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A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.

ConditionCharcot-Marie-Tooth Disease, Type 1A
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-60
SponsorNovartis Pharmaceuticals

About this trial

The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Provide written informed consent before any assessment is performed.

Be male or female and 18 to 60 (inclusive) years of age at the time of screening.

Participant must have a clinical diagnosis of Charcot-Marie-Tooth Disease Type 1A (CMT1A) including verified documentation of genetic testing showing duplication of the PMP22 gene by an accredited/certified laboratory (according to local regulations)

Have detectable upper extremity nerve conduction velocities (sensory and/or motor) in at least one extremity at screening.

Disqualifiers

Unable to communicate well with the investigator, to understand and comply with the visits and procedures of the study.

History of cardiac, renal, liver, hematological, immune system disorders.

Pregnant/nursing female participants. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for the duration of the follow-up period. Sexually active males unless they use a condom during intercourse.

Inability or unwillingness to provide serial skin biopsy samples.

Trial design

Design model

Parallel

Treatments tested in this trial

  • EDK060, dose A

    Drug

    EDK060, dose A, Single dose, IV infusion

  • EDK060, dose B

    Drug

    EDK060, dose B, Single dose, IV infusion

  • EDK060, dose C

    Drug

    EDK060, dose C, Single dose, IV infusion

  • EDK060, dose D

    Drug

    EDK060, dose D, Single dose, IV infusion

  • Placebo

    Other intervention

    Placebo

Treatment groups

28 Participants
are divided into 2 treatment groups
Group A: EDK060Experimental treatment 4 interventions
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Incidence of AEs and SAEs by treatment group, including changes in vital signs, laboratory measures, neurological examinations and electrophysiology measures qualifying and reported as AEs.

Time frame
From baseline up to day 140

Secondary outcomes

1

EDK060 Plasma PK parameters - Cmax

The maximum (peak) observed EDK060 plasma concentration after single dose administration (mass x volume-1)

Time frame
From baseline up to day 140
2

EDK060 Plasma PK parameters - Tmax

The time to reach maximum (peak) plasma, EDK060 concentration after single dose administration (time)

Time frame
From baseline up to day 140
3

EDK060 Plasma PK parameters - AUClast

The area under the concentration-time curve of EDK060 from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1)

Time frame
From baseline up to day 140

Other outcomes

Sponsors and contacts

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

Novartis Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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