Ignite Creation Date:
2024-05-06 @ 1:19 AM
Last Modification Date:
2024-10-26 @ 11:02 AM
Study NCT ID:
NCT01775462
Status:
COMPLETED
Last Update Posted:
2016-01-20
First Post:
2013-01-17
Brief Title:
Phase 2 Study to Evaluate Safety Pharmacokinetics Immunogenicity and PharmacodynamicsEfficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia XLHED
Sponsor:
Edimer Pharmaceuticals
Organization:
Edimer Pharmaceuticals
Study Overview
Official Title:
A Phase 2 Open-label Dose-escalation Study to Evaluate the Safety Pharmacokinetics Immunogenicity and PharmacodynamicsEfficacy of EDI200 an EDA-A1 Replacement Protein Administered to Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia XLHED
Status:
COMPLETED
Status Verified Date:
2016-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ECP-002
Brief 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 dosesweek for a total of 5 doses The study will enroll subjects in two cohorts with subjects in cohort 1 dosed at 3 mgkgdose associated with partial efficacy and cohort 2 dosed at 10 mgkgdose 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
Detailed Description:
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 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 dosesweek for a total of 5 doses This dosing regimen mirrors that used to enhance efficacy in the dog XLHED model considered to be most relevant to the clinical study design The study will enroll subjects in two cohorts with subjects in cohort 1 dosed at 3 mgkgdose associated with partial efficacy and cohort 2 dosed at 10 mgkgdose 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 The sponsor anticipates enrollment and dosing of 6-10 subjects over a 12-18 month period 3-5 subjects per cohort
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None