A Study of BBP-711 (ORF-229) in Healthy Adult Volunteers

A Phase 1, Randomized, Double-Blinded, Placebo-controlled, Single and Multiple-Ascending Dose Study of the Safety, Tolerability, Food Effect, Pharmacokinetics, and Pharmacodynamics of BBP-711 (ORF-229) in Healthy Adult Subjects

The purpose of this study is to evaluate the safety and tolerability of BBP-711 in healthy adult volunteers.

Study Overview

• Status: Completed
• Conditions: Healthy
• Intervention / Treatment: Drug: BBP-711; Drug: Placebo

Detailed Description

This is a single-center, two-part, randomized, double-blinded, placebo-controlled, ascending dose study of BBP-711 in healthy male and female adult volunteers. The purpose of this study is to evaluate the safety and tolerability of BBP-711 in healthy adult volunteers. Each volunteer will participate in the study for about 20 days.

• Study Type: Interventional
• Enrollment (Actual): 92
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tempe, Arizona, United States, 85283
      • Celerion

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Adult male or female who is 18 to 65 years old,
• Weight >50 kg and ≤110 kg at Screening
• Body mass index (BMI) 20 to 32 kg/m2, inclusive, at Screening
• In generally good health
• Nonsmoker, or not using tobacco or nicotine-containing products for at least 6 months

Exclusion Criteria:

• Use of any over-the-counter medications, including herbals or routine vitamins or minerals, or other supplements, within 7 days before admission to the research center.
• Pregnant or breastfeeding
• eGFR <90 mL/minute
• Abnormal ECG
• Abnormal laboratory results
• Positive test result for HIV, Hepatitis B, Hepatitis C, or COVID-19
• History of substance dependency (alcohol or other drugs of abuse) in the last 2 years
• Use of study drug in any clinical trial within 30 days of admission to the research center, or in the active follow-up phase of another clinical trial involving study drug

Study Plan

How is the study designed?

Design Details

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

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BBP-711 for SAD; A single dose of BBP-711 will be administered orally.	Drug: BBP-711; BBP-711, oral suspension
Placebo Comparator: Placebo for SAD; A single dose of matching placebo will be administered orally.	Drug: Placebo; Placebo matching BBP-711
Experimental: BBP-711 for MAD; A dose of BBP-711 will be administered orally for multiple days.	Drug: BBP-711; BBP-711, oral suspension
Placebo Comparator: Placebo for MAD; A dose of matching placebo will be administered orally for multiple days.	Drug: Placebo; Placebo matching BBP-711
Experimental: BBP-711 for SAD Food Effect; A single dose of BBP-711 will be administered orally.	Drug: BBP-711; BBP-711, oral suspension
Placebo Comparator: Placebo for SAD Food Effect; A single dose of matching placebo will be administered orally.	Drug: Placebo; Placebo matching BBP-711

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and Tolerability	Incidence of Adverse Events (AEs)	Baseline to Day 20

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetic Assessments: Cmax	Maximum observed plasma concentration (Cmax)	Blood samples will be taken pre-dose up to Day 10 for SAD and pre-dose up to Day 20 for MAD
Pharmacokinetic Assessments: Cmin	Minimum observed plasma concentration (Cmin)	Blood samples will be taken pre-dose up to Day 10 for SAD and pre-dose up to Day 20 for MAD
Pharmacokinetic Assessments: AUC	Area under the plasma concentration-time curve from 0 to last measurable concentration (AUC(0-last)) computed using the linear trapezoidal rule	Blood samples will be taken pre-dose up to Day 10 for SAD and pre-dose up to Day 20 for MAD
Pharmacodynamic Assessment: Baseline plasma glycolate	Baseline plasma glycolate	Baseline
Pharmacodynamic Assessment: Percentage change from baseline plasma glycolate	Percentage change from baseline plasma glycolate	Blood samples will be taken pre-dose up to Day 10 for SAD and pre-dose up to Day 20 for MAD
Pharmacodynamic Assessment: Baseline 24 Hour urinary glycolate:creatinine ratio	Baseline 24 Hour urinary glycolate:creatinine ratio	Baseline
Pharmacodynamic Assessment: Percentage change from baseline of 24 Hour urinary glycolate:creatinine ratio	Percentage change from baseline of 24 Hour urinary glycolate:creatinine ratio	Urine samples will be taken pre-dose up to Day 3 for SAD and pre-dose up to Day 7 for MAD

Collaborators and Investigators

• Sponsor: Cantero Therapeutics, a BridgeBio company
• Collaborators: Celerion

Study record dates

Study Major Dates

• Study Start (Actual): April 29, 2021
• Primary Completion (Actual): February 27, 2022
• Study Completion (Actual): February 27, 2022

Study Registration Dates

• First Submitted: May 3, 2021
• First Submitted That Met QC Criteria: May 3, 2021
• First Posted (Actual): May 7, 2021

Study Record Updates

• Last Update Posted (Actual): March 21, 2022
• Last Update Submitted That Met QC Criteria: March 18, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Other Study ID Numbers: ORF-01-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

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