A Phase 1 Study of BLKR201 in Healthy Adult Participants

NCT07501039 | Recruiting Phase 1 FDA Drug

• 1 other identifier: BLKR201-101-HV
• Study Type: interventional
• Target: 128
• Locations: 1 country
• Sites: 1

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.

Conditions

Healthy

Keywords

Healthy; Healthy Volunteers

Outcome Measures

Primary Outcomes (8)

• Number of Participants with Treatment-Emergent Adverse Events (TEAEs)

  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.

  Baseline through approximately 7 days after final dose

• Change From Baseline in Clinical Laboratory Parameters

  Clinical laboratory parameters include hematology, clinical chemistry, coagulation, lipid panel, and urinalysis. Change from baseline values will be summarized at scheduled post-dose assessments.

  Baseline through approximately 7 days after final dose

• Change From Baseline in Systolic Blood Pressure

  Systolic blood pressure will be measured in millimeters of mercury (mmHg). Change from baseline will be summarized at scheduled post-dose assessments.

  Baseline through approximately 7 days after final dose

• Change From Baseline in Diastolic Blood Pressure

  Diastolic blood pressure will be measured in millimeters of mercury (mmHg). Change from baseline will be summarized at scheduled post-dose assessments.

  Baseline through approximately 7 days after final dose

• Change From Baseline in Pulse Rate

  Pulse rate will be measured in beats per minute (bpm). Change from baseline will be summarized at scheduled post-dose assessments.

  Baseline through approximately 7 days after final dose

• Change From Baseline in Respiratory Rate

  Respiratory rate will be measured in breaths per minute. Change from baseline will be summarized at scheduled post-dose assessments.

  Baseline through approximately 7 days after final dose

• Change From Baseline in Body Temperature

  Body temperature will be measured in degrees Fahrenheit (°F). Change from baseline will be summarized at scheduled post-dose assessments.

  Baseline through approximately 7 days after final dose

• Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters

  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.

  Baseline through approximately 7 days after final dose

Secondary Outcomes (8)

• Maximum Observed Plasma Concentration (Cmax) of BLKR201 After Single Oral Dose

  From predose through approximately 7 days after final dose

• Area Under the Plasma Concentration-Time Curve (AUC) of BLKR201 After Single Oral Dose

  From predose through approximately 7 days after final dose

• Maximum Observed Plasma Concentration (Cmax) After Multiple Doses

  From predose through approximately 7 days after final dose

• Area Under the Plasma Concentration-Time Curve Over the Dosing Interval (AUCtau) After Multiple Doses

  From predose through approximately 7 days after final dose

• BLKR201 Concentration in Cerebrospinal Fluid (CSF)

  Day 7 post-dose in the CSF cohort (single CSF sampling time point per participant)

Study Arms (3)

Single Ascending Dose (SAD) Cohorts (BLKR201 or Placebo)

EXPERIMENTAL

Participants receive a single oral dose of BLKR201 or matching placebo in sequential dose-escalation cohorts (up to six escalating doses of BLKR201 to be evaluated) under fasted conditions. One cohort will receive a second dose under fed conditions. Participants are randomized within each cohort.

Drug: BLKR201; Drug: Placebo

Multiple Ascending Dose (MAD) Cohorts (BLKR201 or Placebo)

EXPERIMENTAL

Participants receive BLKR201 or matching placebo orally once daily for 7 consecutive days in sequential dose-escalation cohorts (up to four escalating doses of BLKR201 to be evaluated). A twice-daily (BID) cohort may be evaluated based on emerging safety and PK data. Participants are randomized within each cohort.

Drug: BLKR201; Drug: Placebo

Cerebrospinal Fluid (CSF) Cohort (BLKR201, Open-Label)

EXPERIMENTAL

Participants receive BLKR201 orally for 7 consecutive days (planned dose: 200 mg once daily). This cohort is non-randomized and open-label. Cerebrospinal fluid samples are collected to evaluate central nervous system exposure.

Drug: BLKR201

Interventions

BLKR201 DRUG

BLKR201 is administered orally as a single ascending dose in the SAD stage. In the food effect cohort, BLKR201 is administered under both fasted and fed conditions in separate periods.

Single Ascending Dose (SAD) Cohorts (BLKR201 or Placebo)

Placebo DRUG

A matching placebo tablet administered orally, corresponding to the dose level and dosing condition of BLKR201 in each assigned cohort.

Multiple Ascending Dose (MAD) Cohorts (BLKR201 or Placebo); Single Ascending Dose (SAD) Cohorts (BLKR201 or Placebo)

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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
• Regular heavy alcohol use

Sponsors & Collaborators

• Formation Bio, Inc.: lead

Study Sites (1)

Celerion

Lincoln, Nebraska, 68502, United States

RECRUITING

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Open-label for CSF cohort only
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2026
• First Posted: March 30, 2026
• Study Start: April 27, 2026
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)