating the safety and effectiveness of deuruxolitinib in adolescents aged 12 to less than 18 years who have severe alopecia areata with 50% or more scalp hair loss. This Phase 3 study includes participants with a current alopecia areata episode lasting between 6 months and 10 years. The goal is to assess how well deuruxolitinib works compared to a placebo in improving hair regrowth and to monitor its safety in this age group. Participants will be randomly assigned to receive either oral deuruxolitinib tablets at a dose of 8 mg or matching placebo tablets during a 24-week double-blind treatment period. After this period, all participants can join an open-label extension lasting 52 weeks, during which they will receive deuruxolitinib. This design allows researchers to evaluate the drug's effects over both the initial treatment and a longer-term period. Throughout the study, participants will undergo evaluations including measuring scalp hair loss using the Severity of Alopecia Tool (SALT) score, monitoring for adverse events, checking vital signs, conducting electrocardiograms, lab tests, and physical exams. The main outcome is the percentage of subjects achieving a SALT score of 20 or less at week 24. Safety and tolerability will also be assessed throughout the 24 weeks. Participants are expected to comply with study visits and procedures during the entire study duration.

Researchers are evaluating the effectiveness and safety of ruxolitinib cream in people with hidradenitis suppurativa (HS), a chronic skin condition. This Phase 3 study compares ruxolitinib cream to a vehicle cream, aiming to see if the treatment helps clear HS symptoms better than a non-active cream. The study focuses on participants with mild to moderate HS who have had the condition for at least six months. Participants will apply either ruxolitinib cream or a matching vehicle cream as a thin layer twice daily to the affected areas. The study is double-blind and randomized, meaning neither the participants nor the researchers know who receives the active or vehicle cream during the treatment period. Participants must avoid using antibiotics or topical antiseptics on HS areas during the study and extension periods. Throughout the study, participants will be monitored to evaluate the proportion achieving a clinical response, specifically a 75% improvement in HS symptoms by week 16. Safety and treatment effects will be assessed through regular visits, and participants will be observed for any side effects or changes in their condition. The study's total duration includes the treatment and extension phases, with careful adherence to treatment application and restrictions to ensure accurate results.

Researchers are evaluating the use of ruxolitinib cream in adults and adolescents with hidradenitis suppurativa, a chronic skin condition. This Phase 1 trial aims to assess the maximal use of this topical treatment to better understand its safety and effects in this population. Participants must have had hidradenitis suppurativa for at least six months and show specific disease characteristics to be eligible. Participants will apply ruxolitinib cream as a thin layer twice daily on affected areas of the skin. The treatment focuses on individuals with a significant body surface area affected by the condition. During the study, participants agree to avoid other topical or systemic antibiotics for hidradenitis suppurativa, and pregnancy prevention measures must be followed as outlined in the study protocol. Throughout the study, researchers will monitor participants for treatment-emergent adverse events up to 24 weeks, including any events that lead to treatment interruption or discontinuation. The study involves careful assessments to ensure safety and to evaluate the treatment's impact on the condition. Participants' health and laboratory values will be regularly checked, and the total study duration includes this monitoring period.