Myotonic Dystrophy Type 1 (DM1)Phase 1/2GalileoNCT06185764
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
A Phase 1/2 study testing VX-670 in people with Myotonic Dystrophy Type 1 (DM1), run by Vertex Pharmaceuticals Incorporated at 26 sites in 10 countries.
Trial summary
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1. Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Myotonic Dystrophy Type 1 (DM1)
- What is being tested?
- VX-670
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 47 participants
- Where
- 26 sitesin 10 countries
- Study dates
- 20 Feb 2024to 2 Feb 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100
Who cannot join
- History of any illness or any clinical condition as pre-specified in the protocol
- Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
26 sites in 10 countries.
26 sites in 10 countries
United States8 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
University of Kansas Medical Center
Fairway, Kansas, 66205
University of Florida Clinical Research Center
Gainesville, Florida, 32608
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Virginia Commonwealth University (Sanger Hall)
Richmond, Virginia, 23298
Stanford Neuromuscular Research
San Carlos, California, 94070
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27104
Canada5 sites
Hopital de Chicoutimi
Chicoutimi
Altasciences Montreal
Montreal
McGIll University
Montreal
University of Ottawa
Ottawa
CHU Research Centre of Quebec
Québec
United Kingdom5 sites
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow
Leonard Wolfson Experimental Neurology Centre CRF
London
St. George's University Hospital
London
Salford Royal Hospital
Salford
Royal Hallamshire Hospital
Sheffield
Australia2 sites
Wesley Research Institute
Auchenflower
Neuroscience Clinical Trials Unit, Alfred Brain
Melbourne
About this study
Official title: A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Being tested
- VX-670, Placebo
- Keywords
- DM1, DM2, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Myotonia, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Vertex, Entrada, VX-670, VX670, PMO, ASO, Myotonic Muscular Dystrophy, Muscular Disorders, Atrophic, Muscular Diseases
- Medical subject headings
- Myotonic Dystrophy, Myotonia, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-670Drug
Solution for intravenous administration.
PlaceboDrug
Solution for intravenous administration.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Single Ascending Dose | Experimental | Participants will be randomized to receive a single dose of different dose levels of VX-670. Receives: Drug: VX-670 |
| Part A: Placebo | Placebo comparator | Participants will be randomized to receive single dose of placebo matched to VX-670. Receives: Drug: Placebo |
| Part B: Single and Multiple Ascending Dose | Experimental | Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A. Receives: Drug: VX-670 |
| Part B: Placebo | Placebo comparator | Participants will be randomized to receive single or multiple doses of placebo matched to VX-670. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Measured: Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
Secondary outcomes (6)
Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
Measured: From Day 1 up to Day 42
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma
Measured: From Day 1 up to Day 42
Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose
Measured: From Day 1 up to Day 168
Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose
Measured: From Day 1 up to Day 168
Part B: Concentration of VX-670 and its Active Component in Muscle
Measured: Baseline, Day 15 and Day 120
Part B: Change in Splicing Index in Muscle Biopsy
Measured: Baseline, Day 15 and Day 120
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2023
- Study start20 Feb 2024
- Primary completion2 Feb 2027 (estimated)
- Study completion2 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy Type 1 (DM1)Studies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06185764
- Sponsor's protocol ID
- VX23-670-001
- Other IDs
- 2023-506028-10-00, 2023
- Acronym
- Galileo
- Status last verified
- Aug 2026
- First submitted
- 15 Dec 2023
- Last updated
- 20 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
- ClinicalTrials.gov ID
- NCT06185764
- Acronym / sponsor ID
- Galileo / VX23-670-001
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Keywords
- DM1, DM2, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Myotonia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 20 Feb 2024 (actual)
- Primary completion
- 2 Feb 2027 (estimated)
- Study completion
- 2 Feb 2027 (estimated)
- First posted
- 29 Dec 2023 (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.
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part A: Single Ascending Dose | Experimental | Drug: VX-670Participants will be randomized to receive a single dose of different dose levels of VX-670. |
| Part A: Placebo | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive single dose of placebo matched to VX-670. |
| Part B: Single and Multiple Ascending Dose | Experimental | Drug: VX-670Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A. |
| Part B: Placebo | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive single or multiple doses of placebo matched to VX-670. |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100
Key exclusion criteria (2 listed)
- History of any illness or any clinical condition as pre-specified in the protocol
- Other protocol defined Inclusion/Exclusion criteria may apply.
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 |
|---|---|---|
| VX-670Solution for intravenous administration. | Drug | Part A: Single Ascending Dose, Part B: Single and Multiple Ascending Dose |
| PlaceboSolution for intravenous administration. | Drug | Part A: Placebo, Part B: Placebo |
Primary outcome
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time frame: Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168
6 secondary outcomes
Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
Time frame: From Day 1 up to Day 42
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma
Time frame: From Day 1 up to Day 42
Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose
Time frame: From Day 1 up to Day 168
Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose
Time frame: From Day 1 up to Day 168
Part B: Concentration of VX-670 and its Active Component in Muscle
Time frame: Baseline, Day 15 and Day 120
Part B: Change in Splicing Index in Muscle Biopsy
Time frame: Baseline, Day 15 and Day 120
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2026.
- Individual participant dataSharing planned: No. Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
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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.
26 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VX-670 in people with Myotonic Dystrophy Type 1 (DM1), run by Vertex Pharmaceuticals Incorporated at 26 sites in 10 countries.
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 1 inclusion and 2 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: under way, not looking for participants, as of the sponsor's last update on 20 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Canada, United Kingdom, Australia, Belgium and 5 more countries. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) (time frame: Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168). 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 20 Aug 2026.