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A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

ConditionMyotonic Dystrophy Type 1 (DM1)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-64
SponsorVertex Pharmaceuticals Incorporated

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

None

Trial design

Design model

Sequential

Treatments tested in this trial

  • VX-670

    Drug

    Solution for intravenous administration.

  • Placebo

    Drug

    Solution for intravenous administration.

Treatment groups

52 Participants
are divided into 4 treatment groups
Group A: Part A: Single Ascending DoseExperimental treatment 1 intervention
Group B: Part A: PlaceboPlacebo comparator 1 intervention
Group C: Part B: Single and Multiple Ascending DoseExperimental treatment 1 intervention
Group D: Part B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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

Secondary outcomes

1

Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma

Time frame
From Day 1 up to Day 42
2

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
3

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
4

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Vertex Pharmaceuticals Incorporated

Lead sponsor

Source ClinicalTrials.gov
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