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A Phase 1/2 Study of VX-670 in Adult Subjects with Myotonic Dystrophy Type 1
EU CTIS ID: 2023-506028-10-00
What this study is testing
Part A: To evaluate the safety and tolerability of single ascending doses of VX 670 in subjects with myotonic dystrophy type 1 (DM1) Part B: To evaluate the safety and tolerability of single and multiple ascending doses of VX 670 in subjects with DM1
- Phase I and Phase II (Integrated)- First administration to humans
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
- 3. Body mass index (BMI) <35.0 kg/m2, inclusive, and a total body weight >40 kg.
- 4. Subjects (male and female) between the ages of 18 to 64 years, inclusive. Women of childbearing potential may be enrolled as allowed by local regulatory guidance.
- 5. Documented clinical diagnosis of DM1 with age of onset >1 year of age and documented positive genetic test for DM1.
- 7. Part B: Ambulatory, defined as having the ability to complete 10-meter walk/run timed test unassisted (e.g., without the use of a cane or walker) or with the use of a brace or other orthotic device only.
- 8. Part B: Evidence of myotonia, defined by HOT of ≥2 seconds.
- 9. Part B: Evidence of weakness, defined by percent predicted QMT of hand grip of 5 to 80%
You likely can't join if
- 1. History of any illness or any clinical condition that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of or participation in the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, history of relevant drug or food allergies; history of cardiovascular or central nervous system disease (other than DM1); history or presence of clinically significant pathology; had major surgery or had significant trauma within 4 weeks before the first study drug dose;history of mental disease; significant intellectual or behavioral disability; and history of cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 diagnosed ≥ 3 years ago with no recurrence in the past 3 years).
- 11. Clinically significant liver disease, even if intermittent.
- 12. History of cardiac anomalies.
- 13. Clinically significant kidney disease.
- 14. Exposure to any other investigational nucleic acid, cell and genetic therapies, including siRNA, ASO, mRNA within 6 months before Day 1 or 5 half-lives of investigational agent (confirmed at Screening), whichever is longer.
- 15. Exposure to any other investigational drugs or devices within 1 month before Day 1
See the full eligibility criteria
Who can join
- 3. Body mass index (BMI) <35.0 kg/m2, inclusive, and a total body weight >40 kg.
- 4. Subjects (male and female) between the ages of 18 to 64 years, inclusive. Women of childbearing potential may be enrolled as allowed by local regulatory guidance.
- 5. Documented clinical diagnosis of DM1 with age of onset >1 year of age and documented positive genetic test for DM1.
- 7. Part B: Ambulatory, defined as having the ability to complete 10-meter walk/run timed test unassisted (e.g., without the use of a cane or walker) or with the use of a brace or other orthotic device only.
- 8. Part B: Evidence of myotonia, defined by HOT of ≥2 seconds.
- 9. Part B: Evidence of weakness, defined by percent predicted QMT of hand grip of 5 to 80%
- 6. Left ventricular ejection fraction (LVEF) >55% within the past 3 months.
What rules you out
- 1. History of any illness or any clinical condition that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of or participation in the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, history of relevant drug or food allergies; history of cardiovascular or central nervous system disease (other than DM1); history or presence of clinically significant pathology; had major surgery or had significant trauma within 4 weeks before the first study drug dose;history of mental disease; significant intellectual or behavioral disability; and history of cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 diagnosed ≥ 3 years ago with no recurrence in the past 3 years).
- 11. Clinically significant liver disease, even if intermittent.
- 12. History of cardiac anomalies.
- 13. Clinically significant kidney disease.
- 14. Exposure to any other investigational nucleic acid, cell and genetic therapies, including siRNA, ASO, mRNA within 6 months before Day 1 or 5 half-lives of investigational agent (confirmed at Screening), whichever is longer.
- 15. Exposure to any other investigational drugs or devices within 1 month before Day 1
- 16. Part B: Any contraindication to a muscle biopsy in the opinion of the investigator including but not limited to: a limb injury, bleeding diathesis, ascites, or significant atrophy of the tibialis anterior muscles.
- 17. Thyroid dysfunction that is untreated (if on thyroid hormone replacement therapy, need to have adequate and stable replacement over the previous 6 months).
- 18. Diabetes that is uncontrolled, in the opinion of the Investigator.
- 2. History of febrile illness or other acute illness that has not fully resolved within 14 days before the first dose of study drug.
- 3. Median QTcF of triplicate standard 12-lead ECGs >450 msec at Screening.
- 4. For female subjects: Females who are currently breastfeeding or pregnant or planning to become pregnant during the study or within 180 days after the last dose of study drug. a. 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.
- 5. Blood donation (of approximately 1 pint [500 mL] or more) within 56 days before the first dose of study drug.
- 6. Use of the substances, activities, or devices within the specified duration before the first study drug dose.
- 7. A screen positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or antibodies against human immunodeficiency virus 1 or 2 (HIV1 and HIV2 Abs).
- 8. Hypersensitivity to any component of the investigational drug product or placebo
- 10. Abnormal and clinically significant values for clinical chemistry, hematology, coagulation, or urinalysis parameters at Screening unless explained by disease or are benign in nature (such as Gilbert’s disease).
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- Australia
- United States
- 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 Canada; Australia; United States; 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.