NCT03401112

A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 100 (actual)
• Sponsor: Cardurion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).

Detailed description

This is a proof-of-concept study in adult SCA participants, ages 18 to 55 years old, to examine the safety, tolerability, and pharmacokinetic (PK), as well as the potential pharmacodynamic (PD) effects and clinical efficacy, of IMR-687 across a range of doses. IMR-687 was administered in 2 populations of participants with SCA: those who were not receiving hydroxyurea (HU) and those who were receiving a stable dose of HU according to standard of care.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	IMR-687	Oral administration of IMR-687 once daily with or without HU.
DRUG	Placebo	Oral administration of placebo once daily with or without HU.

Timeline

• Start date: 2018-01-26
• Primary completion: 2020-08-28
• Completion: 2020-08-28
• First posted: 2018-01-17
• Last updated: 2025-05-15
• Results posted: 2022-04-14

Locations

13 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study