An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia

• ClinicalTrials.gov Identifier: NCT04053803
• Recruitment Status: Active, not recruiting
• First Posted: August 13, 2019
• Last Update Posted: March 4, 2022
• Sponsor: Imara, Inc.
• Information provided by (Responsible Party): Imara, Inc.

Tracking Information

• First Submitted Date: July 18, 2019
• First Posted Date: August 13, 2019
• Last Update Posted Date: March 4, 2022
• Actual Study Start Date: May 22, 2019
• Estimated Primary Completion Date: March 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 29, 2020)

• Proportion of patients with adverse events and serious adverse events [ Time Frame: Baseline to Month 49 ]
  1. Incidence of Adverse Events
  2. Incidence of Serious Adverse Events
• Proportion of patients with changes in safety cardiac parameters [ Time Frame: Baseline to Month 49 ]
  a. Changes in 12-lead ECG parameters that are clinically significant and measured in milliseconds (ms). The parameters are: PR interval, QRS duration, QT interval, ST segment duration and T wave duration.
• Proportion of patients with changes in clinical laboratory tests [ Time Frame: Baseline to Month 49 ]
  a. Clinically significant changes in clinical laboratory tests including serum chemistry, serum hematology and urinalysis
• Proportion of patients with clinically significant abnormal vital signs [ Time Frame: Baseline to Month 49 ]
  1. Blood pressure measured in mmHg
  2. Pulse measured in beats per minute
  3. Respiration rate measured in breaths per minutes
  4. Temperature as measured in degrees F0 or C0

Original Primary Outcome Measures (submitted: August 8, 2019)

• Proportion of patients with adverse events and serious adverse events [ Time Frame: Baseline to Month 49 ]
  1. Incidence of Adverse Events
  2. Incidence of Serious Adverse Events
• Proportion of patients with changes in safety cardiac parameters [ Time Frame: Baseline to Month 49 ]
  1. Changes in 12-lead ECG parameters that are clinically significant and measured in milliseconds (ms). The parameters are: PR interval, QRS duration, QT interval, ST segment duration and T wave duration.
  2. Changes in 12-lead ECG parameters that are clinically significant and interpreted by the investigator as consistent with ischemia or infarction.
• Proportion of patients with changes in clinical laboratory tests [ Time Frame: Baseline to Month 49 ]
  a. Clinically significant changes in clinical laboratory tests including serum chemistry, serum hematology and urinalysis
• Proportion of patients with clinically significant abnormal vital signs [ Time Frame: Baseline to Month 49 ]
  1. Blood pressure measured in mmHg
  2. Pulse measured in beats per minute
  3. Respiration rate measured in breaths per minutes
  4. Temperature as measured in degrees F0 or C0

• Current Secondary Outcome Measures: Not Provided

Original Secondary Outcome Measures (submitted: August 8, 2019)

• Change in total hemoglobin levels (Hb) measured in g/dL [ Time Frame: Baseline to Month 48 ]
  a. Change in total hemoglobin levels (Hb) measured in g/dL.
• Change in total fetal hemoglobin level (HbF) measured as a percent (%) of the total Hb [ Time Frame: Baseline to Month 48 ]
  a. Change in total fetal hemoglobin level (HbF) measured as a percent (%) of the total Hb
• Change in F cells measured as a percent (% [ Time Frame: Baseline to Month 48 ]
  a. Change in F cells measured as a percent (%)
• Change Soluble E-selectin, Soluble P-selectin, and ICAM-1 as measured in ng/ml [ Time Frame: Baseline to Month 48 ]
  a. Change Soluble E-selectin, Soluble P-selectin, and ICAM-1 as measured in ng/ml
• IMR-687 effect on renal function [ Time Frame: Baseline to Month 48 ]
  a. Change in clinically significant renal function as measured by glomerular filtration rate (GFR) as measured in mL/min/1.73sqm
• IMR-687 effect on cardiac function [ Time Frame: Baseline to Month 48 ]
  a. Change in clinically significant cardiac function as measured by transthoracic echocardiogram measurement of i. Left Ventricular Ejection Fraction measured as a percent (%) ii. Tricuspid Valve Jet Velocity as measured in m/s
• IMR-687 clinical outcomes as measured by the ASCQ-Me Questionnaire [ Time Frame: Baseline to Month 49 ]
  1. Change in clinical Measured by the Adult Sickle Cell Quality-of-Life Measurement Information System (ASCQ-Me).
  2. Higher scores (better outcome) on the ASCQ-Me questionnaire means less interference of SCD on the parameters measured
• IMR-687 clinical outcomes as measured by opioid use [ Time Frame: Baseline to Month 49 ]
  a. Change in opioid use as measured by a change in frequency of use documented in electronic data capture system
• IMR-687 clinical outcomes as measured by inpatient hospitalization [ Time Frame: Baseline to Month 49 ]
  a. Change in hospitalization rates as measured by the change in frequency of inpatient hospitalization

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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.

• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Sickle Cell Disease

Intervention

Drug: IMR-687

Oral administration of once daily IMR-687

Study Arms

Experimental: Open Label

Intervention: Drug: IMR-687

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: September 29, 2020): 30
• Original Estimated Enrollment (submitted: August 8, 2019): 70
• Estimated Study Completion Date: March 2025
• Estimated Primary Completion Date: March 2025 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United Kingdom, United States

Administrative Information

• NCT Number: NCT04053803
• Other Study ID Numbers: IMR-SCD-102-EXT; 2018-003805-25 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Imara, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Imara, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Karen Tang, MD; Imara, Inc.

• PRS Account: Imara, Inc.
• Verification Date: March 2022