Muscular DystrophiesPhase 1NCT05492734
A Study to Assess the Feasibility of Non-invasive Dried Blood Sampling
A Phase 1 study testing EDG-5506 in people with Muscular Dystrophies, run by Edgewise Therapeutics, Inc. at 1 site in United States.
Trial summary
This is a Phase I, open-label, single-dose, 1-period study to compare EDG-5506 concentrations measured from venous and capillary blood sampling in healthy adult subjects. EDG-5506 is an investigational product designed to selectively modulate a fast skeletal muscle motor protein called myosin that is part of the… Read more about this study
- Who is it for?
- Ages 19 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Muscular Dystrophies
- What is being tested?
- EDG-5506
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Aug 2022to 19 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 19 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy, adult, males or females, aged 19-55 years (inclusive) at the Screening visit.
- Females must be of non-childbearing potential. Males with female partners must use a medically accepted contraceptive regimen or abstain from sexual intercourse for 90 days after dosing.
- Non-smoker and must not have used any tobacco products within 3 months prior to the Screening visit.
- Body weight greater than 50 kg and body mass index (BMI) between 18.0 and 30.0 kg/m2 inclusive at the Screening visit.
- Medically healthy with no clinically significant findings for oculofacial and proximal limb strength assessments and swallowing function assessment, medical history, physical examination, laboratory profiles, vital signs, and ECGs.
Who cannot join
- History of, or physical examination findings indicating clinically significant endocrine, neurological, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases that, in the opinion of the PI, would render the subject being unsuitable for the study.
- Female participants with a positive pregnancy test at the screening visit or are lactating.
- Poor venous access and/or cannot tolerate venipuncture.
- Unable to refrain from or anticipates the use of any drugs (including prescription and non-prescription medications, herbal remedies, vitamin supplements, or grapefruit/Seville orange containing products) beginning 14 days prior to dosing. Any drugs known to be moderate or strong inducers of CYP3A4 enzymes cannot be used within 28 days prior to dosing.
- Participation in any other investigational drug study within 30 days prior to dosing.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Study to Assess the Feasibility of Non-invasive Dried Blood Sampling for Assessment of EDG-5506 Concentrations
This is a Phase I, open-label, single-dose, 1-period study to compare EDG-5506 concentrations measured from venous and capillary blood sampling in healthy adult subjects.
EDG-5506 is an investigational product designed to selectively modulate a fast skeletal muscle motor protein called myosin that is part of the contraction machinery of the damage-prone muscle fibers.
Read the detailed description
Ten (10) healthy, adult male and female (of non-childbearing potential only) participants will be enrolled in the single-site study. Participants will receive a single oral dose of EDG-5506 on Day 1. Serial blood sampling will be collected pre-dose and up to 26 hours post-dose using venous blood sampling by venipuncture and capillary blood sampling by a capillary blood self-sampling device. On Day 3, participants will self-collect a capillary blood sample at home. The final on-site visit will be on Day 7 when a last blood sample will be collected using venous blood sampling by venipuncture and capillary blood sampling by a capillary blood self-sampling device.
- Conditions
- Muscular Dystrophies
- Being tested
- EDG-5506
- Keywords
- Healthy Volunteers
- Medical subject headings
- Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EDG-5506Drug
single oral dose
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Drug: EDG-5506 Receives: Drug: EDG-5506 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference in area under the concentration-time curve (AUC0-24, AUC0-26, AUC0-144 and AUC0-t) measurements for EDG-5506 in blood collected by capillary blood self-sampling versus venous blood sampling
Measured: 7 days
Plasma maximum measured drug concentration (Cmax) measurements for EDG-5506 in blood collected by capillary blood self-sampling versus venous blood sampling
Measured: 7 days
Time of maximum concentration (Tmax) measurements for EDG-5506 in blood collected by capillary blood self-sampling versus venous blood sampling
Measured: 7 days
Secondary outcomes (7)
Area under the concentration-time curve (AUC0-24, AUC0-26, AUC0-144 and AUC0-t) measurements for EDG-5506 in plasma.
Measured: 7 days
Plasma maximum measured drug concentration (Cmax) measurements for EDG-5506 in plasma.
Measured: 7 days
Time of maximum concentration (Tmax) measurements for EDG-5506 in plasma.
Measured: 7 days
Incidence of treatment-emergent adverse events
Measured: 7 days
Incidence of abnormal clinical laboratory test results
Measured: 7 days
Incidence of abnormal vital signs
Measured: 7 days
Incidence of abnormal electrocardiograms (ECGs)
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2022
- Study start12 Aug 2022
- Primary completion19 Aug 2022
- Study completion19 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular DystrophiesStudies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sam Collins, MBBS, PhD, Study chair, Edgewise Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05492734
- Sponsor's protocol ID
- EDG-5506-104
- Status last verified
- Sep 2022
- First submitted
- 5 Aug 2022
- Last updated
- 2 Sep 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Assess the Feasibility of Non-invasive Dried Blood Sampling for Assessment of EDG-5506 Concentrations
- ClinicalTrials.gov ID
- NCT05492734
- Acronym / sponsor ID
- EDG-5506-104
- Conditions
- Muscular Dystrophies
- Keywords
- Healthy Volunteers
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Edgewise Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Aug 2022 (actual)
- Primary completion
- 19 Aug 2022 (actual)
- Study completion
- 19 Aug 2022 (actual)
- First posted
- 8 Aug 2022 (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.
This is a Phase I, open-label, single-dose, 1-period study to compare EDG-5506 concentrations measured from venous and capillary blood sampling in healthy adult subjects.
EDG-5506 is an investigational product designed to selectively modulate a fast skeletal muscle motor protein called myosin that is part of the contraction machinery of the damage-prone muscle fibers.
Detailed description
Ten (10) healthy, adult male and female (of non-childbearing potential only) participants will be enrolled in the single-site study. Participants will receive a single oral dose of EDG-5506 on Day 1. Serial blood sampling will be collected pre-dose and up to 26 hours post-dose using venous blood sampling by venipuncture and capillary blood sampling by a capillary blood self-sampling device. On Day 3, participants will self-collect a capillary blood sample at home. The final on-site visit will be on Day 7 when a last blood sample will be collected using venous blood sampling by venipuncture and capillary blood sampling by a capillary blood self-sampling device.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: EDG-5506Drug: EDG-5506 |
Who may qualify
- Ages
- 19 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Healthy, adult, males or females, aged 19-55 years (inclusive) at the Screening visit.
- Females must be of non-childbearing potential. Males with female partners must use a medically accepted contraceptive regimen or abstain from sexual intercourse for 90 days after dosing.
- Non-smoker and must not have used any tobacco products within 3 months prior to the Screening visit.
- Body weight greater than 50 kg and body mass index (BMI) between 18.0 and 30.0 kg/m2 inclusive at the Screening visit.
- Medically healthy with no clinically significant findings for oculofacial and proximal limb strength assessments and swallowing function assessment, medical history, physical examination, laboratory profiles, vital signs, and ECGs.
Key exclusion criteria (5 listed)
- History of, or physical examination findings indicating clinically significant endocrine, neurological, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases that, in the opinion of the PI, would render the subject being unsuitable for the study.
- Female participants with a positive pregnancy test at the screening visit or are lactating.
- Poor venous access and/or cannot tolerate venipuncture.
- Unable to refrain from or anticipates the use of any drugs (including prescription and non-prescription medications, herbal remedies, vitamin supplements, or grapefruit/Seville orange containing products) beginning 14 days prior to dosing. Any drugs known to be moderate or strong inducers of CYP3A4 enzymes cannot be used within 28 days prior to dosing.
- Participation in any other investigational drug study within 30 days prior to dosing.
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 |
|---|---|---|
| EDG-5506single oral dose | Drug | Treatment |
Primary outcome
Difference in area under the concentration-time curve (AUC0-24, AUC0-26, AUC0-144 and AUC0-t) measurements for EDG-5506 in blood collected by capillary blood self-sampling versus venous blood sampling
Time frame: 7 days
Plasma maximum measured drug concentration (Cmax) measurements for EDG-5506 in blood collected by capillary blood self-sampling versus venous blood sampling
Time frame: 7 days
Time of maximum concentration (Tmax) measurements for EDG-5506 in blood collected by capillary blood self-sampling versus venous blood sampling
Time frame: 7 days
7 secondary outcomes
Area under the concentration-time curve (AUC0-24, AUC0-26, AUC0-144 and AUC0-t) measurements for EDG-5506 in plasma.
Time frame: 7 days
Plasma maximum measured drug concentration (Cmax) measurements for EDG-5506 in plasma.
Time frame: 7 days
Time of maximum concentration (Tmax) measurements for EDG-5506 in plasma.
Time frame: 7 days
Incidence of treatment-emergent adverse events
Time frame: 7 days
Incidence of abnormal clinical laboratory test results
Time frame: 7 days
Incidence of abnormal vital signs
Time frame: 7 days
Incidence of abnormal electrocardiograms (ECGs)
Time frame: 7 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2022.
- Sponsor WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Sam Collins, MBBS, PhD study chair
Edgewise Therapeutics, 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 EDG-5506 in people with Muscular Dystrophies, run by Edgewise Therapeutics, 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 5 inclusion and 5 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: finished, as of the sponsor's last update on 2 Sep 2022. 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. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Difference in area under the concentration-time curve (AUC0-24, AUC0-26, AUC0-144 and AUC0-t) measurements for EDG-5506 in blood collected by capillary blood self-sampling versus venous blood sampling (time frame: 7 days). The study lists 3 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 2 Sep 2022.