Sickle Cell Disease
Conditions
Brief summary
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
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.
Interventions
DRUGIMR-687
Oral administration of once daily IMR-687
Sponsors
Imara, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients with adverse events and serious adverse events | Baseline to Month 49 | 1. Incidence of Adverse Events 2. Incidence of Serious Adverse Events |
| Proportion of patients with changes in safety cardiac parameters | Baseline to Month 49 | 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. |
| Proportion of patients with changes in clinical laboratory tests | Baseline to Month 49 | a. Clinically significant changes in clinical laboratory tests including serum chemistry, serum hematology and urinalysis |
| Proportion of patients with clinically significant abnormal vital signs | Baseline to Month 49 | 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 |
Countries
United Kingdom, United States
Outcome results
None listed