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

NCT ID: NCT03401112

Last Updated: 2025-05-15

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-01-26

Study Completion Date

2020-08-28

Brief Summary

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

Detailed Description

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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.

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
Double-blind

Study Groups

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IMR-687 50 mg/100 mg

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).

Group Type EXPERIMENTAL

IMR-687

Intervention Type DRUG

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

IMR-687 100 mg/200 mg

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).

Group Type EXPERIMENTAL

IMR-687

Intervention Type DRUG

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

Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Oral administration of placebo once daily with or without HU.

Interventions

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IMR-687

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

Intervention Type DRUG

Placebo

Oral administration of placebo once daily with or without HU.

Intervention Type DRUG

Eligibility Criteria

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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

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Imara, Inc.

INDUSTRY

Sponsor Role collaborator

Cardurion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Regulatory Operations

Role: STUDY_DIRECTOR

Cardurion Pharmaceuticals

Locations

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UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Site Status

University of Connecticut Health Center

Farmington, Connecticut, United States

Site Status

Foundation for Sickle Cell Disease Research

Hollywood, Florida, United States

Site Status

University of Illinois

Chicago, Illinois, United States

Site Status

Loretto Hospital

Chicago, Illinois, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Baylor Scott & White Health

Temple, Texas, United States

Site Status

Sandwell & West Birmingham Hospital

Birmingham, , United Kingdom

Site Status

Bristol Haematology and Oncology Centre

Bristol, , United Kingdom

Site Status

Royal London Hospital

London, , United Kingdom

Site Status

University College London Hospital

London, , United Kingdom

Site Status

Guy's Hospital

London, , United Kingdom

Site Status

Oxford Cancer & Haematology Centre, The Churchill Hospital

Oxford, , United Kingdom

Site Status

Countries

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United States United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2017-000653-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

IMR-SCD-102

Identifier Type: -

Identifier Source: org_study_id

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