This study will evaluate the safety and effectiveness of fazirsiran (investigational drug) compared with placebo (an inactive substance) in patients with alpha-1 antitrypsin deficiency associated liver disease (AATD-LD). If eligible, subjects will be randomized (assigned to a group by chance) to receive either fazirsiran or placebo to be administered subcutaneously (an injection under the skin). Subjects will be treated on Day 1, at Week 4, and then every 12 weeks for 196 weeks. Subjects will be followed for 6 months after their last dose of study drug or placebo for a total study duration of approximately 230 weeks (including 10 weeks of the screening period which is the time needed to assess if a subject is eligible for the study).
The study will be an open-label, randomized, parallel arm study that will include a
treatment arm and control arm. Participants will have clinic visits at screening, randomization (day 1) and weeks 4, 12, 18, and 24. After week 24, participants will have clinic visits at weeks 32, 40, and 48. Participants will also have a telehealth visit on day 2 and phone calls to assess adverse events (AEs), serious adverse events (SAEs), and review patient education will occur during weeks 5, 8, 36, and 44. The phone calls may be converted to telehealth visits or clinic visits and / or followed by clinic visits, if the study team deems it necessary. Pulmonary function testing, quality of life survey (St. George's Respiratory Questionnaire (SGRQ)), and blood draw will occur at each clinic visit.
1) You will be asked to provide a blood sample; up to two tubes for a total of up to 10mL (less than 2 teaspoons).
2) Samples will be processed and tested on the MeMed BV device.
3) You will be asked about your medical history, medications, and current illness, as well as demographic information (i.e. date of birth) and a contact phone number.
4) If eligible, it will be decided by chance, using a computer, if you will be put into one of two arms: the MeMed BV arm or the control arm. You have an equal chance of being placed in each group. You cannot choose your study group.
a. The MeMed BV arm: your clinician will receive the BV result, this will include a recommendation regarding antibiotic treatment
b. The control arm: your clinician will not receive the MeMed BV results and will treat you according to standard of care.
5) You will be contacted by a member of the study team at 28 (+/- 3) days after the day of consent to complete a short questionnaire regarding your current illness
1) You will be asked to provide a blood sample; up to two tubes for a total of up to 10mL (less than 2 teaspoons).
2) Samples will be processed and tested on the MeMed BV device.
3) You will be asked about your medical history, medications, and current illness, as well as demographic information (i.e. date of birth) and a contact phone number.
4) If eligible, it will be decided by chance, using a computer, if you will be put into one of two arms: the MeMed BV arm or the control arm. You have an equal chance of being placed in each group. You cannot choose your study group.
a. The MeMed BV arm: your clinician will receive the BV result, this will include a recommendation regarding antibiotic treatment
b. The control arm: your clinician will not receive the MeMed BV results and will treat you according to standard of care.
5) You will be contacted by a member of the study team at 28 (+/- 3) days after the day of consent to complete a short questionnaire regarding your current illness
Intimate partner violence (IPV) is a serious public health problem that results in significant health and economic burdens including mortality, morbidity, and poor treatment outcomes. A well-developed field of research suggests that alcohol misuse and posttraumatic stress disorder (PTSD) can lead to IPV. Individuals with PTSD and/or problematic drinking behaviors are at risk for IPV because of several factors that are common symptoms of PTSD. Because individuals with PTSD often drink alcohol to "self-medicate" or cope with distressing PTSD symptoms, PTSD co-occurs with alcohol misuse and alcohol use disorder at extraordinarily high rates. However, few studies have examined the combined effects of alcohol misuse and PTSD on any form of violence.
This study will examine the effects of alcohol misuse and posttraumatic stress disorder (PTSD) on alcohol-related intimate partner violence (IPV). We will examine these associations among couples (N=70) in a controlled laboratory setting using validated, standardized methods in a 'real-world' settings using 28 days of ecological momentary assessment (EMA). EMA includes very short surveys (5 minutes or less) that participants respond to on a smartphone application. Participants will be asked to respond to these surveys in the morning and three additional times throughout the day.
This study is enrolling emerging adults (ages 18-25) with cannabis use disorder (CUD) to examine sex differences in (a) cannabis withdrawal symptoms during short-term cannabis abstinence, (b) cannabidiol (CBD) versus placebo effects on stress reactivity during short-term cannabis abstinence, and (c) the relationship between stress reactivity and time to cannabis relapse after short-term cannabis abstinence. The proposed study is designed to reveal sex differences and guide the development of tailored treatments that address factors disproportionately affecting emerging adult females with CUD.
Participants will complete an assessment visit to determine eligibility. Eligible participants will be scheduled for their next visit and will be instructed to abstain from cannabis use for 3 days. Participants will be set up with a phone application (app) and given instructions on its use. This app will send twice daily, random surveys everyday throughout study participation with questions about cannabis use, cravings, and overall mood. Participants will also complete twice daily saliva samples.
At the end of the 3 days, participants will return to the clinic for their second visit. Participants will complete a urine and blood sample at each visit. After eating a snack, participants will receive one dose of CBD (800mg) or placebo and then participate in a stress task. Upon completion of the stress task, participants will complete 3 saliva samples and then be discharged after evaluation by research staff. After the completion of Visit 2, participants will continue to complete twice daily surveys for 10 days. The study will last approximately 14 days.
There are risks involved with participating in this study, including risks associated with CBD, risks associated with the stress task and study procedures, emotional distress from answering personal questions, and loss of confidentiality. There is a risk of experiencing cannabis withdrawal symptoms during the 3-day period of cannabis abstinence. Some potential risks related to CBD include dry mouth, diarrhea, reduced appetite, drowsiness, and fatigue. There is a risk of loss of confidentiality, but the researchers will code the samples and research information to protect privacy. There are no direct benefits to the participant, but we hope the knowledge gained will help us inform future clinical strategies to address cannabis use in emerging adults.
The purpose of this study is to learn more about how cannabis use affects memory, thinking, and stress response in older adults, and how ovarian hormones affect them in older women. Participants will undergo a screening process over telehealth to confirm eligibility. There is a second telehealth visit to complete questionnaires, a cognitive testing battery, and a medical history. There is one visit in the clinic where participants will provide urine and saliva samples for drug and alcohol testing and a blood sample for a lipid testing, and complete additional cognitive tasks. They will then complete CREMA sessions (Cue Reactivity Ecologic Momentary Assessment) at home, two times a day for ten days. CREMA sessions include answering questions about marijuana use and sleep, and rating stress and craving.
The purpose of this study is to learn more about how cannabis use affects memory, thinking, and stress response in older adults, and how ovarian hormones affect them in older women. Participants will undergo a screening process over telehealth to confirm eligibility. There is a second telehealth visit to complete questionnaires, a cognitive testing battery, and a medical history. There is one visit in the clinic where participants will provide urine and saliva samples for drug and alcohol testing and a blood sample for a lipid testing, and complete additional cognitive tasks. They will then complete CREMA sessions (Cue Reactivity Ecologic Momentary Assessment) at home, two times a day for ten days. CREMA sessions include answering questions about marijuana use and sleep, and rating stress and craving.
The purpose of this study is to learn more about how cannabis use affects memory, thinking, and stress response in older adults, and how ovarian hormones affect them in older women. Participants will undergo a screening process over telehealth to confirm eligibility. There is a second telehealth visit to complete questionnaires, a cognitive testing battery, and a medical history. There is one visit in the clinic where participants will provide urine and saliva samples for drug and alcohol testing and a blood sample for a lipid testing, and complete additional cognitive tasks. They will then complete CREMA sessions (Cue Reactivity Ecologic Momentary Assessment) at home, two times a day for ten days. CREMA sessions include answering questions about marijuana use and sleep, and rating stress and craving.
This study is testing citicoline as a possible medication to treat alcohol use disorder. Youth (ages 16-22) will be randomly assigned to receive either citicoline (2000mg per day) and or a placebo for four weeks.
All participants will receive brief counseling from a trained clinician and will undergo a brain scan and cognitive testing at the beginning and end of the treatment.
Participants must provide informed consent and youth under 18 must have parental consent to participate. The full study will last approximately one month.
Compensation is available to those who qualify.