Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen ExtractionPhase 1NCT04855201

A Study to Evaluate ASP0367 in Participants With Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction

A Phase 1 study testing Bocidelpar in people with Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction, run by Astellas Pharma Global Development, Inc. at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 18 to 60 years
Conditions the study is for: Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction
What is being tested?: Bocidelpar
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 31 participants
Where: 1 siteUnited States
Study dates: 14 Jun 2021to 16 Dec 2022
Phase: Phase 1
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 60 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Participant agrees and is able to adhere to the study requirements for the length of the study, including undergoing both aCPET and SHAPE Test.
Participant has a body mass index (BMI) range of 17.0 to 36 kg/m^2, inclusive and weighs at least 50 kg (male participant) or 40 kg (female participant) at screening.
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.
Female participant is not pregnant and at least 1 of the following conditions apply:
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.

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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 with female partner(s) of childbearing potential (including breastfeeding partner[s]) must agree to use contraception throughout the treatment 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.
Participant agrees not to participate in another interventional study while participating in the present study.

Who cannot join

Participant has signs and/or symptoms due to a cerebellar, pyramidal, extrapyramidal or other nonmyopathic process (e.g., cerebellar dysfunctions, movement disorder) that would interfere in any nontrivial manner with aCPET or SHAPE Test.
Participant has received any investigational therapy within 28 days or 5 half-lives, whichever is longer, prior to screening.
Participant has any condition which makes the participant unsuitable for study participation.
Participant has used moderate or strong inducers of CYP3A, CYP2C8 and CYP2C19 within the 3 months prior to day -1.
Participant has cardiac troponin I (cTnI) > ULN (or cardiac troponin T > ULN if cTnI is not available) at screening.
Participant has estimated glomerular filtration rate (eGFR) calculated by the modification of diet in renal disease equation < 60 mL/min per 1.73 m^2 at screening.

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Participant has at screening: total bilirubin (TBL) > upper limit of normal (ULN) or transaminase(s) (aspartate aminotransferase [AST] or alanine aminotransferase [ALT]) > ULN in the absence of elevations in creatine kinase.
Participant has diabetes mellitus (types 1 or 2).
Participant has mental conditions such as schizophrenia, bipolar disorder or major depressive disorder that has not been under control within 1 year prior to screening.
Participant has severe behavioral or cognitive problems that preclude participation in the study.
Participant has a history of suicide attempt, suicidal behavior or has any suicidal ideation within 1 year prior to screening that meets criteria at a level of 4 or 5 by using the Columbia-Suicide Severity Rating Scale (C-SSRS) or who is at significant risk to commit suicide.
Participant has undergone an inpatient hospitalization within the 30 days prior to the randomization or has a planned hospitalization or a surgical procedure during the study, which may affect the study assessments.
Participant has clinically significant respiratory disease (such as chronic obstructive pulmonary disease, cystic fibrosis, severe asthma, lung infections including tuberculosis, sarcoidosis, thoracic endometriosis, pulmonary fibrosis and lung cancers) and/or cardiac disease (medical history or current clinical findings) or prior interventional cardiac procedure (e.g., left heart catheterization which resulted in angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to randomization.
Participant has a pacemaker, implantable cardioverter-defibrillator or cardiac resynchronization therapy device, or has a mean corrected QT interval using Fridericia's formula (QTcF) > 450 msec for male participants and > 470 msec for female participants at screening or randomization. If QTcF exceeds these limits, 1 additional triplicate ECG can be taken on the same day in order to determine the participant's eligibility.
Participant has ECG evidence of acute ischemia, atrial fibrillation or active conduction system abnormalities at screening, with the exception of any of the following:
First degree atrioventricular (AV)-block
Second degree AV-block type 1 (Mobitz type 1/Wenckebach type)
Right bundle branch block
Participant has a seizure disorder that may interfere with their ability to complete all study requirements.
Participant has an active malignancy or any other cancer from which the participant has been disease-free for < 5 years.
Participant has a solid organ transplant and is currently receiving treatment with therapy for immunosuppression.
Participant has a positive serology test for human immunodeficiency virus, hepatitis B or hepatitis C infection at screening.
Participant has previously received ASP0367.
Participant has a history of active substance abuse within 1 year prior to randomization.
Participant has smoked, used tobacco-containing products and nicotine or nicotine-containing products (e.g., electronic vapes) within 6 months prior to screening or the participant tests positive for cotinine at screening or on day -1.
Participant has a history of consuming > 14 units for male participants or 7 units for female participants of alcoholic beverages per week within 6 months prior to screening or has a history of alcoholism or drug/chemical/substance abuse within 2 years prior to screening (note: 1 unit = 12 ounces of beer, 4 ounces of wine, 1 ounce of spirits/hard liquor) or the participant tests positive for alcohol at screening or on day -1.
Participant has used any drugs of abuse, that are not prescribed for a medical diagnosis (amphetamines, barbiturates, benzodiazepines, cannabinoids, cocaine and/or opiates) within 3 months prior to day -1 or the participant tests positive for drugs of abuse without a prescription (amphetamines, barbiturates, benzodiazepines, cannabinoids, cocaine and/or opiates) at screening or on day -1.
Participant has used any PPAR ligands such as fibrates and thiazolidinediones (e.g., clinofibrate, clofibrate, fenofibrate, gemfibrozil, pioglitazone, rosiglitazone) within 4 weeks prior to randomization.
Participant has used mestinon within 2 weeks prior to randomization.
Participant has initiated the use of coenzyme Q10 (CoQ10), carnitine, creatine or other mitochondrial-focused supplements within 4 weeks prior to randomization.
Participant has initiated an exercise regimen within 4 weeks prior to randomization.
Participant has a known or suspected hypersensitivity to ASP0367 or any components of the formulation used.
Participant has a positive result for SARS-CoV-2 polymerase chain reaction (PCR) test at screening.

Trial locations and contacts

1 site in 1 country.

United States1 site

Brigham and Women's Hospital
Boston, Massachusetts, 02115

Other trials in United States

About this study

Official title: A Phase 1b Multiple Oral Dose Study to Evaluate the Pharmacological Effect, Safety and Tolerability of ASP0367 in Participants With Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction

The purpose of this study is to to evaluate the effect of ASP0367 on improvement of aerobic capacity relative to placebo, as well as evaluate the safety and tolerability of ASP0367 relative to placebo. This study will also evaluate the effect of ASP0367 on improvement of other aerobic capacity parameters relative to placebo, as well as evaluate the effect of ASP0367 on improvement of functional capacity relative to placebo.

Read the detailed description

The study will consist of a screening period (up to 27 days [approximately 4 weeks]), treatment period (up to 44 days [6 weeks]) and follow-up period (7 days [1 week]).

The study will be completed with an end-of-study visit (ESV). The ESV will take place 7 days (1 week) after the end-of-treatment on day 44 (week 6) or 7 days (1 week) after early discontinuation from the study.

The anticipated total duration of the study for each participant, including screening and follow-up, is approximately 75 days (11 week).

An interim analysis may be performed before the end of the study.

Conditions: Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction
Being tested: Bocidelpar, Placebo
Keywords: Maximum oxygen intake, Peak exercise, Bocidelpar

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BocidelparDrug
Also called MA-0211, ASP0367
Oral
PlaceboDrug
Oral

Study groups in NCT04855201
Study group	Role	What they receive
Bocidelpar (ASP0367)	Experimental	Participants will receive Bocidelpar (ASP0367) once daily in the morning for 6 weeks. Receives: Drug: Bocidelpar
Placebo	Placebo comparator	Participants will receive placebo once daily in the morning for 6 weeks. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change from baseline in maximum oxygen uptake at peak exercise (VO2max) during Advanced Cardiopulmonary Exercise Test (aCPET)
V02max is defined as maximum amount of oxygen body can utilize during exercise.
Measured: Baseline and Week 6
Safety and tolerability assessed by nature, frequency, and severity of Adverse Events (AEs)
Adverse Events (AEs) will be coded using medical dictionary for regulatory activities (MedDRA). An AE is any untoward medical occurrence in a participant, temporally associated with the use of investigational product (IP), whether or not considered related to the IP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IP. This includes events related to the comparator and events related to the (study) procedures.
Measured: Baseline up to week 7
Number of Participants With Laboratory Value Abnormalities and/or Adverse Events (AEs)
Number of participants with potentially clinically significant laboratory values.
Measured: Baseline up to week 7
Number of Participants With Vital Sign Abnormalities and/or Adverse Events (AEs)
Number of participants with potentially clinically significant vital signs values.
Measured: Baseline up to week 7
Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities and/or Adverse Events (AEs)
Number of participants with potentially clinically significant 12-ECG values.
Measured: Baseline up to week 7
Number of participants with suicidal ideation and/or behavior as assessed by Columbia-Suicide Severity Rating Scale (C-SSRS)
The Columbia-Suicide Severity Rating Scale (C-SSRS) is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response provided to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 6 items for suicidal behavior (1. Actual attempt, 2. Interrupted attempt, 3. Aborted attempt, 4. Preparatory acts or behavior, 5. Suicidal Behavior 6. Completed suicide,) will be reported.
Measured: Baseline up to week 7

Secondary outcomes (5)

Change from baseline in systemic oxygen extraction (SOE)
Systemic oxygen extraction is defined as the difference between the arterial and venous concentrations of oxygen.
Measured: Baseline and Week 6
Change from baseline in percent predicted VO2max during aCPET
Predicted V02max is measured by the maximum oxygen uptake at peak exercise.
Measured: Baseline and Week 6
Change from baseline in workload at peak during aCPET
Change from baseline in workload at peak during aCPET
Measured: Baseline and Week 6
Change from baseline in workload at ventilatory anaerobic threshold (VAT) during aCPET
Change from baseline in workload at ventilatory anaerobic threshold (VAT) during aCPET
Measured: Baseline and Week 6
Change from baseline in multivariable gas exchange index (MGI) during SHAPE Test.
Change from baseline in multivariable gas exchange index (MGI) during SHAPE Test. The SHAPE Test is a noninvasive exercise test of the functional status of participants.
Measured: Baseline and Week 6

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

14 Jun 202116 Dec 2022

First posted22 Apr 2021
Study start14 Jun 2021
Primary completion16 Dec 2022
Study completion16 Dec 2022

Why it stopped: Lack of Pharmacological effect

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen ExtractionStudies of the same condition, open and under way.
More trials run by Astellas Pharma Global Development, Inc.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 31 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04855201
Sponsor's protocol ID: 0367-CL-1101
Status last verified: Apr 2025
First submitted: 20 Apr 2021
Last updated: 16 Apr 2025
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No: Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Study at a glance

From the registry record, last updated by the sponsor on 16 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1b Multiple Oral Dose Study to Evaluate the Pharmacological Effect, Safety and Tolerability of ASP0367 in Participants With Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction
ClinicalTrials.gov ID: NCT04855201
Acronym / sponsor ID: 0367-CL-1101
Conditions: Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction
Keywords: Maximum oxygen intake, Peak exercise, Bocidelpar
Phase: Phase 1
Study type: Interventional
Recruitment status: TerminatedReason stopped: Lack of Pharmacological effect
Enrolment: 31 participants (actual)
Lead sponsor: Astellas Pharma Global Development, Inc.Industry (pharma, biotech, devices)
Study start: 14 Jun 2021 (actual)
Primary completion: 16 Dec 2022 (actual)
Study completion: 16 Dec 2022 (actual)
First posted: 22 Apr 2021 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

The study will consist of a screening period (up to 27 days [approximately 4 weeks]), treatment period (up to 44 days [6 weeks]) and follow-up period (7 days [1 week]).

The anticipated total duration of the study for each participant, including screening and follow-up, is approximately 75 days (11 week).

An interim analysis may be performed before the end of the study.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Bocidelpar (ASP0367)	Experimental	Drug: BocidelparParticipants will receive Bocidelpar (ASP0367) once daily in the morning for 6 weeks.
Placebo	Placebo comparator	Drug: PlaceboParticipants will receive placebo once daily in the morning for 6 weeks.

Who may qualify

Ages: 18 to 60 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (12 listed)

Participant agrees and is able to adhere to the study requirements for the length of the study, including undergoing both aCPET and SHAPE Test.
Participant has a body mass index (BMI) range of 17.0 to 36 kg/m^2, inclusive and weighs at least 50 kg (male participant) or 40 kg (female participant) at screening.
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.
Female participant is not pregnant and at least 1 of the following conditions apply:
Female participant must agree not to breastfeed starting at screening and throughout the study period and for 28 days after final IP administration.

Key exclusion criteria (33 listed)

Participant has signs and/or symptoms due to a cerebellar, pyramidal, extrapyramidal or other nonmyopathic process (e.g., cerebellar dysfunctions, movement disorder) that would interfere in any nontrivial manner with aCPET or SHAPE Test.
Participant has received any investigational therapy within 28 days or 5 half-lives, whichever is longer, prior to screening.
Participant has any condition which makes the participant unsuitable for study participation.
Participant has used moderate or strong inducers of CYP3A, CYP2C8 and CYP2C19 within the 3 months prior to day -1.
Participant has cardiac troponin I (cTnI) > ULN (or cardiac troponin T > ULN if cTnI is not available) at screening.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BocidelparAlso: MA-0211, ASP0367Oral	Drug	Bocidelpar (ASP0367)
PlaceboOral	Drug	Placebo

Primary outcome

Change from baseline in maximum oxygen uptake at peak exercise (VO2max) during Advanced Cardiopulmonary Exercise Test (aCPET)
Time frame: Baseline and Week 6
V02max is defined as maximum amount of oxygen body can utilize during exercise.
Safety and tolerability assessed by nature, frequency, and severity of Adverse Events (AEs)
Time frame: Baseline up to week 7
Adverse Events (AEs) will be coded using medical dictionary for regulatory activities (MedDRA). An AE is any untoward medical occurrence in a participant, temporally associated with the use of investigational product (IP), whether or not considered related to the IP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IP. This includes events related to the comparator and events related to the (study) procedures.
Number of Participants With Laboratory Value Abnormalities and/or Adverse Events (AEs)
Time frame: Baseline up to week 7
Number of participants with potentially clinically significant laboratory values.
Number of Participants With Vital Sign Abnormalities and/or Adverse Events (AEs)
Time frame: Baseline up to week 7
Number of participants with potentially clinically significant vital signs values.
Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities and/or Adverse Events (AEs)
Time frame: Baseline up to week 7
Number of participants with potentially clinically significant 12-ECG values.
Number of participants with suicidal ideation and/or behavior as assessed by Columbia-Suicide Severity Rating Scale (C-SSRS)
Time frame: Baseline up to week 7
The Columbia-Suicide Severity Rating Scale (C-SSRS) is a clinician administered assessment tool that evaluates suicidal ideation and behavior. Number of participants that have an affirmative response provided to the 5 items for suicidal ideation (1. Wish to be dead, 2. Non-specific active suicidal thoughts, 3. Active suicidal ideation with any methods (not plan) without intent to act, 4. Active suicidal ideation with some intent to act, without specific plan, 5. Active suicidal ideation with specific plan and intent) and/or to the 6 items for suicidal behavior (1. Actual attempt, 2. Interrupted attempt, 3. Aborted attempt, 4. Preparatory acts or behavior, 5. Suicidal Behavior 6. Completed suicide,) will be reported.

5 secondary outcomes

Change from baseline in systemic oxygen extraction (SOE)
Time frame: Baseline and Week 6
Change from baseline in percent predicted VO2max during aCPET
Time frame: Baseline and Week 6
Change from baseline in workload at peak during aCPET
Time frame: Baseline and Week 6
Change from baseline in workload at ventilatory anaerobic threshold (VAT) during aCPET
Time frame: Baseline and Week 6
Change from baseline in multivariable gas exchange index (MGI) during SHAPE Test.
Time frame: Baseline and Week 6

Results and safety

The study was stopped early (reason given: Lack of Pharmacological effect). Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2025.
Link to plain language summary of the study on the Trial Results Summaries websiteLink provided by the sponsor.
Individual participant dataSharing planned: No. Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Senior Medical Director study director

Astellas Pharma Global Development, Inc.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Bocidelpar in people with Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction, run by Astellas Pharma Global Development, Inc. at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 33 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 16 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Lack of Pharmacological effect). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline in maximum oxygen uptake at peak exercise (VO2max) during Advanced Cardiopulmonary Exercise Test (aCPET) (time frame: Baseline and Week 6). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04855201.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2025.