Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1.

ABSTRACT:

Objective

To evaluate the safety and tolerability of recombinant human insulin-like growth factor 1 (rhIGF-1) complexed with IGF binding protein 3 (rhIGF-1/rhIGFBP-3) in patients with myotonic dystrophy type 1 (DM1).

Design

Open-label dose-escalation clinical trial.

Setting

University medical center.

Participants

Fifteen moderately affected ambulatory participants with genetically proven myotonic dystrophy type 1.

Intervention

Participants received escalating dosages of subcutaneous rhIGF-1/rhIGFBP-3 for 24 weeks followed by a 16-week washout period.

Main outcome measures

Serial assessments of safety, muscle mass, muscle function, and metabolic state were performed. The primary outcome variable was the ability of participants to complete 24 weeks

SUBMITTER: Heatwole CR 

PROVIDER: S-EPMC3374954 | biostudies-literature | 2011 Jan

REPOSITORIES: biostudies-literature