A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease

IMARA, Inc.0 sites99 target enrollmentSeptember 18, 2020

ConditionsThe population for this study includes subjects with the following forms of SCD: homozygous sickle hemoglobin (HbSS), sickle-ß° (HbSB°) thalassemia, and sickle-ß? (HbSB?) thalassemia.Sickle cell

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: The population for this study includes subjects with the following forms of SCD: homozygous sickle hemoglobin (HbSS), sickle-ß° (HbSB°) thalassemia, and sickle-ß? (HbSB?) thalassemia.
Sponsor: IMARA, Inc.
Enrollment: 99
Status: Not yet recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

September 18, 2020

End Date

TBD

Last Updated

last year

Study Type

Interventional

Sex

All

Investigators

Sponsor

IMARA, Inc.

Eligibility Criteria

Inclusion Criteria

•1\. Male or female aged \=18 to \=65 years at the time of informed consent form (ICF) signing.
•2\. Confirmed diagnosis of SCD (HbSS, HbSB° thalassemia, or HbSB? thalassemia) in the medical record; if not available, the diagnosis must be confirmed at the site’s local laboratory instead.
•3\. Subjects must have had at least 1 and no more than 12 documented episodes of VOC in the past 12 months at the time of ICF signing and at randomization (Day 1\). For study eligibility, VOC is defined as a documented episode of an acute painful crisis (for which there was not an explanation other than VOC) that involved moderate to severe pain lasting for at least 2 hours and at least one of the following:
•Use of escalated analgesia (including healthcare professional\-instructed use of an analgesic prescription)
•A hospital, emergency department, or clinic visit and/or healthcare telephone consultation at the time of occurrence
•Diagnosis of acute chest syndrome (ACS) (defined as an acute illness characterized by fever and/or respiratory symptoms, accompanied by a new pulmonary infiltrate on a chest X\-ray), hepatic sequestration, or splenic sequestration
•4\. Hemoglobin (Hb) of \>5\.5 and \<10\.5 g/dL.
•5\. Absolute reticulocyte count \=80 × 10?/L.
•6\. Subjects receiving HU must have received it continuously for at least 6 months prior to signing the ICF, and must have been on a stable dose for at least 3 months prior to signing the ICF, with no anticipated need for dose adjustments during the study including the screening period, in the opinion of the investigator.
•7\. Female subjects must not be pregnant or breastfeeding and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner. Male or female subjects must meet at least one of the following criteria:

Exclusion Criteria

•Subjects who meet any of the following criteria will be excluded from the study:
•1\. Hospital discharge for sickle cell crisis or other vaso\-occlusive event within the 4 days prior to randomization (Day 1\).
•2\. Red blood cell transfusion within 60 days of signing the ICF or on chronic transfusion therapy regimen. Transfusion status must be reassessed at randomization (Day 1\).
•Note: If a subject requires a transfusion during the screening period, they may be rescreened up to one time.
•3\. Subjects with hereditary persistence of HbF (i.e., HbF \>25% at screening).
•4\. Subjects with known active hepatitis A, hepatitis B, or hepatitis C, with active or acute event of malaria, or who are known to be positive for human immunodeficiency virus (HIV).
•5\. For female subjects of childbearing potential, a positive serum human chorionic gonadotropin (hCG) test (screening) or a positive urine hCG test at randomization (Day 1\).
•6\. Estimated glomerular filtration rate (eGFR) \<45 mL/min as calculated by the equation from the Modification of Diet in Renal Disease Study using creatinine, age, sex, and ethnicity.
•7\. Alanine aminotransferase or aspartate aminotransferase \>3 × the upper limit of normal.
•8\. Body mass index (BMI) \<17\.0 kg/m² and a total body weight \<45 kg; or a BMI \>35 kg/m2\.