Johnson City

Researchers are evaluating the safety and effectiveness of three different doses of MORF-057 in adults with moderately to severely active Crohn's disease (CD). This Phase 2 study is randomized, double-blind, placebo-controlled, and conducted at multiple centers. It aims to compare MORF-057 to placebo to see how well it works in reducing disease activity and symptoms in this patient population. Participants will first go through a 14-week induction period where they receive one of three doses of MORF-057 or a matching placebo, all given orally. After this, all participants will enter a 38-week maintenance phase where they receive open-label MORF-057. Those who complete these 52 weeks of treatment may continue in a 52-week long-term extension to further monitor treatment effects and safety. Throughout the study, participants will have evaluations to assess their response to treatment using endoscopic scoring at Week 14. Researchers will monitor safety, symptom changes, and disease activity over the full treatment and extension periods. Study visits will include assessments, questionnaires, and clinical monitoring to track participants' health and treatment adherence over time.

Researchers are evaluating the safety and effectiveness of fixed dose combinations of ensifentrine with two different doses of glycopyrrolate compared to placebo and each drug alone in adults with chronic obstructive pulmonary disease (COPD). This phase IIb study focuses on measuring lung function improvements using bronchodilator effects in people with COPD. Participants have a history of smoking and meet specific lung function criteria to be included. Participants will be randomly assigned to one of six groups: two combination treatments of ensifentrine (3 mg) with glycopyrrolate at either 21.25 or 42.5 mcg, each drug alone as monotherapy, or placebo. All treatments are given twice daily for 28 days using a standard jet nebulizer. The study includes 1 to 2 weeks of screening, 4 weeks of treatment, followed by 1 week of follow-up. During the study, participants will undergo lung function testing at baseline and on days 1, 14, 28, and 29 to measure changes in forced expiratory volume in one second (FEV1). They will also have chest X-rays or CT scans reviewed, complete questionnaires on breathlessness, and have regular assessments to monitor safety and treatment effects. Participants must be able to use the nebulizer properly and attend all study visits over approximately 7 weeks.

Researchers are evaluating the effectiveness and safety of a gene therapy called AAV2-hAQP1 in adults who have Grade 2 or Grade 3 late xerostomia caused by radiation treatment for cancers of the upper aerodigestive tract, excluding the parotid glands. This Phase 2 study focuses on people who completed radiation therapy at least three years ago and still experience dry mouth symptoms. The goal is to see if this therapy can improve saliva production and relieve symptoms associated with radiation-induced xerostomia. The study involves administering different concentrations of AAV2-hAQP1 directly into each parotid gland through Stensen's duct. There are four different dosing groups receiving varying concentrations of the gene therapy and a placebo group receiving a diluent. The treatment is given bilaterally to both parotid glands, allowing comparison of safety and efficacy across doses and with placebo. Participants will be closely monitored over a 12-month period. Researchers will measure changes from the start of the study to month 12 in a xerostomia-specific questionnaire score to assess symptom improvement. Other assessments include saliva flow rates and imaging to rule out cancer recurrence. Medication use, adverse events, and overall safety will be tracked throughout the study. The total participation time includes screening, treatment administration, and follow-up visits to evaluate both short- and long-term effects of the gene therapy.

This trial investigates the safety and effectiveness of risankizumab compared to vedolizumab in adults with moderate to severe ulcerative colitis (UC) who have not previously received targeted therapies. Ulcerative colitis is an inflammatory bowel disease causing inflammation and bleeding in the rectum and colon. The study is a Phase 3b, randomized, open-label trial enrolling about 530 participants across 285 sites worldwide. Participants will be randomly assigned to receive either risankizumab or vedolizumab. Those in the risankizumab group will receive the drug intravenously during the initial induction phase, followed by subcutaneous injections for maintenance. Participants in the vedolizumab group will receive the drug intravenously throughout the study. The treatment period lasts 44 weeks for risankizumab and 46 weeks for vedolizumab, following a screening period of up to 35 days. During the study, participants will attend regular outpatient visits for medical assessments, side effect evaluations, and to complete questionnaires. Researchers will monitor disease activity and drug safety, focusing on the percentage of participants achieving endoscopic improvement by week 48. The total study duration is approximately 69 weeks for risankizumab and 71 weeks for vedolizumab recipients.

Researchers are evaluating the effects and safety of AZD6793 tablets in adults aged 40 years and older who have moderate to very severe chronic obstructive pulmonary disease (COPD). This is a Phase IIb, multicenter, randomized, double-blind, placebo-controlled study involving approximately 1160 participants at around 400 sites worldwide. The study aims to compare three different doses of AZD6793 against placebo tablets over 24 weeks to assess how well the treatment works and its safety profile in this population. Participants will be randomly assigned to one of four groups receiving either one of three doses of AZD6793 or a placebo in equal proportions. The treatment involves oral administration of AZD6793 tablets or placebo tablets daily for 24 weeks. The study is designed with parallel groups and includes careful dose-ranging to evaluate different levels of the investigational drug. During the study, participants will be monitored for the annualized rate of moderate or severe COPD exacerbations from baseline up to 24 weeks. Assessments include lung function tests such as pre- and post-bronchodilator FEV1/FVC ratios, symptom questionnaires like the COPD Assessment Test (CAT), and documentation of COPD exacerbation history. Safety will be continually evaluated through clinical assessments and laboratory tests throughout the treatment period.

Researchers are evaluating the safety and effectiveness of tezepelumab in adults aged 40 to 80 years with moderate to very severe chronic obstructive pulmonary disease (COPD). These participants must have a history of COPD for at least one year and have experienced multiple COPD exacerbations despite using inhaled maintenance therapy. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study focuses on those who have had at least two moderate or one severe exacerbation in the prior year while on inhaled triple or dual therapy. Participants will receive monthly subcutaneous injections of either one of two doses of tezepelumab or a placebo. Treatment will last for a minimum of 52 weeks and up to 76 weeks. After the treatment period, there will be a 12-week off-treatment safety follow-up to monitor any lasting effects or safety concerns. During the study, researchers will assess the participants' lung function and monitor the annual rate of moderate or severe COPD exacerbations. Participants will undergo screening to confirm eligibility based on lung function tests, eosinophil counts, and symptom scores. Safety will be closely monitored throughout the treatment and follow-up periods to evaluate adverse effects and overall participant health.

PRIMARY OBJECTIVE: I. To compare the proportion of participants who develop clinically meaningful chemotherapy induced peripheral neuropathy (CIPN) at 12 weeks, in participants treated with taxane-based chemotherapy randomized to cryocompression therapy versus continuous compression therapy versus low cyclic compression therapy. SECONDARY OBJECTIVES: I. To compare trajectories over time (6, 12, 24, and 52 weeks) by intervention study arm in clinically meaningful CIPN. II. To compare mean European Organization for Research and Treatment of Cancer Chemotherapy-Induced Peripheral Neuropathy 20 (EORTC-CIPN-20) sensory neuropathy subscale scores at 12 weeks by intervention study arm. III. To compare differences by intervention study arm at 12 weeks in changes from baseline in objective sensory and motor function tests (Neuropen, tuning fork, Timed Get Up and Go test). IV. To compare the proportion of participants who develop clinically meaningful CIPN at 12 weeks in a sensitivity analysis with dropouts treated as failures. V. To compare rates of adverse events related to study device at 12 weeks (including cold intolerance, skin changes, other adverse events \[AEs\] as assessed by Common Terminology Criteria for Adverse Events \[CTCAE\]) between the three interventions. ADDITIONAL OBJECTIVES: I. To compare the proportion of participants who develop clinically meaningful CIPN separately at weeks 6, 24, and 52. II. To compare the proportion of participants who develop clinically meaningful CIPN at week 12 with additional covariate adjustment for age and body mass index (BMI). III. To compare differences by intervention study arm at 12 weeks in mean EORTC CIPN-20 motor subscale score and autonomic subscale score, and in mean individual Patient-Reported Outcomes Measurement Information System (PROMIS)-29 domain (Physical Functioning, Anxiety, Depression, Fatigue, Sleep Disturbance, Social Functioning, Pain Interference, and Pain Intensity) scores. IV. To compare trajectories over time (6, 12, 24, and 52 weeks) by intervention study arm in mean EORTC CIPN-20 sensory neuropathy subscale score, motor subscale score, and autonomic subscale score; and in mean PROMIS-29 individual domains (Physical Functioning, Anxiety, Depression, Fatigue, Sleep Disturbance, Social Functioning, Pain Interference, and Pain Intensity) scores; and in changes in objective sensory and motor function tests (Neuropen, tuning fork, Timed Get Up and Go test). V. To evaluate the differences by intervention study arm in proportion of participants who develop clinically meaningful CIPN at 12 weeks by chemotherapy regimen. VI. To assess the effect of the intervention in reducing CIPN occurring in the upper extremities and, separately, in the lower extremities. VII. To explore the relationship between duration of intervention received at the prescribed level and outcome, analogous to a dose-delivered analysis in a treatment trial. VIII. To compare rates by study arm of CTCAE Grade 2 or higher sensory and motor neuropathy at 12 weeks. IX. To evaluate tolerability of cryocompression compared to continuous compression therapy and low cyclic compression therapy, as assessed by rate of temperature and/or pressure adjustments, interruptions, and early discontinuation of the device. X. To determine participant satisfaction of cryocompression compared to continuous compression therapy and low cyclic compression therapy, assessed by patient questionnaire. XI. To compare taxane dose-reductions, treatment delays/discontinuation due to CIPN, and relative taxane dose intensity and total taxane dose received, between intervention study arms. XII. To evaluate differences of intervention effect by sex, race, and ethnicity. XIII. To confirm pretreatment biomarkers of CIPN risk (vitamin D) and on-treatment biomarker changes indicative of CIPN severity (Neurofilament light chain, NFL) as well as additional biomarkers of interest generated in S1714 for validation. BANKING OBJECTIVE: I. To bank specimens for future correlative studies. OUTLINE: Patients are randomized to 1 of 3 arms. ARM 1: Patients undergo cryocompression (cooling plus moderate and low pressure to the arms and legs) for 30-minutes pre-taxane chemotherapy infusion, during taxane chemotherapy infusion, and for 30 minutes after completion of each taxane infusion. Patients may also undergo collection of blood, serum and plasma samples during screening and on study. ARM 2: Patients undergo continuous compression (moderate, steady pressure to the arms and legs) for 30-minutes pre-taxane chemotherapy infusion, during taxane chemotherapy infusion, and for 30 minutes after completion of each taxane infusion. Patients may also undergo collection of blood, serum and plasma samples during screening and on study. ARM 3: Patients undergo low cyclic compression (low pressure that comes and goes to the arms and legs) for 30-minutes pre-taxane chemotherapy infusion, during taxane chemotherapy infusion, and for 30 minutes after completion of each taxane infusion. Patients may also undergo collection of blood, serum and plasma samples during screening and on study.

Researchers are evaluating the effects of an experimental treatment called corneal crosslinking (CXL) for conditions where the cornea becomes thin, steep, and misshapen, leading to blurred vision. This Phase 3 trial focuses on participants aged 8 years and older with diagnoses such as keratoconus, ectasia after LASIK or PRK, pellucid marginal degeneration, progressive ectasia after previous CXL, or forme fruste keratoconus. The study aims to determine whether CXL can prevent or slow the progression of these corneal conditions and associated vision loss. The treatment involves applying riboflavin (vitamin B2 eye drops) to the eye followed by exposure to ultraviolet (UV-A) light. Participants are divided into two groups: one receives UV-A treatment for 18 minutes, and the other for 24 minutes. This procedure is designed to strengthen the cornea and potentially halt disease progression. Participants will attend up to 7 office visits over 6 months, undergoing various eye and vision tests. The main outcomes measured are changes in corneal curvature using Kmax via Pentacam imaging and changes in corrected distance visual acuity (CDVA) from enrollment through the 6-month treatment period. The study monitors participants closely to assess treatment effects and safety throughout this timeframe.

Researchers are evaluating an experimental treatment called corneal crosslinking (CXL) for people with conditions where the cornea becomes thin, steep, and misshapen, leading to blurred vision. This study focuses on participants aged 8 years or older with Down syndrome and related corneal diseases such as keratoconus, pellucid marginal degeneration, and forme fruste keratoconus. The main goal is to determine if CXL can prevent or slow the worsening of corneal shape and vision loss. The treatment involves applying riboflavin (Vitamin B2 eye drops) to the eye, followed by exposure to ultraviolet (UV-A) light for 20 minutes, aiming to strengthen the cornea. Participants will receive this treatment during the study, which is a phase 3 compassionate use trial. The study includes up to 7 office visits over 6 months for treatment and follow-up evaluations. During these visits, participants will undergo several eye and vision tests, including measuring corneal curvature with the Pentacam device and assessing vision improvement through corrected distance visual acuity. The study monitors changes from enrollment through the 6-month treatment period to evaluate the treatment's effects. Participants must comply with visit schedules and follow instructions to support the study's goals.

Researchers are evaluating the effects of an 8-week independent walking program on people with Parkinson disease, comparing walking with and without walking poles. The study aims to understand changes in walking ability, movement, and thinking through various tests and questionnaires. Participants must be English-speaking, diagnosed with Parkinson disease, able to walk safely, and medically cleared for walking exercise. Participants will first undergo an initial assessment including demographic data, cognitive and movement tests, and training on how to use walking poles if assigned to that group. The walking pole group will practice proper pole technique and demonstrate proficiency before beginning the program. During the 8-week program, all participants will walk at least three times per week, record their walking activity and exertion in logs, and receive regular check-ins from study staff. The walking pole group will use poles during all intentional walking exercises, while the control group will walk without poles. Before, immediately after, and three months following the walking program, participants will complete walking tests using motion sensors and heart rate monitors, cognitive assessments, and self-reported questionnaires on balance confidence, Parkinson's symptoms, disability, and freezing of gait. Researchers will track changes in walking performance and collect information about falls, exercise habits, and experiences with walking poles. The total participation includes the initial assessments, the 8-week walking intervention, and a 3-month follow-up period.