Sickle Beta 0 Thalassemia, Sickle Cell Disease, Sickle-Cell; Hb-SC
Conditions
Keywords
Sickle Cell Disease, Sickle Beta 0 Thalassemia
Brief summary
The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects.
Interventions
DRUGIMR-687
1 of 6 possible single doses administered orally following overnight fast
DRUGPlacebo Oral Capsule
Placebo oral capsule with 50 mg microcrystalline cellulose in capsules identical to those used for the active pharmaceutical ingredient.
Sponsors
Quintiles, Inc.
Imara, Inc.
Cardurion Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
* Be healthy as judged by the Investigator on the basis of pre-study tests performed at Screening, with healthy body mass index (BMI), healthy body weight, and laboratory results within normal laboratory reference range or determined not to be clinically significant by the Investigator; and be free from drugs of abuse.
Exclusion criteria
* Females who are pregnant, trying to become pregnant, or breastfeeding; and males with female partners who are trying to conceive. * Asthmatics or other individuals who use or may use albuterol rescue inhalers or nebulizers. * A significant history of cardiovascular disease. * On ECG, a QTcF \>450 ms or the presence of clinically significant abnormalities as determined by the Investigator. * Elevated blood pressure. * Use within 30 days prior to Day 1 of any inhibitors or substrates of targets of IMR-687.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Use of concomitant medications and therapies, medication type and frequency | 5 Days | — |
| Number of participants with clinically significant changes from baseline in hematology, chemistry, coagulation and urinalysis laboratory values | Baseline to Day 5 | — |
| Number of participants with clinically significant changes from baseline in 12-lead ECG parameters | Baseline to Day 2 | — |
| Number of participants with treatment emergent adverse events and serious adverse events | 5 Days | — |
| Number of participants with clinically significant changes from baseline in vital signs | Baseline to Day 5 | Vital signs include blood pressure, heart rate, pulse rate, and oral temperature |
| Number of participants with clinically significant changes from baseline in physical examination | Baseline to Day 5 | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The change from baseline in QTcF interval. | 2 Days | — |
| Pharmacokinetics (PK) of IMR-687 | Day 1 prior to administration of drug and 0.25, 0.5, 0.75, 1, 1.5, 2, 4, 6, 8, 12, 24 hours post dose | Maximum Observed Plasma Concentration (Cmax) of IMR-687 |
Countries
United States
Outcome results
None listed