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

Cardurion Pharmaceuticals, Inc.100 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

100

Conditions

Sickle Cell Disease

Interventions

IMR-687DRUG

Oral administration of IMR-687 once daily with or without HU.

PlaceboDRUG

Oral administration of placebo once daily with or without HU.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 55 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Male or female participants with confirmed SCA * Age 18 to 55 years, inclusive * For participants on HU, must have been on a stable dose for at least 60 days prior to screening Key Exclusion Criteria: * Total hemoglobin \>12.5 or \<6 grams/deciliter * Red blood cell transfusion within 60 days of baseline * \>7 hospitalizations for vaso-occlusive crises (VOCs) within the last year * Estimated glomerular filtration rate \<50 milliliter/minute * Aspartate aminotransferase/alanine aminotransferase \>3x the upper limit of normal

Locations (13)

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

University of Connecticut Health Center

Farmington, Connecticut, United States

Foundation for Sickle Cell Disease Research

Hollywood, Florida, United States

Outcomes

Primary Outcomes

Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs)

An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. An SAE was defined as any AE that resulted in 1 or more of the following outcomes: death, required or prolonged hospitalization, life-threatening, persistent or significant disability/incapacity, congenital anomaly or birth defect, or other medically important event. A summary of serious and all other non-serious AEs, regardless of causality, is located in the Reported Adverse Events module.

Time frame: Day 1 (after dosing) through up to Week 24

Secondary Outcomes

Pharmacokinetic (PK) Of Participants Who Did Not Concomitantly Receive HU: Maximum Plasma Concentration (Cmax) Of IMR-687

For PK assessments of participants who did not concomitantly receive HU, serial blood samples for IMR-687 plasma concentrations were drawn predose at 0.5, 1, 1.5, 2, 4, 6, and 8 hours after administration of study drug; and at 24 hours after administration of study drug. Day 1 (single-dose) and steady-state (Week 25) assessments are presented.

Time frame: Day 1 and Week 25

PK Of Participants Who Did Not Concomitantly Receive HU: Area Under The Concentration-time Curve (AUC) From Time 0 To 24 Hours Postdose (AUC0-24h) Of IMR-687

Time frame: Day 1 and Week 25

PK Of Participants Who Concomitantly Received HU: Cmax Of IMR-687

For PK assessments of participants who concomitantly received HU, serial blood samples for IMR-687 PK were drawn predose at 0.5, 1, 1.5, 2, 4, 6, and 8 hours after administration of study drug; and at 24 hours after administration of study drug. Day 1 (single-dose) and steady-state (Week 17) assessments are presented.

Data from ClinicalTrials.gov

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