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A Phase 3, Single-Blind Study to Evaluate the Effect of Eleclazine on Shortening of the QT Interval, Safety, and Tolerability in Subjects with Long QT Syndrome type 3.

The primary objective of this study is to evaluate in subjects with LQT3:- The effect of oral eleclazine on mean daytime QTcF interval (in msec) after 24 weeks of treatment with eleclazine (based on standard 12-lead electrocardiogram [ECG] data)The…

Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with Hereditary Angioedema (HAE)

This study has been transitioned to CTIS with ID 2024-512317-40-00 check the CTIS register for the current data. Phase I Primary Objective• To evaluate the safety of NTLA-2002 and identify dose(s) for use in Phase 2Secondary Objectives• To evaluate…

A 2-Stage (Open-Label Followed by Randomized Double-Blind, Placebo-Controlled Stage), Phase 2 Trial of Setmelanotide in Patients with Specific Gene Variants in the Melanocortin-4 Receptor Pathway

Primary:• To evaluate the proportion of patients with obesity with genetic variants in a specific gene in the melanocortin-4 receptor (MC4R) pathway who achieve a clinically meaningful reduction in body weight in response to setmelanotide at the end…

Phase 1 Dose Escalation Study Assessing the Safety and Pharmacokinetics of PTC518 in Healthy Subjects

Primary ObjectivesPart 1:To characterize the safety and tolerability of single ascending doses of PTC518 in healthy subjects.Part 2:To characterize the safety and tolerability of PTC518 administered for 14 or up to 21 days in healthy subjects.Part 3…

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

This study has been transitioned to CTIS with ID 2024-511852-42-00 check the CTIS register for the current data. Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or…

A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis patients with Fused in Sarcoma mutations (FUS-ALS)

This study has been transitioned to CTIS with ID 2024-512163-31-00 check the CTIS register for the current data. Primary objective: To evaluate the clinical efficacy of ION363 in clinical functioning and survival in FUS-ALS patients. Secondary…

A Phase 1, Double-blind, Randomized, Placebo-controlled, Single Ascending Dose Study To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Paltusotine In Healthy Subjects

In this study we will investigate how safe specific doses of the compound paltusotine are and how well these are tolerated when they are used by healthy participants.We also investigate how much of the compound is broken down and absorbed in the…

Can ELABELA induce monozygotic twinning?

The primary objective of this study is to investigate if exogenously supplied ELABELA can induce MZ twinning in cultured human embryos. This will be done using time-lapse monitoring of donated human embryos that will be treated with synthetic…