Health TrialsbyHealthPanel

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

ConditionMyotonic Dystrophy 1
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age16-65
SponsorPepGen Inc

About this trial

The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats

Presence of myotonia

Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed

Body Mass Index (BMI) of < 35.0 kg/m²

Disqualifiers

Congenital DM1

Known history or presence of any clinically significant conditions that may interfere with study safety assessments

Abnormal laboratory tests at screening considered clinically significant by the Investigator

Medications specific for the treatment of myotonia within 2 weeks prior to screening

Trial design

Design model

Parallel

Treatments tested in this trial

  • PGN-EDODM1

    Drug

    Administered by intravenous (IV) infusion

  • Placebo

    Other intervention

    Administered by intravenous (IV) infusion

Treatment groups

24 Participants
are divided into 2 treatment groups
Group A: PGN-EDODM1Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Safety and tolerability as assessed by number of participants with Adverse Events (AEs)

Time frame
Baseline through Day 112

Secondary outcomes

1

Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1

Time frame
Day 1 through Day 84
2

Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1

Time frame
Day 1 through Day 84
3

Apparent Terminal Half-Life (t½) of PGN-EDODM1

Time frame
Day 1 through Day 84
4

Area Under the Concentration-time Curve of PGN-EDODM1

Time frame
Day 1 through Day 84

Other outcomes

Sponsors and contacts

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

PepGen Inc

Lead sponsor

Source ClinicalTrials.gov
Apply now