A Phase 2 interventional study of EDI200 in X-Linked Hypohidrotic Ectodermal Dysplasia, sponsored by Edimer Pharmaceuticals. Completed at 7 sites in 5 countries. Open to male participants aged 48 Hours to 14 Days. Per ClinicalTrials.gov, last updated 2016-01-20.
Sponsored by Edimer Pharmaceuticals · Phase 2, Interventional, and Treatment
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.
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 doses/week 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 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. The sponsor anticipates enrollment and dosing of 6-10 subjects over a 12-18 month period, 3-5 subjects per cohort.
21 studies on the registry are indexed under Ectodermal Dysplasia 1, Anhidrotic; 1 is open to participants now.
Browse Ectodermal Dysplasia 1, Anhidrotic studies →Edimer Pharmaceuticals is the lead sponsor of 13 studies on the registry; none are open to participants now.
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Subjects for study drug administration must meet all of the following criteria to be enrolled:
Siblings of subjects receiving study drug must meet all of the following criteria to be enrolled in the natural history sub-study (no age limit involved):
Exclusion Criteria:
Subjects for study drug administration who meet any of the following criteria cannot be enrolled in this study:
Siblings of subjects receiving study drug who meet any of the following criteria cannot be enrolled in the natural history sub-study:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Five doses of EDI200 given at 3 mg/kg twice weekly
Drug: EDI200
Five doses of EDI200 given at 10 mg/kg twice weekly
Drug: EDI200
3 or 10 mg/kg of EDI200
Also known as: APO200
Incidence and severity of adverse events
Time frame: Up to 6 months after dosing
To assess the antibody response to EDI200
Time frame: Up to 6 months after dosing
Area under the concentration time curve to the end of the dosing period (AUC0-tau) of EDI200
Time frame: 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
Peak plasma concentration (Cmax) of EDI200
Time frame: 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
Time at which maximum concentration is observed (Tmax) of EDI200
Time frame: 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
To assess the pharmacodynamics/efficacy (growth and development) of EDI200
Time frame: Baseline and 2, 4 and 6 months
To assess the pharmacodynamics/efficacy (dentition) of EDI200
Time frame: Baseline and post-six months (extension study)
To assess the pharmacodynamics/efficacy (craniofacial development) of EDI200
Time frame: Baseline and 6 months
To assess the pharmacodynamics/efficacy (sweat duct density) of EDI200
Time frame: Baseline and 2 and 6 months
To assess the pharmacodynamics/efficacy (sweat rate) of EDI200
Time frame: Baseline and 2 and 6 months
To assess the pharmacodynamics/efficacy (Dry eye signs and symptoms) of EDI200
Time frame: Baseline and 2 and 6 months
To assess the pharmacodynamics/efficacy (thermoregulation) of EDI200
Time frame: Baseline and study day 21
To assess the pharmacodynamics/efficacy (molecular expression profile of skin biopsy tissue) of EDI200
Time frame: Baseline, study days 1 and 15
The record gives a start of Apr 2013, a primary completion of Dec 2015 and a completion of Dec 2015. It does not say whether these dates are actual or estimated.
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This study is completed, as verified in Jan 2016. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Fc-EDA
Ectodermal Dysplasia 1, Anhidrotic →
Edimer Pharmaceuticals