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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04053803
Recruitment Status : Enrolling by invitation
First Posted : August 13, 2019
Last Update Posted : August 13, 2019
Sponsor:
Information provided by (Responsible Party):
Imara, Inc.

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: IMR-687 Phase 2

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. Patients in this study will not be stratified on the basis of prior baseline hydroxyurea use.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 22, 2019
Estimated Primary Completion Date : June 2024
Estimated Study Completion Date : June 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arm Intervention/treatment
Experimental: Open Label Drug: IMR-687
Oral administration of once daily IMR-687




Primary Outcome Measures :
  1. 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

  2. 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.

  3. 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

  4. 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


Secondary Outcome Measures :
  1. 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.

  2. 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

  3. Change in F cells measured as a percent (% [ Time Frame: Baseline to Month 48 ]
    a. Change in F cells measured as a percent (%)

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Must have been previously enrolled in the IMR-687 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)", i.e., Study IMR-SCD-102.
  2. Female subjects must not be pregnant and be highly unlike to become pregnant. Male subjects must be unlikely to impregnate their 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 at screening >12.5 g/dL or <6 g/dL
  2. Subjects who test positive for human immunodeficiency virus (HIV), hepatitis C (HCV) antibodies (unless the patient has successfully completed drug therapy that results in cure/clearance of HCV), and hepatitis B surface antigen (HBsAg)
  3. Female subjects who test positive for a serum pregnancy test at screening or a urine pregnancy test at Day 1
  4. eGFR <50 mL/min
  5. AST/ALT > 3x the upper limit of normal

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04053803


Locations
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United States, Florida
Foundation for Sickle Cell Disease Research
Hollywood, Florida, United States, 33021
Sponsors and Collaborators
Imara, Inc.
Investigators
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Principal Investigator: Timothy Mant, MB FFPM FRCP Guy's and St Thomas Hospital CRF

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Responsible Party: Imara, Inc.
ClinicalTrials.gov Identifier: NCT04053803    
Other Study ID Numbers: IMR-SCD-102-EXT
First Posted: August 13, 2019    Key Record Dates
Last Update Posted: August 13, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn