Clinical trial NCT04053803: An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Sickle Cell Disease
Lead sponsor: Imara, Inc.
Sponsor class: INDUSTRY
First posted: 2019-08-13
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Proportion of patients with adverse events and serious adverse events
timeFrame: Baseline to Month 49
measure: Proportion of patients with changes in safety cardiac parameters
timeFrame: Baseline to Month 49
measure: Proportion of patients with changes in clinical laboratory tests
timeFrame: Baseline to Month 49
measure: Proportion of patients with clinically significant abnormal vital signs
timeFrame: Baseline to Month 49
Secondary outcomes:
Other outcomes:
Locations:
city: Farmington
country: United States
facility: University of Connecticut Health Center
state: Connecticut
city: Hollywood
country: United States
facility: Foundation for Sickle Cell Disease Research
state: Florida
city: Temple
country: United States
facility: Baylor Scott & White Medical Center - Temple
state: Texas
city: Bristol
country: United Kingdom
facility: Bristol Haematology and Oncology Centre
city: London
country: United Kingdom
facility: University College London Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Guy's and St Thomas Hospital CRF
city: London
country: United Kingdom
facility: Royal London Hospital
Study officials:
affiliation: Imara, Inc.
name: Karen Tang, MD
role: STUDY_DIRECTOR
References: