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A Study of the Long-Term Safety and Efficacy of VX-670 in Patients With Myotonic Dystrophy Type I
EU CTIS ID: 2024-517983-47-00
What this study is testing
To evaluate the long-term safety and tolerability of VX-670 in subjects with myotonic dystrophy type 1 (DM1)
- Phase I and Phase II (Integrated)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Subject (or his or her impartial witness if the subject is cognitively able to provide consent but unable to write) will sign and date an informed consent form (ICF).
- 2. Subject must have completed an interventional VX-670 study (per interventional study completion definition) a. Study 001: Subjects in Part B must have received at least 2 doses of study drug.
- 3. Willing and able to comply with scheduled visits, treatment plan, study restrictions, safety tests, contraceptive guidelines, and other study procedures.
You likely can't join if
- 1. History or evidence of any illness or any clinical condition (e.g. clinically significant kidney disease) that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include but is not limited to major surgery, significant trauma, or clinically significant laboratory abnormalities (e.g., hypomagnesemia) within 4 weeks before the first study drug dose.
- 2. Subject developed any VX-670-related serious or severe AE in the interventional study that, in the opinion of the sponsor, might pose an additional risk to the subject.
- 3. For female subjects: Pregnant, nursing, or planning to become pregnant during the study or within 180 days after the last dose of study drug. For male subjects: Male subjects with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 180 days after the last dose of study drug.
- 4. Use of the substances, activities, or devices as indicated in Section 9.4, during the specified times.
- 5. Exposure to any investigational nucleic acid or cell and genetic therapy other than VX-670, including siRNA, ASO, mRNA within 6 months before Day 1 or 5 half-lives of investigational agent (confirmed at Screening), whichever is longer.
- 6. Exposure to any cell and genetic therapies. Note that antisense oligonucleotides are not considered a genetic therapy.
See the full eligibility criteria
Who can join
- 1. Subject (or his or her impartial witness if the subject is cognitively able to provide consent but unable to write) will sign and date an informed consent form (ICF).
- 2. Subject must have completed an interventional VX-670 study (per interventional study completion definition) a. Study 001: Subjects in Part B must have received at least 2 doses of study drug.
- 3. Willing and able to comply with scheduled visits, treatment plan, study restrictions, safety tests, contraceptive guidelines, and other study procedures.
What rules you out
- 1. History or evidence of any illness or any clinical condition (e.g. clinically significant kidney disease) that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include but is not limited to major surgery, significant trauma, or clinically significant laboratory abnormalities (e.g., hypomagnesemia) within 4 weeks before the first study drug dose.
- 2. Subject developed any VX-670-related serious or severe AE in the interventional study that, in the opinion of the sponsor, might pose an additional risk to the subject.
- 3. For female subjects: Pregnant, nursing, or planning to become pregnant during the study or within 180 days after the last dose of study drug. For male subjects: Male subjects with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 180 days after the last dose of study drug.
- 4. Use of the substances, activities, or devices as indicated in Section 9.4, during the specified times.
- 5. Exposure to any investigational nucleic acid or cell and genetic therapy other than VX-670, including siRNA, ASO, mRNA within 6 months before Day 1 or 5 half-lives of investigational agent (confirmed at Screening), whichever is longer.
- 6. Exposure to any cell and genetic therapies. Note that antisense oligonucleotides are not considered a genetic therapy.
- 7. Exposure to any other investigational drugs or devices within 1 month before Day 1 or 5 half-lives before the first dose of study drug, whichever is longer
- 8. Subject, or close relative of the subject, is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.
- 9. Subject is an employee of the sponsor.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Canada
- Australia
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Canada; Australia; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.