Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy … (NCT01775462) | Clinical Trial Compass
CompletedPhase 2
Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
United States, France, Germany6 participantsStarted 2013-04
Plain-language summary
This Phase 2 first-in-neonate EDI200 study will enroll treatment-naïve, XLHED-affected male newborns in the first two weeks of life. All subjects will meet entry criteria including documentation of an Ectodysplasin (EDA) mutation associated with XLHED. Following Baseline evaluations, EDI200 dosing will be initiated between day-of-life 2 and 14, with each study subject receiving 2 doses/week for a total of 5 doses. The study will enroll subjects in two cohorts with subjects in cohort 1 dosed at 3 mg/kg/dose, associated with partial efficacy, and cohort 2 dosed at 10 mg/kg/dose where enhanced efficacy was demonstrated in the most relevant preclinical model. Given the challenge of identifying families where the subject is yet to be born, it is expected that cohort size and time for recruitment will be variable.
Who can participate
Age range48 Hours – 14 Days
SexMALE
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Inclusion criteria
✓. Male with genetic confirmation of an XLHED diagnosis.
✓. Subject must be at least 48 hours age and no older than 14 days.
✓. Subject will have reached term (defined as 37 weeks gestation or older) prior to receiving first dose study drug.
✓. Written informed consent of both parents (if reasonably available) must be obtained for treatment of their XLHED-affected male infant.
✓. Neither mother nor the XLHED-affected male infant known to have received an investigational study drug in the 9 months prior to study subject enrollment in this study.
✓. No major medical issues that the PI considers a contraindication to participation.
✓. Provide written informed consent/assent.
✓. A full or half-sibling of a study subject where the study subject has received at least one dose of study drug in the Phase 2 XLHED Neonate Study and has not yet completed the study.
Exclusion criteria
What they're measuring
1
Incidence and severity of adverse events
Timeframe: Up to 6 months after dosing
2
To assess the antibody response to EDI200
Timeframe: Up to 6 months after dosing
3
Area under the concentration time curve to the end of the dosing period (AUC0-tau) of EDI200
Timeframe: Pre-dose and 15 minutes and 3, 8, 24 and 48 hours post-dose 1 and pre-dose and 15 minutes and 3, 18, 48 and 168 hours post-dose 5
4
Peak plasma concentration (Cmax) of EDI200
Timeframe: Pre-dose and 15 minutes and 3, 8, 24 and 48 hours post-dose 1 and pre-dose and 15 minutes and 3, 18, 48 and 168 hours post-dose 5
5
Time at which maximum concentration is observed (Tmax) of EDI200
Timeframe: Pre-dose and 15 minutes and 3, 8, 24 and 48 hours post-dose 1 and pre-dose and 15 minutes and 3, 18, 48 and 168 hours post-dose 5