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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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An Open-label Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia) Who Participated in Study IMR-SCD-102

Conditions
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Sickle Cell Disease

Intervention / Treatment
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* Drug: IMR-687


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Completed Study IMR-SCD-102. 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. 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 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: Subjects with Hb >12.5 g/dL or <6 g/dL 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) eGFR <50 mL/min AST/ALT > 3x the upper limit of normal

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Open Label<br> | Drug: IMR-687<br>* Oral administration of once daily IMR-687<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of patients with adverse events and serious adverse events | Incidence of Adverse Events Incidence of Serious Adverse Events | 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. | 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 | Baseline to Month 49 | 
| Proportion of patients with clinically significant abnormal vital signs | Blood pressure measured in mmHg Pulse measured in beats per minute Respiration rate measured in breaths per minutes Temperature as measured in degrees F0 or C0 | Baseline to Month 49 |