Status:
TERMINATED
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
Lead Sponsor:
CoA Therapeutics, Inc., a BridgeBio company
Conditions:
Healthy Volunteers
Propionic Acidemia
Eligibility:
All Genders
15-55 years
Phase:
PHASE1
Brief 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.
Detailed Description
This is the first-in-human study with BBP-671 and is designed to provide healthy subjects single- and multiple-dose and patient multidose safety, tolerability, PK, and PD data regarding BBP-671 for fu...
Eligibility Criteria
Inclusion
- 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 history or presence of ECG findings
- Subject must be in good general health
- Inclusion Criteria (PA or MMA Patients):
- Patient is male or female 15 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 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 (contraceptive medications are allowed) 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 is unable or unwilling to refrain from wearing contact lenses during participation in the study.
- Subject has a history of dry eye or eye surgery, including radial keratotomy and LASIK surgery.
- 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
- Subject has undergone prior liver and/ or kidney transplant.
- Exclusion Criteria (PA or MMA Patients):
- Patient has used prescription drugs (contraceptive medications are allowed) 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 (or booster) must be at least 14 days prior to first dose of study drug.
- Patient is unable or unwilling to refrain from wearing contact lenses during participation in the study.
- Patient has a history of dry eye or eye surgery, including radial keratotomy and LASIK surgery.
- 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 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
- PA patient has undergone prior liver and/ or kidney transplant. Prior liver and/or kidney transplant is allowed for patients with MMA.
- Patient has had a recent infection requiring system antibiotics within 4 weeks of Baseline or any active infection that precludes the patient from participation
- Patient has Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association.
- Patient has been exposed to gene therapy for PA or MMA at any time prior to study entry.
- Patient is currently taking sensitive CYP3A4 substrates (e.g., tacrolimus or sirolimus)
Exclusion
Key Trial Info
Start Date :
March 25 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 20 2023
Estimated Enrollment :
79 Patients enrolled
Trial Details
Trial ID
NCT04836494
Start Date
March 25 2021
End Date
November 20 2023
Last Update
December 21 2023
Active Locations (3)
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1
Community Health Clinic
Topeka, Indiana, United States, 46571
2
UPMC Children's Hospital of Pittsburg
Pittsburgh, Pennsylvania, United States, 15224
3
PPD Development, LP
Austin, Texas, United States, 78744