A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia

• Status: Recruiting
• Trial end date: 2022-07-28
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: CoA Therapeutics Inc.; CoA Therapeutics, Inc., a BridgeBio company

Criteria

Inclusion Criteria (Healthy Volunteers):

• Subject is male or female 18 to 55 yrs old

• Subject has a BMI 18 to 32 kg/m^2

• Female and male subjects must use effective method of birth control

• Female subjects must have negative pregnancy test prior to first dose of study drug

• Subject must not have any clinically significant eye abnormality

• Subject must not have any clinically significant history or presence of ECG findings

• Subject must be in good general health

Inclusion Criteria (PA or MMA Patients):

• Patient is male or female 18 to 55 yrs old

• Patient has a BMI 18 to 32 kg/m^2

• Female and male patients must use effective method of birth control

• Female patients must have negative pregnancy test prior to first dose of study drug

• Patient must not have any clinically significant eye abnormality

• Patient must not have any clinically significant history or presence of ECG findings

• Patient must have confirmed PA or MMA diagnosis

• Patient with MMA must have elevated plasma MMA levels

• Patient is willing to provide access to medical records for the last 6-12 months of care prior to study initiation

• Patient is on consistent disease management and treatment regimen is stable for at least 30 days prior to study initiation.

Exclusion Criteria (Healthy Volunteers):

• Subject has used prescription drugs within 4 weeks before first dose of study drug or over-the-counter medication within 7 days of the first dose of study drug

• Subject who has taken the COVID-19 vaccine, the last vaccine dose must be at least 14 days prior to first dose of study drug.

• Subject has abnormal laboratory test results

• Subject has a baseline eGFR <90 mL/minute

• Subject has positive result for Hepatitis B, Hepatitis C, or HIV

• Female subject is non-pregnant and non-lactating

• Subject is a smoker or has used nicotine or nicotine-containing products

• Subject has a history of alcohol or drug abuse within 12 months prior to first dose of study drug and/or has a positive result prior to dosing or throughout the study

• Subject has donated blood or blood products >450mL within 30 days prior to study drug dosing

• Subject has a history of relevant drug or food allergies

• Subject has received study drug in another investigational study within 30 days of dosing

Exclusion Criteria (PA or MMA Patients):

• Patient has used prescription drugs within 4 weeks before first dose of study drug or over-the-counter medication within 7 days of the first dose of study drug that is not part of their PA or MMA disease management and treatment

• Patient who has taken the COVID-19 vaccine, the last vaccine dose must be at least 14 days prior to first dose of study drug.

• Patient has clinically significant abnormal laboratory test results unrelated to PA or MMA

• Patient has a baseline eGFR <60 mL/minute

• Patient has positive result for Hepatitis B, Hepatitis C, or HIV

• Female patient is non-pregnant and non-lactating

• Patient is a smoker or has used nicotine or nicotine-containing products

• Patient has a history of alcohol or drug abuse within 12 months prior to first dose of study drug and/or has a positive result prior to dosing or throughout the study

• Patient has donated blood or blood products >450mL within 30 days prior to study drug dosing

• Patient has a history of relevant drug or food allergies

• Patient has received study drug in another investigational study within 30 days of dosing

• Patient with PA has undergone prior liver and/or kidney transplant