Study of BLU-808 in Healthy Adult Participants

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BLU-808 and to Assess the Drug-Drug Interaction With a CYP3A Substrate (Midazolam) and the Effect of Food in Healthy Adult Subjects

This is a 3-part study. Parts 1 and 2 are randomized, double-blind, placebo-controlled investigations of single ascending doses (SAD) (Part 1) and multiple ascending doses (MAD) (Part 2) of orally administered BLU-808 in healthy adult participants. Part 2 will also include an evaluation of the effect of multiple doses of BLU-808 on the single-dose pharmacokinetics (PK) of midazolam. Part 3 is an open-label, 2-sequence, 2-period, food effect (FE) study in healthy adult participants.

Study Overview

• Status: Completed
• Conditions: Healthy Participants
• Intervention / Treatment: Drug: BLU-808; Drug: Placebo; Drug: Midazolam

• Study Type: Interventional
• Enrollment (Actual): 95
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Nebraska
    • Lincoln, Nebraska, United States, 68502
      • Celerion, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Key Inclusion Criteria:

• Continuous non-smoker who has not used nicotine- and tobacco-containing products for at least 3 months prior to the first dosing based on subject self-reporting.
• Medically healthy with no clinically significant medical history, physical examination, clinical laboratory profiles, or vital signs, as deemed by the Principal Investigator (PI) or designee.
• No clinically significant cardiac history as judged by the PI or designee at the screening visit and check-in.

Key Exclusion Criteria:

• Participant is mentally or legally incapacitated or has significant emotional problems at the time of the screening visit or expected during the conduct of the study.
• Participant has history or presence of clinically significant medical or psychiatric condition or disease in the opinion of the PI, designee, or Sponsor.
• Participant has history or presence of alcohol or drug abuse within the past 2 years prior to the first dosing.
• Participant has positive results at the screening visit for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV).
• Participant has been on a diet incompatible with the on-study diet (that is, unusual meal habits and special diet requirements or unwillingness to eat the food provided in the trial), in the opinion of the PI or designee, within the 30 days prior to the first dosing and throughout the study.
• Participant has participated in another clinical study within 30 days prior to the first dosing. The 30-day window will be derived from the date of the last dosing in the previous study to the first dosing of the current study.
• Participant has positive coronavirus disease 2019 (COVID-19) results at first check-in.

Note: Other protocol-specified inclusion and exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

8

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part 1: BLU-808; BLU-808 will be administered orally.	Drug: BLU-808; BLU-808 will be administered per schedule specified in the arm description.
Placebo Comparator: Part 1: Placebo; Matching placebo for BLU-808 will be administered orally.	Drug: Placebo; BLU-808 matching placebo will be administered per schedule specified in the arm description.
Experimental: Part 2: BLU-808; BLU-808 will be administered orally.	Drug: BLU-808; BLU-808 will be administered per schedule specified in the arm description.
Placebo Comparator: Part 2: Placebo; Matching placebo for BLU-808 will be administered orally.	Drug: Placebo; BLU-808 matching placebo will be administered per schedule specified in the arm description.
Experimental: Part 2: Midazolam Alone Followed by BLU-808 and Midazolam; Participants will receive oral midazolam in Period 1. Participants will then receive oral BLU-808 with midazolam in period 2.	Drug: BLU-808; BLU-808 will be administered per schedule specified in the arm description.; Drug: Midazolam; Midazolam will be administered per schedule specified in the arm description.
Placebo Comparator: Part 2: Midazolam Alone Followed by BLU-808 Matching Placebo and Midazolam; Participants will receive oral midazolam in Period 1. Participants will then receive oral matching placebo for BLU-808 with midazolam in period 2.	Drug: Placebo; BLU-808 matching placebo will be administered per schedule specified in the arm description.; Drug: Midazolam; Midazolam will be administered per schedule specified in the arm description.
Experimental: Part 3: BLU-808 (Treatment D, E); BLU-808 will be administered orally.	Drug: BLU-808; BLU-808 will be administered per schedule specified in the arm description.
Experimental: Part 3: BLU-808 (Treatment E, D); BLU-808 will be administered orally.	Drug: BLU-808; BLU-808 will be administered per schedule specified in the arm description.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Part 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Up to Day 20
Part 2: Number of Participants With TEAEs	Up to Day 50
Part 3: Area Under the Plasma Concentration-time Curve From Time 0 to the Last Measurable Non-zero Concentration (AUC0-t)	Up to Day 6
Part 3: Maximum Observed Plasma Concentration (Cmax)	Up to Day 6

Secondary Outcome Measures

Outcome Measure	Time Frame
Part 1: AUC0-t	Up to Day 6
Part 1: Cmax	Up to Day 6
Part 1: Terminal Elimination Half-life (t1/2)	Up to Day 6
Part 1: Change From Baseline in QTc (Corrected Value of the Interval Between the Q and T Waves on the Electrocardiogram Tracing)	Up to Day 6
Parts 1 and 2: Change From Baseline in Serum Tryptase Concentrations	Up to Day 6
Part 2: AUC0-t	Up to Day 14
Part 2: Cmax	Up to Day 14
Part 2: t1/2	Up to Day 19
Part 3: Number of Participants With TEAEs	Up to Day 26

Collaborators and Investigators

• Sponsor: Blueprint Medicines Corporation

Study record dates

Study Major Dates

• Study Start (Actual): August 7, 2024
• Primary Completion (Actual): January 21, 2025
• Study Completion (Actual): January 21, 2025

Study Registration Dates

• First Submitted: April 22, 2025
• First Submitted That Met QC Criteria: April 22, 2025
• First Posted (Actual): April 28, 2025

Study Record Updates

• Last Update Posted (Actual): April 28, 2025
• Last Update Submitted That Met QC Criteria: April 22, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anesthetics; Central Nervous System Depressants; Neurotransmitter Agents; Adjuvants, Anesthesia; Hypnotics and Sedatives; Anti-Anxiety Agents; Tranquilizing Agents; Psychotropic Drugs; Anesthetics, Intravenous; Anesthetics, General; GABA Modulators; GABA Agents; Midazolam
• Other Study ID Numbers: BLU-808-0101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No