Unknown Brain & Nervous System NIHR-supported project Cancer

VX24-670-101 OLE - An Open-label Extension Study Evaluating the Long term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX 670 in Adult Subjects with Myotonic Dystrophy Type I

Funder NIHR (part of DHSC)
Recipient organisation NIHR Wellcome Trust UCL Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Feb 2025 — Ongoing

In plain English

AI plain-English summary

A drug designed to directly counteract the genetic cause of myotonic dystrophy type 1 (DM1) is now being tested for long-term safety and effectiveness in patients who have already received it in an earlier trial. This matters because DM1 is a progressive, multi-system disease affecting at least 1 in 10,000 people worldwide, yet no approved treatment targets its root cause. Current care only manages symptoms like muscle weakness, myotonia (inability to relax muscles), fatigue, and digestive problems. The experimental drug VX-670 works by binding directly to the abnormal nucleotide repeats in the DMPK gene transcript, aiming to restore normal cell function in muscle and other tissues. If this open-label extension study shows that repeated intravenous doses of VX-670 are safe and tolerable over time, and that muscle strength and myotonia improve, it could pave the way for the first disease-modifying therapy for DM1. Success would mean a fundamental shift from symptom management to treating the underlying biology, potentially slowing or halting disease progression for thousands of patients. The study also collects muscle biopsies to confirm the drug is affecting the molecular cause of the disease, not just the symptoms.

View original technical description
Myotonic Dystrophy Type 1 (DM1) is a serious, progressive, & disabling disease affecting multiple body systems. Its clinical presentation varies among patients. Patient symptoms often include muscle weakness and myotonia (the inability to relax contracted muscles), as well as fatigue, excessive daytime sleepiness, swallowing, digestive, endocrine, & reproductive symptoms. Patient management options are limited to treating symptoms. There are no approved disease-modifying treatments for this disease. DM1 affects at least 1 in 10,000 people worldwide. DM1 is caused by a genetic variation in the DM1 protein kinase (DMPK) gene. This variation causes increased nucleotide repeats to block normal protein function in many cell types, including those that make up muscle tissue. VX-670, is designed to target the cause of DM1 by directly binding the increased nucleotide repeats in the DMPK transcript, allowing normal cell function to proceed. It is anticipated that overall muscle weakness and myotonia will improve in subjects treated with VX-670. This research study, VX24-670-101, is offered to subjects who took part in parent study VX23-670-001. The objective of study is to learn how safe, tolerable and effective multiple doses of VX-670 are in people with DM1. This will be accomplished through collection of participant safety data throughout the time of treatment. Symptom improvement will also be measured through various functional assessments and participant -reported outcome questionnaires (PROs). In addition, the effect of VX-670 on the biology that underlies all symptoms of DM1 will be evaluated by looking at muscle biopsies performed before the first dose (participants from parent Part A only) and again at study week 24 to accomplish this objective. VX-670 is investigational. It will be administered intravenously once every 6 weeks. Participant dose will be linked to that received in the parent study, with any changes overseen by an Independent Data Monitoring Committee

Researchers

Chris Turner (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type I
Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type 1.
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
VX23-670-001 - A Phase 1/2, Randomized, Double-blind, Placebo controlled Single- and Multiple dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX23-670 in Adult Subjects with Myotonic Dystrophy Type 1
Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Pharmacokinetics, and PharmacodyN/Amics of VX-670 in Adult Subjects with Myotonic Dystrophy Type 1.

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