ANZCTR search results

This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously participated in Study MLB-01-005 (Fortify). Participants will receive BBP-418 administered orally at protocol-defined doses and schedules. The study will assess long-term safety through monitoring of adverse events, clinical laboratory evaluations, and other safety assessments. Efficacy will be evaluated using functional measures and other clinical endpoints relevant to LGMD2I/R9. Participants will be followed for up to 36 months, with a final safety follow-up assessment conducted approximately 30 days after the last dose of study drug.

A Study of GFH276 Combined With Cetuximab or Chemotherapy in Participants With Solid Tumors and Pancreatic Ductal Adenocarcinoma (PDAC) Harboring RAS Mutation

The primary purpose of this study is to evaluate the safety and tolerability of QX-4533 following oral administration of single and multiple ascending doses in healthy participants.

This Phase 2 study aims to assess the efficacy of SC injections of STP705 for the reduction of Submental fat in apopulation of healthy adult volunteers, and to further establish its safety and tolerability. The primary objective is to evaluate the efficacy of STP705 injection for Submental fat reduction. The second objective is to evaluate the safety and tolerability of STP705 injection. This is an open-label study. Eligible participants will be sequentially enrolled into 1 of 3 treatment cohorts, as follows: Cohort 1: 40 µg STP705 per injection. Up to 280 µg in total (n = 10 participants) Cohort 2: 64 µg STP705 per injection. Up to 448 µg in total (n = 10 participants) Cohort 3: 80 µg STP705 per injection. Up to 560 µg in total (n = 10 participants) The STP705 powder will be reconstituted with dextrose 5% in water (D5W). A total of 7 injections (3.5 mL total volume) in a single treatment may be administered to each participant in the submental triangle area (ie, for a total dose of up to 280 µg, 448 µg or 560 µg STP705 for each cohort). The Submental fat injections will be performed at Day 1/Baseline, Day 29, and Day 57 , pending favourable reviews by the Investigator of injection site reactions (ISRs), local skin reactions (LSRs), and participant assessments of the injection sites (pain, stinging, and burning). To proceed with treatment at Day 29 or Day 57, the following factors will be considered: 1. the total tolerability score per injection site (defined as the sum of the "Investigator assessed LSRscores" and the "participant-reported scores of injection site pain and stinging/burning" measured prior totreatment must be \< 6, and 2. there are no findings (eg, systemic and/or local adverse events \[AEs\]) that would preclude treatment, in the opinion of the Investigator. The tolerability score threshold should be interpreted in conjunction with clinical judgment. If condition a) is not met, participants with a total tolerability score = 6 will not be injected with the study medications. After the 3rd injection, the participant will be followed for 1 month (through to Day 85). To proceed with dose escalation (ie, from Cohort 1 to Cohort 2 or from Cohort 2 to Cohort 3), the last participant in the previous dose cohort must have completed at least 7 days of postdose safety follow-up after the first injection, and available safety data will be reviewed by the Sponsor and Medical Monitor. The decision to escalate dose will be made upon review of all data by the Medical Monitor and the Sponsor and recommendation by the Sponsor.

TABLO (Tranexamic Acid to reduce Blood Loss after varus derotation Osteotomy) is a clinical trial of postoperative tranexamic acid vs placebo in non-ambulatory children with cerebral palsy (CP) undergoing reconstructive hip surgery. Improving surgical outcomes is a high priority in this patient population given the high risk of bleeding and the diminished capacity for these children to withstand substantial blood loss. Preliminary data from the study institution indicates that approximately one third of these patients receive transfusion of blood products in the postoperative period. There is growing evidence that hidden blood loss occurring in the postoperative period is substantial and can potentially be attenuated with the administration of Tranexamic Acid (TXA). However, trials on postoperative TXA have been carried out exclusively in adult surgical populations.

The goal of this clinical trial is to determine safety, tolerability, pharmacokinetics, preliminary efficacy, and Maximum Tolerable Dose (MTD), of intralesional FLD-103 when administered to subjects with Basal Cell Carcinoma (BCC). The main questions it aims to answer are: * Is FLD-103 safe and well tolerated? * What is a safe dose of FLD-103 for future studies? * How much FLD-103 enters the bloodstream and how long does it take to be cleared from the body? * Does FLD-103 reduce the size of the tumor? Participants will: * Receive either a Single Ascending Dose (SAD) of FLD-103 or Multiple Ascending Doses (MAD) of FLD-103 once weekly for four (4) weeks. * Visit the clinic a day after receiving a dose and once weekly for four (4) weeks after that for checkups and tests.

This study will evaluate the safety, tolerability, and efficacy of XYA02 in participants with advanced solid tumors.

The goal of this clinical trial is evaluate the safety, pharmacokinetics, and pharmacodynamics of NS-079 with its main metabolite (NS-079-M1) in healthy participants. The main questions it aims to answer are: * Is NS-079 safe and tolerable in heathy participants under tested dosing regimen? * What is the pharmacokinectic profile of NS-079 in healthy participants under tested dosing regimen and the effect of paroxetine? Researchers will compare NS-079 to a placebo to see the safety and tolerability when use NS-079.

This study will evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of TO-O-1007, a biodegradable sustained-release intravitreal implant, for the treatment of Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). A long-acting intravitreal implant providing sustained delivery of API over 6 months may slow the progression of GA lesions while reducing treatment burden associated with frequent intravitreal injections. TO-O-1007 may offer a convenient treatment option with the potential to preserve retinal structure and visual function while maintaining an acceptable safety profile.

Study investigating the safety, tolerability, pharmacokinetics and early efficacy with KIT2014 over 7 days of treatment in moderate to severe chronic obstructive pulmonary disease patients.