FORTITUDE

• Research type

  Research Study

• Full title

  A Global Phase 2b, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients with Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy – FORTITUDE-HCM

• IRAS ID

  1012709

• Contact name

  Karen Jauregui

• Contact email

  info@imbria.com

• Sponsor organisation

  Imbria Pharmaceuticals, Inc.

• Clinicaltrials.gov Identifier

  NCT07023614

• Research summary

  Non-obstructive hypertrophic cardiomyopathy (nHCM) is a condition where the heart muscle becomes thicker than normal, affecting how well the heart pumps blood. People with nHCM often experience a range of symptoms, such as shortness of breath during everyday activities or exercise, chest discomfort or tightness, fatigue, dizziness, palpitations (feeling like the heart is beating too hard or too fast). Currently, there are no approved medicines specifically for nHCM. Verapamil or betablockers are often used to help manage symptoms of nHCM but for some people, a heart transplant is the only long-term solution. Therefore, there is a need to find new and effective treatments for symptomatic nHCM.
  Ninerafaxstat is a new investigational medicine being developed to treat people with symptomatic nHCM. It is not yet approved. Ninerafaxstat helps the heart make energy more efficiently by shifting its energy source, so it requires less oxygen. This increased efficiency helps the heart work with less strain, potentially improving symptoms for people with nHCM, meaning it may be easier to exercise or perform daily activities.
  The main goals of this study are to check how well ninerafaxstat works, and how safe it is for people with symptomatic nHCM. The study will compare ninerafaxstat to a placebo. Potential participants can enter the study if they meet certain eligibility criteria. Participants will be in the study for about 20 weeks. This study has 4 parts:
  Screening: Up to 6 weeks, the study doctor will perform a series of tests to check if participants are eligible to take part in the study
  Randomisation: 1 day, participants will be assigned to either treatment group, and take the first dose of the study medicine
  Treatment period: 12 weeks, participants will take study medicine at home and attend several visits at the clinic
  Safety follow-up period: 2 weeks, the study doctor will make sure participants are feeling okay after they finish taking the study medicine

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  25/NE/0160

• Date of REC Opinion

  14 Oct 2025

• REC opinion

  Further Information Favourable Opinion