Healthy
Conditions
Keywords
Healthy, Healthy Volunteers
Brief summary
The goal of this clinical trial is to learn if BLKR201 is safe in healthy adults. Researchers will also learn how the body absorbs and processes BLKR201 and how food may affect it. The main questions this study aims to answer are: * Is BLKR201 safe and well tolerated when taken as a single dose or for several days in a row? * How does BLKR201 move through and leave the body? * Does taking BLKR201 with food change how the body absorbs it? Researchers will compare BLKR201 to a placebo (a look-alike tablet that contains no drug) in most parts of the study to see how the drug affects participants. Participants will: * Take BLKR201 or a placebo by mouth * Stay at a clinical research unit for several days during dosing * Give blood and urine samples * Have heart tests, vital signs, and lab tests * Report any side effects In one part of the study, a small group of participants will receive BLKR201 only (no placebo). These participants will also have a sample of spinal fluid collected to measure how much BLKR201 reaches the fluid around the brain and spinal cord.
Interventions
DRUGBLKR201
BLKR201 is administered orally as a single ascending dose in the SAD stage, under fasted or fed conditions.
DRUGPlacebo
A matching placebo tablet administered orally, corresponding to the dose level and dosing condition of BLKR201 in each assigned cohort.
Sponsors
Formation Bio, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
OTHER
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Open-label for CSF cohort only
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
* Are a healthy man or woman 18 to 55 years old * Weigh at least 50 kilograms (110 pounds) * Have a body mass index (BMI) between 18 and 30 kg/m2 (inclusive) * Are willing to stay at the research unit for the required study visits * Are willing to follow study rules about diet, alcohol, tobacco, and medications * Are able to understand and sign the informed consent form Women who can become pregnant must: * Have a negative pregnancy test before starting * Use a highly effective form of birth control during the study and for a period after the last dose Men with partners who can become pregnant must: * Use highly effective birth control during the study and for a period after the last dose * Not donate sperm during that time
Exclusion criteria
* Have any significant medical condition involving the heart, lungs, liver, kidneys, stomach, intestines, brain, blood, or immune system * Have abnormal laboratory test results at screening that the study doctor considers important * Have a history of heart rhythm problems or abnormal electrocardiogram (ECG) results * Have high blood pressure at screening * Have had a serious infection within the past 6 months * Test positive for HIV, hepatitis B, hepatitis C, or tuberculosis, or show evidence of hepatitis B infection (positive hepatitis B surface antigen or hepatitis B core antibody). Note: participants with hepatitis B immunity from vaccination alone may still be eligible. * Have a history of cancer in the past 5 years (with limited exceptions for certain treated skin cancers) * Have a history of serious stomach problems such as ulcers, stomach surgery, or stomach bleeding * Use tobacco, nicotine, or marijuana products * Test positive for drugs or alcohol at screening * Have taken another investigational drug within the required time before this study * Have received a live vaccine within 30 days before study start * Are pregnant or breastfeeding * Have a known allergy to the study drug or similar medicines Additional exclusions: * Regular heavy alcohol use * Regular use of certain medications such as anti-inflammatory drugs, steroids, or blood thinners * Donation of more than 500 milliliters (about one pint) of blood within 1 month before screening For the spinal fluid (CSF) portion of the study, you cannot take part if you: * Take blood-thinning medications * Have bleeding or clotting problems * Have sensitivity to lidocaine, band aids, adhesive dressing, or medical tape * Have medical conditions that make a spinal tap unsafe
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters | Baseline through approximately 7 days after final dose | ECG parameters include QT interval corrected using Fridericia's formula (QTcF), PR interval, QRS duration, and heart rate. Change from baseline values will be summarized at scheduled post-dose assessments. |
| Number of Participants with Treatment-Emergent Adverse Events (TEAEs) | Baseline through approximately 7 days after final dose | Treatment-emergent adverse events are defined as adverse events that begin or worsen after administration of BLKR201 or placebo. Events will be summarized by frequency and severity. |
| Change From Baseline in Clinical Laboratory Parameters | Baseline through approximately 7 days after final dose | Clinical laboratory parameters include hematology, clinical chemistry, coagulation, lipid panel, and urinalysis. Change from baseline values will be summarized at scheduled post-dose assessments. |
| Change From Baseline in Systolic Blood Pressure | Baseline through approximately 7 days after final dose | Systolic blood pressure will be measured in millimeters of mercury (mmHg). Change from baseline will be summarized at scheduled post-dose assessments. |
| Change From Baseline in Diastolic Blood Pressure | Baseline through approximately 7 days after final dose | Diastolic blood pressure will be measured in millimeters of mercury (mmHg). Change from baseline will be summarized at scheduled post-dose assessments. |
| Change From Baseline in Pulse Rate | Baseline through approximately 7 days after final dose | Pulse rate will be measured in beats per minute (bpm). Change from baseline will be summarized at scheduled post-dose assessments. |
| Change From Baseline in Respiratory Rate | Baseline through approximately 7 days after final dose | Respiratory rate will be measured in breaths per minute. Change from baseline will be summarized at scheduled post-dose assessments. |
| Change From Baseline in Body Temperature | Baseline through approximately 7 days after final dose | Body temperature will be measured in degrees Fahrenheit (°F). Change from baseline will be summarized at scheduled post-dose assessments. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Ratio of Area Under the Plasma Concentration Versus Time Curve (AUC) of BLKR201 Under Fed Versus Fasted Conditions | From predose through 48 hours post-dose (in certain SAD cohorts only) | The ratio of AUC under fed and fasted conditions will be calculated to evaluate the effect of a high-fat meal on BLKR201 exposure. |
| Area Under the Plasma Concentration-Time Curve Over the Dosing Interval (AUCtau) After Multiple Doses | From predose through approximately 7 days after final dose | AUCtau will be calculated to describe total drug exposure during a dosing interval after repeated dosing. |
| BLKR201 Concentration in Cerebrospinal Fluid (CSF) | Day 10 post-dose in the CSF cohort (single CSF sampling time point per participant) | BLKR201 concentrations will be measured in cerebrospinal fluid to assess central nervous system exposure. |
| Amount of BLKR201 Excreted in Urine | From predose through 48 hours post-dose (in certain SAD cohorts only) | The amount of BLKR201 excreted in urine will be calculated over defined collection intervals. |
| Ratio of Maximum Observed Plasma Concentration (Cmax) of BLKR201 Under Fed Versus Fasted Conditions | From predose through 48 hours post-dose (in certain SAD cohorts only) | The ratio of Cmax under fed and fasted conditions will be calculated to evaluate the effect of a high-fat meal on BLKR201 exposure. |
| Maximum Observed Plasma Concentration (Cmax) After Multiple Doses | From predose through approximately 7 days after final dose | Cmax at steady state will be calculated after repeated dosing. |
| Maximum Observed Plasma Concentration (Cmax) of BLKR201 After Single Oral Dose | From predose through approximately 7 days after final dose | Cmax is the highest measured plasma concentration of BLKR201 following a single oral dose. |
| Area Under the Plasma Concentration-Time Curve (AUC) of BLKR201 After Single Oral Dose | From predose through approximately 7 days after final dose | Area under the plasma concentration-time curve (AUClast and AUCinf) will be calculated using non-compartmental analysis to describe overall drug exposure. |
Countries
United States
Outcome results
None listed