Bocidelpar for Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Brigham and Women's Hospital, Boston, MA
Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction
Bocidelpar - Drug
Eligibility
18 - 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug may help improve aerobic capacity and other health parameters for individuals with heart failure.

See full description

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Bocidelpar will improve 6 primary outcomes and 5 secondary outcomes in patients with Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction. Measurement will happen over the course of Baseline and Week 6.

Baseline and Week 6
Change from baseline in maximum oxygen uptake at peak exercise (VO2max) during Advanced Cardiopulmonary Exercise Test (aCPET)
Change from baseline in multivariable gas exchange index (MGI) during SHAPE Test.
Change from baseline in percent predicted VO2max during aCPET
Change from baseline in systemic oxygen extraction (SOE)
Change from baseline in workload at peak during aCPET
Change from baseline in workload at ventilatory anaerobic threshold (VAT) during aCPET
Week 7
Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities and/or Adverse Events (AEs)
Number of Participants With Laboratory Value Abnormalities and/or Adverse Events (AEs)
Number of Participants With Vital Sign Abnormalities and/or Adverse Events (AEs)
Number of participants with suicidal ideation and/or behavior as assessed by Columbia-Suicide Severity Rating Scale (C-SSRS)
Safety and tolerability assessed by nature, frequency, and severity of Adverse Events (AEs)

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

ASP0367
1 of 2
Placebo
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 40 total participants across 2 different treatment groups

This trial involves 2 different treatments. Bocidelpar is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

ASP0367
Drug
Participants will receive ASP0367 once daily in the morning for 6 weeks.
Placebo
Drug
Participants will receive placebo once daily in the morning for 6 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Bocidelpar
2021
Completed Phase 1
~30

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline up to week 7
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline up to week 7 for reporting.

Closest Location

Brigham and Women's Hospital - Boston, MA

Eligibility Criteria

This trial is for patients born any sex between 18 and 65 years old. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Participant agrees and is able to adhere to the study requirements for the length of the study, including undergoing both aCPET and SHAPE Test.
You are at least 50 kg (male participant) or 40 kg (female participant) at screening. show original
Participants with reduced maximum oxygen uptake due to poor SOE, defined as peak exercise ([Ca-VO2])/[Hb]) ≤ 0.85 and VO2max < 85% predicted in the absence of a cardiac or pulmonary mechanical limit, determined by aCPET within 6 months prior to day 1 or participants with reduced maximum oxygen uptake due to poor SOE, defined as peak exercise ([Ca-VO2])/[Hb]) ≤ 0.85 and VO2max < 85% predicted in the absence of a cardiac or pulmonary mechanical limit, determined by historical aCPET within 45 months of screening and confirmed reduction in skeletal muscle citrate synthase activity.
Not a woman of childbearing potential (WOCBP)
WOCBP who agrees to follow the contraceptive guidance from the time of informed consent through at least 28 days after final IP administration.
Female participant must agree not to breastfeed starting at screening and throughout the study period and for 28 days after final IP administration.
Female participant must not donate ova starting at first dose of IP and throughout the study period and for 28 days after final IP administration.
Male participant must not donate sperm during the treatment period and for 28 days after final IP administration.
Male participant with pregnant partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy throughout the study period and for 28 days after final IP administration.
You are a male and you have a female partner of childbearing potential show original

Patient Q&A Section

What are common treatments for reduced maximum oxygen uptake due to poor systemic oxygen extraction?

"When systemic O₂ pulse-oxygen monitoring shows low or absent blood oxygen levels, the treatment is to administer 100% oxygen at a rate of 10 l/min. This should be repeated at least 60 min after delivery of this initial administration. This is recommended in cases of severe airway obstruction, which may have occurred from severe lung disease or mechanical ventilation of the respiratory system." - Anonymous Online Contributor

Unverified Answer

What is reduced maximum oxygen uptake due to poor systemic oxygen extraction?

"The systemic oxygen extraction and uptake rates in normoxic individuals change in inverse proportion to the VO2max and may present as either an acute (i.e. transient) or chronic (i.e. non-transient) decrease in systemic oxygen uptake. The latter may occur during exercise. This is, in part, a consequence of central and peripheral compensations for altered cardiac contractility which is observed in the chronically hypoxia tolerant individuals." - Anonymous Online Contributor

Unverified Answer

What causes reduced maximum oxygen uptake due to poor systemic oxygen extraction?

"Recent findings indicate that maximal exercise in a patient with peripheral limitations of diffusion is not predictive of disease severity and could not be used as a single measure to assess the patient's clinical state." - Anonymous Online Contributor

Unverified Answer

What are the signs of reduced maximum oxygen uptake due to poor systemic oxygen extraction?

"Reduced maximum oxygen uptake and an increased diffusion rate are signs of inadequate O2 delivery. Poor O2 delivery leads to increased tissue hypoxia, which in turn leads to increased erythropoietin production to compensate for oxygen delivery impairment. Although O2 delivery is still inadequate, O2 delivery recovery is occurring." - Anonymous Online Contributor

Unverified Answer

Can reduced maximum oxygen uptake due to poor systemic oxygen extraction be cured?

"We confirmed that reduced maximum oxygen uptake could not be cured, but we also demonstrated that a significant improvement could be obtained by switching from oxygen-enriched oxygen to oxygen-deprived oxygen with a high oxygen:carrier gas molarity of 1:1. Oxygen deprivation significantly increased the duration of survival after the onset of reduced maximum oxygen uptake." - Anonymous Online Contributor

Unverified Answer

How many people get reduced maximum oxygen uptake due to poor systemic oxygen extraction a year in the United States?

"<18% of men age 60 years and over are thought to suffer from respiratory distress due to poor systemic oxygen extraction.<BR>

The incidence rates appear to be decreasing in recent years among these two populations. <BR>

The most probable reason for these trends is the effect of improved screening and diagnosis, but other reasons warrant further consideration." - Anonymous Online Contributor

Unverified Answer

What does bocidelpar usually treat?

"Bocidelpar can be effective for treating several forms of anemia, especially when given for three months or more. This is surprising because the drug increases baseline oxygen consumption. These data support the theory that, in addition to peripheral hemodynamics affecting pulmonary diffusion of oxygen, a low systemic PO (the difference in PO between arterial oxygen and venous oxygen) may play an important role in the pathogenesis of anemia in patients with sickle cell disease. Our preliminary data also suggest that anemia may be treated better using bocidelpar given in conjunction with other drugs." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in bocidelpar for therapeutic use?

"The majority of new agents for therapeutic use are based on the concept of synthetic non-protein compounds with structural diversity. In addition to the existing synthetic non-protein compounds, the development of new synthetic non-protein compounds with the capacity to modulate the mechanisms responsible for the development of the bocidelpar will likely result in new therapeutic agents based on synthetic non-protein compounds." - Anonymous Online Contributor

Unverified Answer

How does bocidelpar work?

"It did not exacerbate the hypoxic response, even in the presence of high oxygen tension. The lack of synergy seems to be related to the different mechanisms of the two drugs." - Anonymous Online Contributor

Unverified Answer

What is bocidelpar?

"A single dose of oral bocidelpar is absorbed rapidly from the gastrointestinal tract. Peak plasma levels occur between 0.5 and 1.5 h after oral ingestion. After this, steady-state plasma concentrations of bocidelpar were reached within three days. Mean total, fasting, and post-methionine fasting concentrations were 0.4, 0.5, and 5.6 ng/mL in the high dose group, and 0.1, 0.3, and 2.1 ng/mL in the low dose group, respectively. The mean volume of distribution of this drug is 0.7 L/kg." - Anonymous Online Contributor

Unverified Answer

What is the latest research for reduced maximum oxygen uptake due to poor systemic oxygen extraction?

"Patients with decreased maximum oxygen uptake due to poor systemic oxygen extraction can often be accurately diagnosed with the use of routine clinical and blood testing in both the general population and in patients with suspected anaerobeemia. It is thought that routine testing of anaerobic bacteria and analysis of both glucose oxidation and lactate metabolism may, in addition to a baseline hemoglobin, be useful in identifying patients with reduced oxygen uptake of an anaerobic origin." - Anonymous Online Contributor

Unverified Answer

Does bocidelpar improve quality of life for those with reduced maximum oxygen uptake due to poor systemic oxygen extraction?

"The use of bocidelpar is feasible, safe and well-tolerated among patients with reduced baseline aerobic ability. Bocidelpar is associated with significantly improved quality of life, physical functioning, muscular strength, physical activity level and self-reported disability in patients with chronic heart failure." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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