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BBP-671 for Propionic and Methylmalonic Acidemia
Phase 1
Waitlist Available
Research Sponsored by CoA Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 49 days
Summary
This trial tests a new drug, BBP-671, in healthy people and those with specific metabolic disorders to see if it is safe and how it works in the body.
Who is the study for?
This trial is for healthy adults aged 18-55 and patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA) aged 15-55. Participants must have a stable health condition, use birth control, and not be pregnant. Exclusions include recent drug/alcohol abuse, certain vaccine timings, organ transplants, infections requiring antibiotics within the last month, and specific medical histories.
What is being tested?
The study tests BBP-671's safety and effects in comparison to a placebo. It aims to understand how the body processes it (pharmacokinetics/PK) and its impact on PA/MMA (pharmacodynamics/PD). The trial involves dose escalation to find the safest effective amount.
What are the potential side effects?
While side effects are being studied as part of this trial's purpose, they may include typical drug reactions such as digestive discomforts, allergic responses or skin irritations. Specific side effects will be monitored closely due to the novel nature of BBP-671.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 49 days
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~49 days
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Pharmacokinetic Assessments: AUC0-tau
Pharmacokinetic Assessments: CL/F
Pharmacokinetic Assessments: CLr
+4 moreSecondary study objectives
Food Effect: AUC
Food Effect: Cmax
Food Effect: Tmax
+1 moreTrial Design
6Treatment groups
Experimental Treatment
Placebo Group
Group I: BBP-671 for SAD Food EffectExperimental Treatment1 Intervention
Eight (8) healthy male or female adult subjects will be randomized to receive BBP-671.
Group II: BBP-671 for SADExperimental Treatment1 Intervention
The SAD portion of the study will consist of up to 8 cohorts. Six (6) healthy male or female adult subjects will be randomized to receive BBP-671 per cohort (6:2 ratio, BBP-671:placebo).
Group III: BBP-671 for PA and MMA PatientsExperimental Treatment1 Intervention
Up to sixteen (16) patients with either PA or MMA will receive BBP-671.
Group IV: BBP-671 for MADExperimental Treatment1 Intervention
The MAD portion of the study will consist of up to 6 cohorts. Six (6) healthy male or female adult subjects will be randomized to receive BBP-671 per cohort (6:2 ratio, BBP-671:placebo).
Group V: Placebo for MADPlacebo Group1 Intervention
The MAD portion of the study will consist of up to 6 cohorts. Two (2) healthy male or female adult subjects will be randomized to receive matching placebo per cohort (6:2 ratio, BBP-671:placebo).
Group VI: Placebo for SADPlacebo Group1 Intervention
The SAD portion of the study will consist of up to 8 cohorts. Two (2) healthy male or female adult subjects will be randomized to receive matching placebo per cohort (6:2 ratio, BBP-671:placebo).
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Organic Acidemia, such as Propionic Acidemia and Methylmalonic Acidemia, include dietary management, supplementation with specific vitamins and cofactors, and in some cases, liver transplantation. Dietary management involves restricting the intake of certain amino acids that the body cannot properly metabolize.
Supplementation with vitamins like biotin and cobalamin (vitamin B12) can help improve enzyme function that is deficient in these conditions. For instance, biotin is essential for the function of carboxylase enzymes, and cobalamin is crucial for the metabolism of certain fatty acids and amino acids.
These treatments are vital as they help reduce the accumulation of toxic metabolites, thereby preventing metabolic crises and improving overall health outcomes. BBP-671, a potential therapeutic agent, aims to further enhance metabolic stability by targeting specific pathways involved in these disorders, offering a promising addition to existing treatment options.
Biotin-responsive alopecia and developmental regression.Interaction between ethanolamine ammonia-lyase and methylcobalamin. Half-site reactivity with an adenosylcobalamin-dependent enzyme.Lipoamide dehydrogenase deficiency with primary lactic acidosis: favorable response to treatment with oral lipoic acid.
Biotin-responsive alopecia and developmental regression.Interaction between ethanolamine ammonia-lyase and methylcobalamin. Half-site reactivity with an adenosylcobalamin-dependent enzyme.Lipoamide dehydrogenase deficiency with primary lactic acidosis: favorable response to treatment with oral lipoic acid.
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Logistics
Participation is compensated
You will be compensated for participating in this trial.
Who is running the clinical trial?
CoA Therapeutics Inc.Lead Sponsor
CoA Therapeutics, Inc., a BridgeBio companyLead Sponsor
Medical MonitorStudy ChairVP Clinical Development, CoA Therapeutics, Inc., a Bridgebio company
1,678 Previous Clinical Trials
990,216 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- If you are a healthy volunteer, you must have received the COVID-19 vaccine at least 14 days before starting the study drug.Healthy volunteers: Your kidney function is below a certain level.Your kidney function is not strong enough, with an eGFR below 60 mL/minute.If you have methylmalonic acidemia, your plasma MMA levels must be high.Healthy volunteers who have had a liver and/or kidney transplant cannot participate.Healthy volunteers with abnormal lab test results are not eligible.You have a history of alcohol or drug abuse within the past year.Healthy volunteers should not have any important heart-related history or problems found in their ECG test.Only people between the ages of 15 and 55 can participate in the study.You smoke or use nicotine products.You have tested positive for Hepatitis B, Hepatitis C, or HIV.You have had dry eye or eye surgery in the past, including LASIK surgery or radial keratotomy.You have abnormal lab test results not related to your condition.You have severe heart failure.You have received gene therapy for PA or MMA before joining the study.If you have a condition called PA, you cannot have had a liver or kidney transplant before. If you have a condition called MMA, you can have had a liver or kidney transplant before.You are currently taking certain medications that can interact with the study drug.Healthy volunteers should have a body mass index (BMI) between 18 and 32 kg/m^2.Patients with a body mass index (BMI) between 18 and 32 kg/m^2 are eligible.Healthy volunteers should not have had dry eye or eye surgery, including LASIK surgery.Healthy Volunteers: You have tested positive for Hepatitis B, Hepatitis C, or HIV.The study is looking for men and women between 18 and 55 years old who are in good health.You have given a large amount of blood within the last month.You have taken the study drug in another research study within the past 30 days.If you have had problems with alcohol or drug abuse in the past year, you cannot participate.If you have a rare disease called PA or MMA and have been vaccinated for COVID-19, your last dose or booster must have been at least 14 days before starting the study drug.You had a serious infection that needed strong antibiotics in the past 4 weeks or you have a current infection that makes it unsafe for you to join the study.Healthy volunteers: You have donated more than 450mL of blood or blood products in the last 30 days before taking the study drug.You must have a confirmed diagnosis of PA (propionic acidemia) or MMA (methylmalonic acidemia).
Research Study Groups:
This trial has the following groups:- Group 1: Placebo for MAD
- Group 2: BBP-671 for MAD
- Group 3: BBP-671 for SAD Food Effect
- Group 4: BBP-671 for PA and MMA Patients
- Group 5: BBP-671 for SAD
- Group 6: Placebo for SAD
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.