- Featured
Evaluating the Psychometric Properties of Three Clinical Trial Outcome Measures for Rett Syndrome
\- Clinically or genetically-diagnosed Rett syndrome, MECP2 duplication syndrome, CDKL5 syndrome, FOXG1 syndrome, or a pathogenic mutation of MECP2, CDKL5, or FOXG1 with intellectual/developmental delay without a diagnosis of Rett syndrome. \- Non-verbal or minimally verbal. \- All ages.
Phase
N/ASpan
Sponsor
St Paul, Minnesota
Recruiting
- Featured
Cerebral Palsy Hip Outcomes Project - International Multi-centre Study
Background: Children with severe cerebral palsy (CP) are at high risk for dislocating their hips. These hips are associated with contractures and pain, which can interfere with care-giving, seating, positioning, mobility and quality of life. The primary purpose of this project (Aim 2) is to evaluate the effectiveness of different intervention strategies to prevent or relieve the symptoms associated with hip instability in children with severe non-ambulatory CP, using the validated Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD©) questionnaire as the primary outcome measure of health related quality of life (HRQL) for this population. Secondarily, this project will also measure the impact of hip displacement on HRQL of these children. This project will be the first of its kind and this scale, which will systematically study the impact of hip instability and its management in children with severe CP, using a meaningful outcome measure that was developed specifically for this purpose. The international network of investigators/sites and the infrastructure established for this project will facilitate the long term follow-up of the participants in this study, as well as the conduct of other multi-centre clinical trials and cohort studies to evaluate the effectiveness of current and future interventions aimed at improving the quality of life of children with severe disabilities. Study Design & Participants: International multi-centre prospective longitudinal cohort study of children with severe (non-ambulant) cerebral palsy (GMFCS levels IV & V) from ages 3 to 18 who have radiographic evidence of hip displacement [Reimer's Migration Percentage (MP) ≥ 30%]. Measures: Detailed demographic information, and prognostic factors, including co-morbid conditions will be recorded at baseline, in addition to self-administered parental reports of HRQL as measured by the CPCHILD. Hip status will be classified using standardized radiographic measures of Reimer's MP and acetabular index (AI). The primary outcome measure CPCHILD, as well as the MP & AI will be measured at 6, 12 and 24 months following initial intervention. Aim 1: Measure the impact of increasing hip displacement in children with severe (non-ambulant) CP on their HRQL as measured by the CPCHILD questionnaire. Aim 2: (Primary Purpose): Measure the effectiveness of different strategies of interventions for hip displacement in children with severe (non-ambulant) CP in a prospective longitudinal comparative cohort study using the CPCHILD as the primary outcome measure of HRQL. Aim 3: Compare the types and rates of adverse events and complications associated with each of the treatment cohorts. Methods: Observational study of usual (site/surgeon specific) clinical practice. Investigators at each site will enroll eligible participants and assign each to one of the following 5 cohorts based on individual treating doctor's &/or parental preferences: A. "Natural" history or watchful waiting (N=100) B. Serial botulinum toxin injections +/- abduction bracing (N=100) C. Adductor (+/- psoas) muscle releases alone (N=100) D. Hip reconstructive surgery (N=100) E. Salvage hip surgery (N=100) The baseline MP and CPCHILD scores for all participants will be analyzed cross-sectionally to evaluate the correlation between hip displacement and the CPCHILD scores to serve Aim 1. For Aim 2, children undergoing interventions for hip instability (Groups B, C, D, & E) will be compared with each other as well as with their respective matched counterparts of untreated children (Group A), using repeated measures of analysis of covariance (ANOCOVA) to measure the mean change in scores from baseline at 6, 12 and 24 months after intervention. Timelines: 500 participants will be recruited in 24 months, and followed for 24 months. The analysis, reporting of results, manuscript development and knowledge transfer will take 12 months. In total, the study will take 5 years to complete.
Phase
N/ASpan
413 weeksSponsor
Holland Bloorview Kids Rehabilitation HospitalSt. Paul, Minnesota
Recruiting
Product Performance Report: Evaluate Long-term Reliability & Performance of Medtronic Marketed Cardiac Therapy Products
All Medtronic market-released leads and all market-released IPG, ICD and CRT devices are eligible to be included in this study.
Phase
N/ASpan
3027 weeksSponsor
MedtronicSt. Paul, Minnesota
Recruiting
Novel Post-Surgical Incision Management to Prevent Ostomy Complications
The Limpet is a single-use device intended for effluent management and promotion of healing of intact and injured skin surrounding enterocutaneous fistulae and ostomies. Our study focuses on small bowel openings, encompassing two important cohorts: 1) ileostomy and 2) enterocutaneous fistulas (ECFs). Our study's success criterion is primarily based on the pooled results from both cohorts. Primary Hypothesis 1: Peristomal Skin Complications (PSCs): We hypothesize that the Limpet will demonstrate a significant improvement in PSCs compared to standard of care adhesive ostomy pouches. Specifically, we anticipate a raw difference of 35% in PSCs for the pooled cohorts, with a complication rate of 50% in the control group and 15% in the Limpet group. Primary Hypothesis 2: Dressing Leak Rates: We hypothesize that the Limpet will demonstrate a significant improvement in dressing leak rates compared to standard of care adhesive ostomy pouches. Specifically, we anticipate a raw difference of 15% in leak rates for the pooled cohorts, with a leak rate of 30% in the control group and 15% in the Limpet group. These hypotheses are grounded in existing literature, supplemented by clinical experience. We intend to conduct subgroup analyses to assess the treatment effect across the two cohorts (ileostomy and ECF) and across other relevant subgroups such as gender, race, etc. Secondary Hypothesis: Total Complications (PSCs and Stomal Complications): We hypothesize that the Limpet will exhibit a raw difference of 35% in total complications (comprising PSCs and stomal complications like mucocutaneous separation and stoma retraction) for the ileostomy cohort. Stoma complication rates vary widely in the literature, and we plan to conduct sub analyses to evaluate the Limpet's effect on stoma complications independent of PSCs. This will be a 30 day, single-site, randomized controlled trial (RCT) for a non-significant risk device, Limpet. The study will have a two-arm parallel group design: - Limpet: Participants in this arm will utilize the subject device as intended to be used. - Control: Standard treatment with standard of care adhesive ostomy pouches. Ninety-two participants (78 stoma and 14 enterocutaneous fistula participants) will be randomized after they have consented to study participation. Initial randomization will occur in a 1:1 ratio (Limpet and Control). Participants will be randomly stratified with permuted blocks by BMI (≤ vs > 40) and stoma type (loop ileostomy, end ileostomy, or enterocutaneous fistula) to account differences in healing and wound severity. This study will be conducted at HealthPartners Institute's Regions Hospital in St. Paul, Minnesota. Data compiled by the Wound, Ostomy, and Continence Nursing Department reveals an annual pool of more than 300 eligible patients at Regions Hospital. The study will consist of the following visits: - Visit C1 (Day -35 to -6): Screening in-person clinic visit - Visit C2, Day 1: Baseline appointment clinic visit - Visit C3, Week 1: Clinic visit - Visit C4, Week 2: Clinic visit - Visit C5, Week 3: Clinic visit - Visit C6, Week 4: Final appointment during treatment period; clinic visit After the Baseline Data Collection Period, participants will be randomized by BMI and three stoma types: ileostomy with loop stoma, ileostomy with end stoma, and enterocutaneous fistulas (ECFs) into 1 of 2 treatment groups: - Limpet: Participants in this arm will utilize the subject device as intended to be used. - Control: Standard treatment with standard of care adhesive ostomy pouches. On Day 1, Limpet participants will have their device placed by a clinician. Upon discharge, a portable vacuum pump will be issued to provide negative pressure (e.g., 3M Prevena or Snap; ConvaTec Avelle; or Pensar MicroDoc). These systems are compact, fit in a pocket, are simple to operate, and commonly used in the outpatient setting to provide multiple weeks of therapy. Participants will be furnished with materials to address potential dressing problems while at home: extra waste pouches, adhesive drape strips for sealing air leaks, and standard of care adhesive ostomy pouches and accessories as a backup plan in the event of a Limpet failure. On Day 1, Control participants will receive standard of care adhesive ostomy pouch dressings. Participants will be furnished with materials to address potential dressing problems while at home: adhesive ostomy pouches and accessories for pouch replacement. The Control group will change their pouches at home 2 to 5 times per week or more frequently if needed, as per the current standard of care. The brand and model of pumps and adhesive ostomy pouches used by Limpet and Control participants will be recorded for comparison purposes. Participants will return for clinic visits approximately every 7 days to have their device replaced. Participants will have their final study appointment approximately 30 days post-surgery. Clinical support will help the participant troubleshoot dressing problems; and if necessary, bring the patient into the study site for an immediate dressing change.
Phase
N/ASpan
46 weeksSponsor
HealthPartners InstituteSaint Paul, Minnesota
Recruiting
Prospective Non-Interventional Study Comparing Osimertinib +/- Chemotherapy for EGFR-Mutated NSCLC Patients
This study tests the feasibility of using real-world data collection through the recruitment of well-characterized patients into a registry, spanning academic and community practice sites to determine patient outcomes in all-comers. The scientific community would further benefit from a greater understanding of the safety and effectiveness of newly approved therapies prescribed in routine clinical practice. Non-interventional (observational) study with 250 participants per exposure group (maximum accrual up to 538 total participants) comparing outcomes in patients with EGFR-mutated NSCLC, not being treated in a clinical trial but receiving standard of care osimertinib +/- chemotherapy. The treating physician determines whether a patient will receive standard of care single agent osimertinib or osimertinib with chemotherapy and the planned treatment is recorded at registration. Clinical/Imaging assessments will be per the treating physician.
Phase
N/ASpan
259 weeksSponsor
PrECOG, LLC.Saint Paul, Minnesota
Recruiting
Study to Investigate Luveltamab Tazevibulin in Adults with Advanced or Metastatic Non-small Cell Lung Cancer
This is a multicenter, open-label study. The study is designed to assess the preliminary efficacy and safety of luveltamab tazevibulin, an anti-FOLR1 antibody drug conjugate (ADC) in previously treated subjects with advanced or metastatic NSCLC that expresses FOLR1. Subjects will receive luveltamab tazevibulin administered intravenously every 3 weeks until disease progression, intolerable toxicity, elective withdrawal from the study, or study termination.
Phase
2Span
115 weeksSponsor
Sutro Biopharma, Inc.St. Paul, Minnesota
Recruiting
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Co-Primary Endpoints - Rett Syndrome Behavior Questionnaire (RSBQ) total score - Change from Baseline to Week 12 - Clinical Global Impression-Improvement (CGI-I) Score at Week 12 Key Secondary Endpoint Change from Baseline to Week 12 in: • Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist- Social Composite Score (CSBS-DP-IT Social)
Phase
3Span
39 weeksSponsor
Biomed Industries, Inc.Saint Paul, Minnesota
Recruiting
Safety and Pharmacokinetics Study of MBX 2109 in Adult Subjects With Normal and Impaired Renal Function
A Phase 1, Open-Label, Parallel-Group, Single-Dose Adaptive Study to Evaluate the Safety and Pharmacokinetics of MBX 2109 in Adult Subjects with Normal and Impaired Renal Function
Phase
1Span
21 weeksSponsor
MBX BiosciencesMinneapolis, Minnesota
Recruiting
Healthy Volunteers
Study of Targeted Therapy vs. Chemotherapy in Patients With Thyroid Cancer
PRIMARY OBJECTIVE: I. To compare progression-free survival (PFS) in patients with BRAF V600Em differentiated thyroid cancer who progressed on frontline multikinase inhibitor treated with dabrafenib/trametinib or cabozantinib. SECONDARY OBJECTIVES: I. To compare the objective response rate in patients with BRAF V600Em differentiated thyroid cancer that progressed on frontline multikinase inhibitor treated with dabrafenib/trametinib or cabozantinib. II. To compare the duration of response in patients with BRAF V600Em differentiated thyroid cancer that progressed on frontline multikinase inhibitor treated with dabrafenib/trametinib or cabozantinib. III. To compare the overall survival in patients with BRAF V600Em differentiated thyroid cancer that progressed on frontline multikinase inhibitor treated with dabrafenib/trametinib or cabozantinib. IV. To compare the PFS2 in patients with BRAF V600Em differentiated thyroid cancer that progressed on frontline multikinase inhibitor treated with dabrafenib/trametinib or cabozantinib. V. To compare the safety/tolerability in patients with BRAF V600Em differentiated thyroid cancer that progressed on frontline multikinase inhibitor treated with dabrafenib/trametinib or cabozantinib. QUALITY OF LIFE OBJECTIVE: I. To assess patient tolerability of treatment using the Functional Assessment Cancer Therapy General (FACT G)P5 and general quality of life using the FACT-G7. OUTLINE: Patients are randomized to 1 of 2 arms. Patients may crossover to other treatment arm at the time of progression. ARM A: Patients receive dabrafenib orally (PO) twice per day (BID) and trametinib PO once per day (QD) on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo computed tomography (CT) scan, blood sample collection and may undergo magnetic resonance imaging (MRI) throughout the study. ARM B: Patients receive cabozantinib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo CT scan, blood sample collection and may undergo MRI throughout the study. After completion of study treatment, patients are followed up every 3 months for 2 years and then every 6 months thereafter up to 5 years.
Phase
3Span
319 weeksSponsor
ECOG-ACRIN Cancer Research GroupSaint Paul, Minnesota
Recruiting