What side effects are associated with this type of intervention?

Common treatments for Organic Acidemia, such as dietary management, carnitine supplementation, and antibiotics, have various side effects. Dietary management can result in nutritional deficiencies if not properly monitored. Carnitine supplementation may cause gastrointestinal issues like diarrhea and abdominal cramps. Antibiotics can lead to antibiotic resistance and gastrointestinal disturbances. Liver transplantation, another treatment option, involves significant surgical risks and lifelong immunosuppression, which increases the risk of infections and potential organ rejection.

What prior FDA approvals exist?

There is limited information on specific FDA-approved drugs for the treatment of Propionic Acidemia and Methylmalonic Acidemia. However, treatments generally focus on managing symptoms and preventing metabolic crises through dietary management, carnitine supplementation, and antibiotics to reduce propionate production. BBP-671 is a potential therapeutic agent currently under investigation, aiming to address the underlying metabolic dysfunction in these conditions.

What other types of research exist?

Promising novel alternatives to BBP-671 for Organic Acidemia patients include: <ol> <li>Oral biotin, which has shown dramatic clinical improvement in patients with biotin-responsive conditions.</li> <li></li> </ol> Lipoic acid, which has demonstrated significant improvement in metabolic conditions like lipoamide dehydrogenase deficiency. 3. Octanoic acid, which has been studied for its efficacy in essential tremor and may have potential metabolic benefits. These alternatives should be discussed with a healthcare provider to determine their suitability for individual patients.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 49 days for reporting.

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 more

Secondary study objectives

Food Effect: AUC

Food Effect: Cmax

Food Effect: Tmax

+1 more

Trial 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).

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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.