Clinical trial • Phase I/II • Musculoskeletal
VX-670 for Myotonic dystrophy type 1
Phase I/II trial of VX-670 for Myotonic dystrophy type 1. open-label. 20 participants.
Overview
- Trial Therapeutic Area
- Musculoskeletal
- Trial Disease
- Myotonic dystrophy type 1
- Trial Stage
- Phase I/II
- Drug Modality
- Oligonucleotide
Key dates
- Initial CTIS Submission Date
- 11-02-2025
- First CTIS Authorization Date
- 03-06-2025
Trial design
open-label Phase I/II trial across 6 sites in Belgium, Netherlands, Spain and others.
- Open Label
- Yes
- Target Sample Size
- 20
Eligibility
Recruits 20 Vulnerable populations are not selected for this trial (isVulnerablePopulationSelected: false). Informed consent must be signed and dated by the subject; if the subject is cognitively able to provide consent but unable to write, an impartial witness may sign ("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)"). All provided ICFs and subject information documents are adult-focused and country/language-specific (multiple language versions available). No assent process for minors is specified..
- Pregnancy Exclusion
- 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.
- Vulnerable Population
- Vulnerable populations are not selected for this trial (isVulnerablePopulationSelected: false). Informed consent must be signed and dated by the subject; if the subject is cognitively able to provide consent but unable to write, an impartial witness may sign ("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)"). All provided ICFs and subject information documents are adult-focused and country/language-specific (multiple language versions available). No assent process for minors is specified.
Inclusion criteria
- {"criterion_text":"- 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).\n- 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.\n- 3. Willing and able to comply with scheduled visits, treatment plan, study restrictions, safety tests, contraceptive guidelines, and other study procedures."}
Exclusion criteria
- {"criterion_text":"- 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.\n- 2.\tSubject 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.\n- 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.\n- 4. Use of the substances, activities, or devices as indicated in Section 9.4, during the specified times.\n- 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.\n- 6. Exposure to any cell and genetic therapies. Note that antisense oligonucleotides are not considered a genetic therapy.\n- 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\n- 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.\n- 9. Subject is an employee of the sponsor."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Safety and tolerability of VX-670 based on the assessment of adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory values","definition_or_measurement_approach":"Assessment of adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory values."}
Secondary endpoints
- {"endpoint_text":"- VX-670 and PMO-0221a concentrations in plasma","definition_or_measurement_approach":"Measurement of plasma concentrations of VX-670 and PMO-0221a (plasma PK)."}
Recruitment
- Planned Sample Size
- 20
- Recruitment Window Months
- 44
- Consent Approach
- Informed consent is obtained from each adult subject; the subject signs and dates the informed consent form (ICF). If the subject is cognitively able to provide consent but unable to write, an impartial witness may sign the ICF on their behalf. ICFs and subject information sheets are provided in multiple language and country-specific versions (e.g., English, French, Dutch, German, Italian, Spanish) as indicated by the available country-specific L1/SIS and ICF documents.
Geography
- Total Number Of Sites
- 6
- Total Number Of Participants
- 20
Belgium
- Earliest CTIS Part Ii Submission Date
- 29-04-2025
- Latest Decision Or Authorization Date
- 01-07-2025
- Processing Time Days
- 63
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- UZ Leuven
- Department Name
- Neurology
- Contact Person Name
- Kristl Claeys
- Contact Person Email
- kristl.claeys@uzleuven.be
- Number Of Participants
- 3
Netherlands
- Earliest CTIS Part Ii Submission Date
- 29-04-2025
- Latest Decision Or Authorization Date
- 03-06-2025
- Processing Time Days
- 35
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Academisch Ziekenhuis Maastricht
- Department Name
- Neurology, School of Mental Health and Neurosciences
- Contact Person Name
- Karin Faber
- Contact Person Email
- c.faber@mumc.nl
- Number Of Participants
- 2
Spain
- Earliest CTIS Part Ii Submission Date
- 23-05-2025
- Latest Decision Or Authorization Date
- 01-07-2025
- Processing Time Days
- 39
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Neurología
- Contact Person Name
- Nuria Muelas Gómez
- Contact Person Email
- nuriamugo@gmail.com
- Number Of Participants
- 2
Italy
- Earliest CTIS Part Ii Submission Date
- 23-04-2025
- Latest Decision Or Authorization Date
- 04-06-2025
- Processing Time Days
- 42
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Centro Clinico Nemo
- Department Name
- UOC Neuroriabilitazione Neurologica
- Contact Person Name
- Valeria Sansone
- Contact Person Email
- valeria.sansone@centrocliniconemo.it
- Number Of Participants
- 4
France
- Earliest CTIS Part Ii Submission Date
- 28-04-2025
- Latest Decision Or Authorization Date
- 04-06-2025
- Processing Time Days
- 37
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Association Institut De Myologie
- Department Name
- Neuromyology
- Contact Person Name
- Guillaume Bassez
- Contact Person Email
- guillaume.bassez@aphp.fr
- Number Of Participants
- 5
Germany
- Earliest CTIS Part Ii Submission Date
- 21-05-2025
- Latest Decision Or Authorization Date
- 01-07-2025
- Processing Time Days
- 41
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Friedrich Baur Institute An Der Neurologischen Klinik Und Poliklinik
- Department Name
- Neurologische Klinik und Poliklinik
- Contact Person Name
- Stephan Wenninger
- Contact Person Email
- stephan.wenninger@med.uni-muenchen.de
- Number Of Participants
- 4
Sponsor
Primary sponsor
- Full Name
- Vertex Pharmaceuticals Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Investigational products
- Investigational Product Name
- VX-670 Solution for Injection/Infusion
- Active Substance
- VX-670
- Modality
- Oligonucleotide
- Routes Of Administration
- Intravenous administration
- Route
- Intravenous administration
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