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RARE Clinical Trials Hub


Clinical trials are posted by members of the Global Genes RARE Corporate Alliance. They are responsible for ensuring that the studies follow all applicable laws and regulations. Global Genes staff do not verify the scientific validity or relevance of the submitted information beyond a verification of Corporate Alliance membership. Note: There may be more study sites and/or diseases associated with a clinical trial than those listed. Please click ‘Learn More’ on a post for more detailed information, including information about the status of the trial.

Posted | 2022-12-08
Estimated Completion | 2024-10-01
Last Updated | 2022-12-09
STEER is a Novartis research study evaluating the clinical efficacy, safety and tolerability of OAV101 IT in patients with SMA Type 2 between 2 to <18 years of age who are able to sit but never walked and have never received treatment for SMA. This research study is a randomized, double-blind, sham-controlled, global Phase 3 clinical trial. In STEER, patients will be randomly assigned to one of two possible treatment groups, including: 1) Treatment with OAV101 IT 2) Sham procedure, a needle prick in the lower back without the administration of OAV101 IT Eligible patients who have the sham procedure in the first period of the study will receive OAV101 IT in the second period of the study.
Type 2 Spinal Muscular Atrophy
Posted | 2022-12-16
Estimated Completion | 2023-08-31
Last Updated | 2023-01-04
California, USA
Massachusetts, USA
Texas, USA
Kentucky, USA
New York, USA
United States
This is a Phase 2, randomized, open-label, parallel-group, multi-center study for patients with documented carcinoid syndrome requiring medical therapy including at least 1 instance of an elevated 5-HIAA level. The study includes a Screening Period of up to 12 weeks. After completion of Screening, subjects will be randomly assigned to 40 mg QD vs. 80 mg QD open-label dose groups for 8 weeks, referred to as the Randomized Treatment Phase. Following the completion of the Randomized Treatment Phase, subjects may be eligible to enter the Open-Label Extension (OLE) Phase of the study in which they will receive paltusotine for 50 weeks. At the end of the treatment period, there will be a follow-up visit 4 weeks after the last dose of paltusotine. The total duration of paltusotine treatment is up to 58 weeks or up to 15 months.
Carcinoid syndrome (Malignant carcinoid syndrome)


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