An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia

Phase 2Active Not RecruitingNCT04053803

Imara, Inc.30 enrolled

Overview

This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102). The open-label extension study will evaluate long-term safety and tolerability.

This is an open-label extension study of IMR-687 in adult patients with SCA who were previously participants in the Phase 2a study titled "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)." This open-label extension study with IMR-687 will evaluate the long-term safety and tolerability of IMR 687 in adult SCA patients. Exploratory long-term PD parameters will also be examined.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sickle Cell Disease

Interventions

IMR-687DRUG

Oral administration of once daily IMR-687

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Completed Study IMR-SCD-102. 2. Female subjects must not be pregnant, not be breast feeding, and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner. 3. Subjects must be capable of giving informed consent and reading and signing the informed consent form after the nature of the study has been fully explained to them 4. Subjects must be willing and able to complete all study assessments and procedures and to communicate effectively with the investigator and site staff. Exclusion Criteria: 1. Subjects with Hb \>12.5 g/dL or \<6 g/dL 2. Subjects with known active hepatitis B or hepatitis C, with active or acute event of malaria or who are known to be positive for human immunodeficiency virus (HIV) 3. eGFR \<50 mL/min 4. AST/ALT \> 3x the upper limit of normal

Locations (7)

University of Connecticut Health Center

Farmington, Connecticut, United States

Foundation for Sickle Cell Disease Research

Hollywood, Florida, United States

Baylor Scott & White Medical Center - Temple

Temple, Texas, United States

Bristol Haematology and Oncology Centre

Bristol, United Kingdom

Outcomes

Primary Outcomes

Proportion of patients with adverse events and serious adverse events

1. Incidence of Adverse Events 2. Incidence of Serious Adverse Events

Time frame: Baseline to Month 49

Proportion of patients with changes in safety cardiac parameters

a. Changes in 12-lead ECG parameters that are clinically significant and measured in milliseconds (ms). The parameters are: PR interval, QRS duration, QT interval, ST segment duration and T wave duration.

Time frame: Baseline to Month 49

Proportion of patients with changes in clinical laboratory tests

a. Clinically significant changes in clinical laboratory tests including serum chemistry, serum hematology and urinalysis

Time frame: Baseline to Month 49

Proportion of patients with clinically significant abnormal vital signs

1. Blood pressure measured in mmHg 2. Pulse measured in beats per minute 3. Respiration rate measured in breaths per minutes 4. Temperature as measured in degrees F0 or C0

Time frame: Baseline to Month 49

Data from ClinicalTrials.gov

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