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NCT05141006
Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) is a chronic and debilitating urological complex of disorders characterized by symptoms of bladder pain or discomfort, mostly upon bladder filling, and often accompanied by lower urinary tract symptoms (LUTS). This study will assess how safe and effective BOTOX (onabotulinumtoxinA) is in treating IC/BPS. Adverse events and change in disease symptoms will be evaluated. BOTOX (onabotulinumtoxinA) is an investigational drug being developed for the treatment of Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS). Study doctors randomly assign the participants to 1 of 2 groups, called treatment arms, to receive BOTOX or placebo. There is a 1 in 2 chance that participants will be assigned to placebo. Approximately 80 female participants, aged 18 to 75 years, with a diagnosis of IC/BPS will be enrolled in approximately 40 sites in the United States and Canada. Participants will receive BOTOX or placebo injected into the bladder on Day 1 and will be followed for at least 12 weeks in treatment 1. Eligible participants may request additional dose of BOTOX between Weeks 12 and 24, and will be followed for 12 weeks in treatment period 2. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.
NCT06333028
A Study to Evaluate the enVista® Aspire (EA) intraocular lens in Subjects Undergoing Cataract Extraction
NCT02646839
This is a phase II, open-label, non-randomized, prospective study of haploidentical transplantation using KIR-favorable donors for children with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). The relationship of KIR2DL1 polymorphisms to survival in children with these diseases undergoing any approach to allogeneic HCT during the study time frame will also be determined.
NCT06465745
This is a prospective, single arm, multicenter, clinical trial designed to evaluate the safety and performance of the AltaValve System for the treatment of mitral regurgitation in a targeted patient population.
NCT04741646
We will conduct a 12-month, double-blind, randomized, placebo-controlled trial to assess the effects of therapy with ferric citrate (FC) on changes in intact FGF23 levels (iFGF23, primary endpoint) in 160 pediatric patients (80 in each of the two arms) aged 6-18 years of either sex with chronic kidney disease (CKD) stages 3-4 and age-appropriate normal serum phosphate levels. Participants will be randomized to one of the two groups: 1) FC or 2) FC placebo. Participants will be recruited from 20 core clinical sites.
NCT05093504
Prospective, Multicenter, Double-blind, Randomized, Study to Evaluate DrugSorb-ATR Removal of Apixaban and Rivaroxaban to Reduce Likelihood of Serious Bleeding in Patients Undergoing Urgent Cardiothoracic Surgery
NCT02476916
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.
NCT04533438
The purpose of this study is to compare the RhinAer procedure with radiofrequency (RF) energy to sham procedure for treatment of chronic rhinitis.
NCT03231306
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. The primary objective is to determine if there is an adequate level of disease responsiveness to binimetinib in children and adults with NF1 and inoperable plexiform neurofibromas. The objective response to binimetinib is defined as ≥ 20% decrease in tumor volume reduction by 12 courses.
NCT05259917
This study is a randomized, double-blind, placebo-controlled, phase III, three-way crossover clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adolescent and adult Patients
NCT05644977
The primary purpose of the study is to evaluate the safety and tolerability of emraclidine administered orally to healthy elderly participants in Part A (multiple ascending doses) and participants with dementia due to Alzheimer's disease (AD) in Part B.
NCT02616484
The objective of this research study is to conduct a pivotal phase 3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital lactic acidosis and is a frequently fatal metabolic disease of childhood for which no proven treatment exists. The investigators predict that DCA represents targeted potential therapy for PDCD because of its ability to increase both the catalytic activity and stability of the enzyme complex. The conclusions of numerous laboratory and clinical investigations are consistent with this postulate and have led to the designation of DCA as an Orphan Product for congenital lactic acidosis by the Food and Drug Administration. A novel Observer reported outcome (ObsRO) survey that is completed by study participant's parent/caregiver, is the efficacy outcome measure. Funding Source - FDA OOPD
NCT04253262
This is a single arm Phase Ib/II, open label, safety, pharmacokinetic and efficacy clinical study in adult patients with metastatic castration-resistant prostate cancer (mCRPC). Patients will be treated with the combination of copanlisib and rucaparib for as long as the patient does not have clinically significant progressive disease and/or unacceptable toxicity and/or as long as the investigator deems that the patient is benefiting from treatment. Treatment may also be stopped if the patient withdraws consent, or study termination occurs.
NCT04016402
This study investigates how brains change as a result of a treatment for depression called repetitive transcranial magnetic stimulation (rTMS). People who receive rTMS will have pictures of their brains and brain activity taken by a magnetic resonance imaging (MRI) scanner before and after their treatment, as well as up to three times during the six-week treatment course. These images will be examined to see if the rTMS is placed correctly to help treat their symptoms, and what changes in brain activity are happening during the rTMS treatment
NCT04500847
This is a randomized, double-blind clinical trial of a daily oral dose of 200 mg emtricitabine vs. placebo in 35 participants with biomarker-confirmed MCI or mild to moderate dementia due to Alzheimer's disease. Study duration for each subject participating in the placebo-controlled research study will be approximately 12 months (up to a 3 months Screening Period, Baseline visit (1 month), 6 months of placebo or emtricitabine dosing, and 1 month follow-up). Participants will have up to 2 months to complete all procedures for the month 6 study visit.
NCT05505578
The goal of "GamerFit" is to test the delivery of a theory-based mHealth app that utilizes social support, exergaming, and telehealth coaching to improve PA levels, sleep, and psychiatric symptoms among youth participants (ages 13-17 y) with PD. In order to aid future intervention optimization, up to 65 participants with at least one PD will be randomized, with 30-35 using the GamerFit app with weekly telehealth coaching sessions and 30-35 using a commercial healthy habits app as a comparator group
NCT02632448
The main purpose of this 3-part study is to evaluate the safety and efficacy of the study drug known as LY2880070 in participants with advanced or metastatic solid tumors.
NCT04865679
This pilot study evaluates the tolerability and feasibility of the Axoguard Large-Diameter Nerve Cap (sizes 5-7 mm) for protecting and preserving terminated nerve endings after limb trauma or amputation when immediate attention to the nerve injuries is not possible.
NCT03569891
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.
NCT04208412
This study is a randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adult subjects.