COPDPhase 1NCT04014712
O2 Transport and Utilization in Health and Lung Disease
A Phase 1 study testing Tetrahydrobiopterin in people with COPD and Tetrahydrobiopterin Deficiency, run by University of Massachusetts, Amherst at 1 site in United States.
Trial summary
Skeletal muscle dysfunction is a frequent and clinically relevant systemic manifestation of Chronic Pulmonary Obstructive Disease (COPD), which is still poorly understood. Therefore, the focus of this study is on the role of a deficit in tetrahydrobiopterin and nitric oxide synthase uncoupling induced by chronic… Read more about this study
- Who is it for?
- Ages 18 to 85 yearsHealthy volunteers can take part
- Conditions the study is for
- COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
- What is being tested?
- Tetrahydrobiopterin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2021to Aug 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 85 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- All subjects must be between the age of 18 and 85 years;
- For the COPD cohort, patients with a diagnosis of stable COPD (i.e., those not experiencing an acute exacerbation of symptoms) and spirometric evidence of airway obstruction (FEV1 <80% predicted, FEV/FVC<0.70);
- Ability to perform motor tests;
- Ability to provide informed consent
Who cannot join
- uncontrolled hypertension;
- hyperlipidemia;
- recent exacerbation;
- Major cardiovascular event procedure (<3 months);
- Pregnancy
- known significant hepatic, renal disease, active substance abuse
- contraindication to MRI, claustrophobia
Trial locations and contacts
1 site in 1 country.
United States1 site
Institute of Applied Life Sciences
Amherst, Massachusetts, 01003
About this study
Official title: Role of Nitric Oxide Coupling in Muscle Dysfunction With COPD
Skeletal muscle dysfunction is a frequent and clinically relevant systemic manifestation of Chronic Pulmonary Obstructive Disease (COPD), which is still poorly understood. Therefore, the focus of this study is on the role of a deficit in tetrahydrobiopterin and nitric oxide synthase uncoupling induced by chronic oxidative stress on metabolic and vascular abnormalities in skeletal muscle of patients suffering from COPD.
Read the detailed description
The purpose of this study is to assess the efficacy of supplementation with tetrahydrobiopterin (BH4) for improving vascular and muscle function in patients with COPD and healthy controls.
- Conditions
- COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
- Being tested
- Tetrahydrobiopterin, Placebo oral tablet
- Keywords
- BH4, Exercise, Mitochondria, Vascular function
- Medical subject headings
- Pulmonary Disease, Chronic Obstructive, Phenylketonurias, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TetrahydrobiopterinDrug
Oral, Single dose
Placebo oral tabletDrug
Single dose
| Study group | Role | What they receive |
|---|---|---|
| acute BH4/Tetrahydrobiopterin treatment | Experimental | Oral supplement, Pill, 10 mg/kg of body weight Receives: Drug: Tetrahydrobiopterin |
| Placebo | Placebo comparator | Oral supplement, Pill, Placebo pill with inert excipient Receives: Drug: Placebo oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mitochondrial function
PCr recovery kinetics
Measured: 4 hours
intracellular PO2
Myoglobin oxygenation
Measured: 4 hours
Secondary outcomes (2)
systemic blood markers of oxidative stress
blood markers
Measured: 4 hours
Peripheral Blood flow
Exercise Blood flow by Doppler Ultrasound
Measured: 4 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2019
- Study startApr 2021 (estimated)
- Primary completionAug 2022 (estimated)
- Study completionAug 2022 (estimated)
Why it stopped: Unable to recruit due to COVID-19 pandemic
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for COPDStudies of the same condition, open and under way.
- 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Massachusetts, Amherst
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04014712
- Sponsor's protocol ID
- 2018-4815
- Status last verified
- Dec 2023
- First submitted
- 3 Jul 2019
- Last updated
- 19 Dec 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Role of Nitric Oxide Coupling in Muscle Dysfunction With COPD
- ClinicalTrials.gov ID
- NCT04014712
- Acronym / sponsor ID
- 2018-4815
- Conditions
- COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
- Keywords
- BH4, Exercise, Mitochondria, Vascular function
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Unable to recruit due to COVID-19 pandemic
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of Massachusetts, AmherstUniversity, hospital or other
- Study start
- Apr 2021 (estimated)
- Primary completion
- Aug 2022 (estimated)
- Study completion
- Aug 2022 (estimated)
- First posted
- 10 Jul 2019 (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.
Skeletal muscle dysfunction is a frequent and clinically relevant systemic manifestation of Chronic Pulmonary Obstructive Disease (COPD), which is still poorly understood. Therefore, the focus of this study is on the role of a deficit in tetrahydrobiopterin and nitric oxide synthase uncoupling induced by chronic oxidative stress on metabolic and vascular abnormalities in skeletal muscle of patients suffering from COPD.
Detailed description
The purpose of this study is to assess the efficacy of supplementation with tetrahydrobiopterin (BH4) for improving vascular and muscle function in patients with COPD and healthy controls.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| acute BH4/Tetrahydrobiopterin treatment | Experimental | Drug: TetrahydrobiopterinOral supplement, Pill, 10 mg/kg of body weight |
| Placebo | Placebo comparator | Drug: Placebo oral tabletOral supplement, Pill, Placebo pill with inert excipient |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- All subjects must be between the age of 18 and 85 years;
- For the COPD cohort, patients with a diagnosis of stable COPD (i.e., those not experiencing an acute exacerbation of symptoms) and spirometric evidence of airway obstruction (FEV1 <80% predicted, FEV/FVC<0.70);
- Ability to perform motor tests;
- Ability to provide informed consent
Key exclusion criteria (7 listed)
- uncontrolled hypertension;
- hyperlipidemia;
- recent exacerbation;
- Major cardiovascular event procedure (<3 months);
- Pregnancy
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 |
|---|---|---|
| TetrahydrobiopterinOral, Single dose | Drug | acute BH4/Tetrahydrobiopterin treatment |
| Placebo oral tabletSingle dose | Drug | Placebo |
Primary outcome
Mitochondrial function
Time frame: 4 hours
PCr recovery kinetics
intracellular PO2
Time frame: 4 hours
Myoglobin oxygenation
2 secondary outcomes
systemic blood markers of oxidative stress
Time frame: 4 hours
Peripheral Blood flow
Time frame: 4 hours
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2023.
- Individual participant dataSharing planned: Yes
Related trials for COPD
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Tetrahydrobiopterin in people with COPD and Tetrahydrobiopterin Deficiency, run by University of Massachusetts, Amherst 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 4 inclusion and 7 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: withdrawn, as of the sponsor's last update on 19 Dec 2023. 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 withdrawn before enrolling participants, so there are no results.
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?
Mitochondrial function (time frame: 4 hours). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Dec 2023.