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NCT ID: NCT05144256 Active, not recruiting - Clinical trials for Pediatric Pyruvate Kinase Deficiency

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

ACTIVATE-KidsT
Start date: June 8, 2022
Phase: Phase 3
Study type: Interventional

ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to < 6 years, 6 to < 12 years, 12 to < 18 years) and splenectomy status. Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 24-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.

NCT ID: NCT05143957 Recruiting - Clinical trials for Phlebotomy Dependent Polycythemia Vera

A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

Start date: December 30, 2021
Phase: Phase 2
Study type: Interventional

The main purpose of this study is to evaluate the efficacy of sapablursen in reducing the frequency of phlebotomy and in improving quality of life assessments in participants with polycythemia vera.

NCT ID: NCT05143463 Completed - Clinical trials for Neurodegenerative Diseases

A FIH Study to Assess the Safety and Tolerability of NS Intravenous NS101 Infusion

Start date: November 4, 2021
Phase: Phase 1
Study type: Interventional

Up to 80 healthy adult males, ≥ 18 and ≤ 55 years of age, are planned to be enrolled in the study. The study will consist of 8 cohorts (Cohorts 1 to 8, 1 cohort per dose level). Each cohort will include 8 subjects (6 subjects receiving a single dose of the study drug NS101 and 2 subjects receiving a single dose of a matching placebo), for a total of 64 subjects planned for evaluation. A total of 21 blood samples will be collected in each cohort for PK analysis and a total of 14 blood samples will be collected in each cohort for PD analysis.

NCT ID: NCT05143437 Completed - Clinical trials for Internet-Based Intervention

Love Together, Parent Together: A Feasibility Study

L2P2
Start date: August 26, 2021
Phase: N/A
Study type: Interventional

The COVID-19 pandemic has introduced additional stressors and challenges to couples' relationships, with potential ripple effects across all family subsystems and child adjustment. Among those who are particularly vulnerable to heightened conflict and lower relationship satisfaction during this time are couples with young children, whose relationships may have already been tenuous prior to the pandemic. The Love Together Parent Together (L2P2) program is a brief, low-intensity writing intervention adapted for parents of young children, designed to reduce conflict-related distress and prevent relationship deterioration. Based on an original writing program by Finkel and colleagues, adaptations include intervention duration and study population. The current study will examine key feasibility metrics related to this adapted intervention program with the goal of identifying problems and informing parameters of future pilot and/or main randomized controlled trials (RCT). The current study is a non-randomised feasibility study, using a single-arm, pre-test/post-test design to primarily assess the feasibility of a large trial, and secondarily to assess the potential effects on outcomes to be used in a future RCT. Couples will be recruited through three community-based agencies with the goal of obtaining a socio-demographically diverse sample. The first 20 couples to enroll will be included. Baseline and post-intervention surveys will be conducted, and a writing intervention will take place (three 7-minute sessions over the course of four weeks). The primary feasibility metrics will include recruitment rates, appropriateness of eligibility criteria, sample diversity, retention, uptake, and adherence, and acceptability. In addition, the researchers will develop an objective primary outcome measure of couple "we-ness" based on analysis of writing samples. The secondary outcomes will include couples' measures (i.e., relationship quality, perceived partner responsiveness, self-reported partner responsiveness, conflict-related distress), and other family outcomes (i.e., parent-child relations, parental/child mental health, and parenting practices). Criteria for success are outlined and failure to meet criteria will result in adaptations to measurement schedule, intervention design, recruitment approaches, and/or other elements of the study design. This feasibility study will inform several components of the procedures used for a subsequent pilot RCT, which will examine the feasibility of the methodology used to evaluate the program (e.g., randomization, attrition to follow up assessment/across groups, and sample size estimation, preliminary effectiveness), and a main trial, which will investigate the effectiveness of the intervention on primary outcome measures as well as mediating pathways.

NCT ID: NCT05142774 Completed - Atopic Dermatitis Clinical Trials

Long Term Extension Study of Tapinarof Cream, 1% for Subjects With Atopic Dermatitis

Start date: October 28, 2021
Phase: Phase 3
Study type: Interventional

This is an open-label, long-term multicenter, study to evaluate the safety and efficacy of topical tapinarof cream, 1% in subjects with atopic dermatitis. Subjects in this study have completed treatment in one of two Phase 3 pivotal studies (DMVT-505-3101 or DMVT-505-3102) or completed treatment in the DMVT-505-2104 study, or directly enrolled into this study. This study will consist of up to 48 weeks of treatment and a 1 week safety follow-up period.

NCT ID: NCT05142735 Recruiting - Clinical trials for Prodromal Schizophrenia

Effects of NAC on Symptoms of CHR Patients

Start date: January 13, 2023
Phase: N/A
Study type: Interventional

Schizophrenia is a chronic debilitating psychotic disorder. Identifying persons with "clinical high-risk" (CHR) symptoms, which are like those of schizophrenia but less severe, and providing psychiatric care to these individuals has been shown to help prevent psychosis. Current medications used for CHR symptoms, however, are associated with substantial side effect burden. Therefore, practice guidelines do not recommend current medications as routine treatment for the CHR state, and there is a need to identify new treatments for this condition. Research suggests that abnormal brain oxidative stress may contribute to schizophrenia, offering a potential novel treatment target in the CHR state. Oxidative stress is an excess of free radicals, which are generated from normal metabolism and environmental exposures, and can damage cells. Antioxidants in the body normally neutralize free radicals. Antioxidant deficiency could result in excess oxidative stress that damages brain cells, leading to schizophrenia. Recent studies suggest that N-acetylcysteine (NAC), a precursor of the most abundant brain antioxidant, glutathione, may be a safe, well-tolerated treatment for schizophrenia. In light of this, NAC may also reduce symptoms and brain abnormalities in CHR patients.

NCT ID: NCT05142683 Recruiting - Clinical trials for Major Depressive Disorder

Effectiveness of an Integrated Care Pathway for Depression: Cluster Randomized Controlled Trial

CARIBOU-2
Start date: February 1, 2022
Phase: N/A
Study type: Interventional

This a stepped wedged cluster RCT with two intervention arms--Treatment As Usual (TAU) and an Integrated Care Pathway (ICP). Eligible participants are between the ages of 13 and 18, who present to community mental health agencies with depressive symptoms as the primary concern. The primary objective is to establish the clinical effectiveness of the ICP intervention in the community setting relative to TAU, with respect to reducing evaluator-rated depressive symptoms. The secondary objectives are to explore changes in clinician-rated function and caregiver-rated symptoms for youth receiving the ICP intervention relative to TAU. The third objective is to explore the implementation effectiveness of the ICP intervention, namely investigating: feasibility, fidelity, cost and acceptability. Edited on March 7th, 2024: This is a quasi-experimental, multi-site cluster controlled clinical trial design with two intervention arms--Treatment As Usual (TAU) and an Integrated Care Pathway (ICP). Eligible participants are between the ages of 13 and 18, who present to community mental health agencies with depressive symptoms as the primary concern. The primary objective is to establish the clinical effectiveness of the ICP intervention in the community setting relative to TAU, with respect to reducing evaluator-rated depressive symptoms. The secondary objectives are to explore changes in clinician-rated function and caregiver-rated symptoms for youth receiving the ICP intervention relative to TAU. The third objective is to explore the implementation effectiveness of the ICP intervention in the community setting, namely investigating: feasibility, fidelity, cost and acceptability.

NCT ID: NCT05142566 Terminated - Clinical trials for Femoral Arteriotomy Closure

MANTA Ultrasound Closure Study

MANTA ULTRA
Start date: August 9, 2022
Phase: N/A
Study type: Interventional

Demonstrate the safety of MANTA Vascular Closure Device (VCD) ultrasound (U/S) guided closure in patients undergoing elective TAVR procedures with planned percutaneous femoral arterial access.

NCT ID: NCT05141942 Recruiting - Bile Leak Clinical Trials

The Need for Repeat ERCP After Endoscopic Treatment of Postsurgical Biliary Leaks

Start date: November 19, 2019
Phase:
Study type: Observational [Patient Registry]

Post-surgical biliary leaks are relatively common after surgeries associated with hepatobiliary health. Left untreated, biliary leaks can lead to significant morbidity. Biliary leaks are most often successfully managed endoscopically, by way of performing an ERCP (endoscopic retrograde cholangio-pancreatography) procedure. These procedures help manage the bile leak by decreasing pressure at the opening of the common bile duct and promoting bile flow into the small bowel (rather than out the leak) via stent insertion. Guidelines published by the American Society for Gastrointestinal Endoscopy (ASGE) report that stents are generally placed for 4 to 6 weeks and recommend longer intervals for more complex leaks. However, formal recommendations concerning the modality of biliary stent removal do not exist. One option is performing a repeat ERCP when removing the stent. While comprehensive, this exposes the patient to additional radiation, and requires additional fluoroscopy resources and/or technicians. Furthermore, ERCPs are less available, especially in smaller centers, and are costly. A second option is a gastroscopy with simple stent removal has, given the low probability of requiring repeat intervention, the relatively low procedural cost, and the relatively favorable adverse event profile, and easier accessibility compared to ERCP procedures. A simple, safe and reliable prediction rule was developed retrospectively to identify patients in whom biliary stent removal via gastroscopy could be safely performed, as opposed to repeat ERCP. A positive result using the rule requires satisfaction of four non-invasive clinical markers: (1) a normal post-surgical serum alkaline phosphatase value, (2) bile leak 'type C' at initial ERCP (a small or absent leak with no other biliary pathology), (3) a bile leak caused by laparoscopic cholecystectomy, and (4) a time between initial and follow-up endoscopy of 4 to 8 weeks. Validating this prediction rule prospectively could have implications on patient safety by decreasing ERCP-related adverse events, and could also have important implications with regard to health resource utilization.

NCT ID: NCT05141916 Recruiting - Choledocholithiasis Clinical Trials

Optimizing the Evaluation and Management of Patients With Suspected Choledocholithiasis

Start date: February 25, 2019
Phase:
Study type: Observational [Patient Registry]

Choledocholithiasis (stone(s) in the common bile duct) is common. Untreated or missed, choledocholithiasis has high morbidity and mortality. Endoscopic retrograde cholangio-pancreatography (ERCP) is recognized as the first-line modality for management. While effective, ERCP is associated with adverse events. Thus, the selection of patients for ERCP should be accompanied by a high pre-test suspicion of choledocholithiasis. Choledocholithiasis is suspected based on clinical, biochemical and radiographic findings. The most relied-upon strategy for risk stratification of choledocholithiasis is based on guidelines from The American Society for Gastrointestinal Endoscopy (ASGE). In it, clinical predictors are defined as "very strong", "strong" or "moderate", and the presence of one or more of these is meant to suggest "high" or "intermediate" probability of choledocholithiasis. A knowledge gap exists in the performance characteristics of intermediate-probability criteria, where overall accuracy is <50% from limited data. Patients in this group are recommended to a) undergo endoscopic ultrasound (EUS) or magnetic resonance cholangio-pancreatography (MRCP), b) undergo cholecystectomy with intra-operative cholangiography (IOC), or c) proceed directly to ERCP. At centres where EUS and MRCP are readily available, these are preferred options, as they are least invasive and sensitive; however, they are often unavailable. Thus, in clinical practice, a high proportion of intermediate-risk patients ultimately proceed directly to ERCP, where likelihood of benefit is only moderate, while procedural risk remains. The role of liver enzyme changes has not been evaluated; however, dynamic changes may offer another method for evaluating patients at intermediate risk of CBD stones that is safe and available. Incorporation of dynamic liver enzymes may improve the test-performance characteristics of the existing framework.