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Purpose

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.

Condition

Eligibility

Eligible Ages
Between 18 Years and 64 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100

Exclusion Criteria

  • History of any illness or any clinical condition as pre-specified in the protocol Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Part A: Single Ascending Dose 		Participants will be randomized to receive a single dose of different dose levels of VX-670.
  • Drug: VX-670
    Solution for intravenous administration.
Placebo Comparator
Part A: Placebo 		Participants will be randomized to receive single dose of placebo matched to VX-670.
  • Drug: Placebo
    Solution for intravenous administration.
Experimental
Part B: Single and Multiple Ascending Dose 		Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A.
  • Drug: VX-670
    Solution for intravenous administration.
Placebo Comparator
Part B: Placebo 		Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.
  • Drug: Placebo
    Solution for intravenous administration.

Recruiting Locations

University of Kansas Medical Center
Fairway, Kansas 66205

More Details

Status
Recruiting
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact

Medical Information
617-341-6777
medicalinfo@vrtx.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.