A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

Imara

Status and phase: Completed; Phase 2

Conditions: Sickle Cell Disease

Treatments: Drug: IMR-687; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03401112

IMR-SCD-102

2017-000653-39 (EudraCT Number)

Details and patient eligibility

About

Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).

Full description

This is a proof-of-concept study in adult SCA participants, ages 18 to 55 years old, to examine the safety, tolerability, and pharmacokinetic (PK), as well as the potential pharmacodynamic (PD) effects and clinical efficacy, of IMR-687 across a range of doses.

IMR-687 was administered in 2 populations of participants with SCA: those who were not receiving hydroxyurea (HU) and those who were receiving a stable dose of HU according to standard of care.

Enrollment: 100 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Male or female participants with confirmed SCA
• Age 18 to 55 years, inclusive
• For participants on HU, must have been on a stable dose for at least 60 days prior to screening

Key Exclusion Criteria:

• Total hemoglobin >12.5 or <6 grams/deciliter
• Red blood cell transfusion within 60 days of baseline
• >7 hospitalizations for vaso-occlusive crises (VOCs) within the last year
• Estimated glomerular filtration rate <50 milliliter/minute
• Aspartate aminotransferase/alanine aminotransferase >3x the upper limit of normal

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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100 participants in 3 patient groups, including a placebo group

IMR-687 50 mg/100 mg

Experimental group

Description:

A starting dose of IMR-687 50 mg with dose escalation after 4 or 12 weeks, up to 100 mg was administered to participants. Duration of administration was 16 (Week 17) or 24 weeks (Week 25).

Treatment:

Drug: IMR-687

IMR-687 100 mg/200 mg

Experimental group

Description:

A starting dose of IMR-687 100 mg with dose escalation after 4 or 12 weeks, up to 200 mg was administered to participants. Duration of administration was 24 weeks (Week 25).

Treatment:

Drug: IMR-687

Placebo

Placebo Comparator group

Description:

Matching placebo was administered for 16 (Week 17) or 24 weeks (Week 25).

Treatment:

Drug: Placebo