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

NCT03401112 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: IMR-SCD-1022017-000653-39
• Study Type: interventional
• Target: 100
• Locations: 2 countries
• Sites: 13

Brief Summary

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

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs)

  An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. An SAE was defined as any AE that resulted in 1 or more of the following outcomes: death, required or prolonged hospitalization, life-threatening, persistent or significant disability/incapacity, congenital anomaly or birth defect, or other medically important event. A summary of serious and all other non-serious AEs, regardless of causality, is located in the Reported Adverse Events module.

  Day 1 (after dosing) through up to Week 24

Secondary Outcomes (6)

• Pharmacokinetic (PK) Of Participants Who Did Not Concomitantly Receive HU: Maximum Plasma Concentration (Cmax) Of IMR-687

  Day 1 and Week 25

• PK Of Participants Who Did Not Concomitantly Receive HU: Area Under The Concentration-time Curve (AUC) From Time 0 To 24 Hours Postdose (AUC0-24h) Of IMR-687

  Day 1 and Week 25

• PK Of Participants Who Concomitantly Received HU: Cmax Of IMR-687

  Day 1 and Week 17

• PK Of Participants Who Concomitantly Received HU: AUC0-24h Of IMR-687

  Day 1 and Week 17

• PK Of Participants Who Concomitantly Received HU: Cmax Of HU

  Baseline (1 and 2) and Week 17

Other Outcomes (1)

• Change From Baseline In F-Cells

  Baseline, EOT (Week 25 for participants without HU and Weeks 17 or 25 for participants with HU)

Study Arms (3)

IMR-687 50 mg/100 mg

EXPERIMENTAL

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).

Drug: IMR-687

IMR-687 100 mg/200 mg

EXPERIMENTAL

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).

Drug: IMR-687

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

IMR-687 DRUG

Oral administration of IMR-687 once daily with or without HU.

IMR-687 100 mg/200 mg; IMR-687 50 mg/100 mg

Placebo DRUG

Oral administration of placebo once daily with or without HU.

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cardurion Pharmaceuticals, Inc.: lead
• Imara, Inc.: collaborator

Study Sites (13)

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

University of Connecticut Health Center

Farmington, Connecticut, 06030, United States

Location

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33021, United States

Location

University of Illinois

Chicago, Illinois, 60612, United States

Location

Loretto Hospital

Chicago, Illinois, 60644, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Baylor Scott & White Health

Temple, Texas, 76508, United States

Location

Sandwell & West Birmingham Hospital

Birmingham, B18 7QH, United Kingdom

Location

Bristol Haematology and Oncology Centre

Bristol, BS2 8ED, United Kingdom

Location

Royal London Hospital

London, E1 1BB, United Kingdom

Location

University College London Hospital

London, NW1 2PG, United Kingdom

Location

Guy's Hospital

London, SE1 9RT, United Kingdom

Location

Oxford Cancer & Haematology Centre, The Churchill Hospital

Oxford, OX3 7LE, United Kingdom

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Imara Clinical Operations
• Organization: Imara Inc.

info@imaratx.com

617-206-2020

Study Officials

• Regulatory Operations

  Cardurion Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 2, 2018
• First Posted: January 17, 2018
• Study Start: January 26, 2018
• Primary Completion: August 28, 2020
• Study Completion: August 28, 2020
• Last Updated: May 15, 2025
• Results First Posted: April 14, 2022

Record last verified: 2025-05

Locations

US (7); GB (6)