X-Linked Hypohidrotic Ectodermal DysplasiaPhase 2ECP-002NCT01775462
Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
A Phase 2 study testing EDI200 in people with X-Linked Hypohidrotic Ectodermal Dysplasia, run by Edimer Pharmaceuticals at 7 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 48 hours to 14 days, men only
- Conditions the study is for
- X-Linked Hypohidrotic Ectodermal Dysplasia
- What is being tested?
- EDI200
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 7 sitesin 5 countries
- Study dates
- Apr 2013to Dec 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 48 hours to 14 days
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Subjects for study drug administration must meet all of the following criteria to be enrolled:
- 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.
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- No major medical issues that the PI considers a contraindication to participation.
- 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):
- 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.
- No major medical issues that the investigator considers contraindications to participation.
Who cannot join
- Subjects for study drug administration who meet any of the following criteria cannot be enrolled in this study:
- 1. Medically significant postnatal complications or congenital anomalies outside of those considered to be associated with the diagnosis of XLHED.
- Siblings of subjects receiving study drug who meet any of the following criteria cannot be enrolled in the natural history sub-study:
- Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists.
- Known hypersensitivity to lidocaine or lidocaine-like agents.
- Presence of pacemaker.
- Subjects who are not able or are not willing to comply with the procedures of this protocol.
- Subject has a condition, which in the opinion of the investigator would not allow for safe conduct of the study.
Trial locations and contacts
7 sites in 5 countries.
7 sites in 5 countries
United States3 sites
University of California, San Francisco
San Francisco, California, 94143
Washington University School of Medicine
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase 2 Open-label, Dose-escalation Study to Evaluate the Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200, an EDA-A1 Replacement Protein, Administered to Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
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.
Read the 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 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.
- Being tested
- EDI200
- Keywords
- Hypohidrotic Ectodermal Dysplasia, XLHED
- Medical subject headings
- Ectodermal Dysplasia 1, Anhidrotic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EDI200Drug
Also called APO200
3 or 10 mg/kg of EDI200
| Study group | Role | What they receive |
|---|---|---|
| EDI200, 3mg/kg | Experimental | Five doses of EDI200 given at 3 mg/kg twice weekly Receives: Drug: EDI200 |
| EDI200, 10 mg/kg | Experimental | Five doses of EDI200 given at 10 mg/kg twice weekly Receives: Drug: EDI200 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events
Measured: Up to 6 months after dosing
To assess the antibody response to EDI200
Measured: Up to 6 months after dosing
Area under the concentration time curve to the end of the dosing period (AUC0-tau) of EDI200
Measured: 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
Measured: 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
Measured: 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
Secondary outcomes (8)
To assess the pharmacodynamics/efficacy (growth and development) of EDI200
Measured: Baseline and 2, 4 and 6 months
To assess the pharmacodynamics/efficacy (dentition) of EDI200
Measured: Baseline and post-six months (extension study)
To assess the pharmacodynamics/efficacy (craniofacial development) of EDI200
Measured: Baseline and 6 months
To assess the pharmacodynamics/efficacy (sweat duct density) of EDI200
Measured: Baseline and 2 and 6 months
To assess the pharmacodynamics/efficacy (sweat rate) of EDI200
Measured: Baseline and 2 and 6 months
To assess the pharmacodynamics/efficacy (Dry eye signs and symptoms) of EDI200
Measured: Baseline and 2 and 6 months
To assess the pharmacodynamics/efficacy (thermoregulation) of EDI200
Measured: Baseline and study day 21
To assess the pharmacodynamics/efficacy (molecular expression profile of skin biopsy tissue) of EDI200
Measured: Baseline, study days 1 and 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2013 (estimated)
- Study startApr 2013
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-Linked Hypohidrotic Ectodermal DysplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Edimer Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kenneth Huttner, MD, PhD, Study director, Edimer Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01775462
- Sponsor's protocol ID
- ECP-002
- Acronym
- ECP-002
- Status last verified
- Jan 2016
- First submitted
- 17 Jan 2013
- Last updated
- 20 Jan 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-label, Dose-escalation Study to Evaluate the Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200, an EDA-A1 Replacement Protein, Administered to Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
- ClinicalTrials.gov ID
- NCT01775462
- Acronym / sponsor ID
- ECP-002 / ECP-002
- Keywords
- Hypohidrotic Ectodermal Dysplasia, XLHED
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Edimer PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Apr 2013
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 25 Jan 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EDI200, 3mg/kg | Experimental | Drug: EDI200Five doses of EDI200 given at 3 mg/kg twice weekly |
| EDI200, 10 mg/kg | Experimental | Drug: EDI200Five doses of EDI200 given at 10 mg/kg twice weekly |
Who may qualify
- Ages
- 48 hours to 14 days
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- Subjects for study drug administration must meet all of the following criteria to be enrolled:
- 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.
Key exclusion criteria (8 listed)
- Subjects for study drug administration who meet any of the following criteria cannot be enrolled in this study:
- 1. Medically significant postnatal complications or congenital anomalies outside of those considered to be associated with the diagnosis of XLHED.
- Siblings of subjects receiving study drug who meet any of the following criteria cannot be enrolled in the natural history sub-study:
- Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists.
- Known hypersensitivity to lidocaine or lidocaine-like agents.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EDI200Also: APO2003 or 10 mg/kg of EDI200 | Drug | EDI200, 10 mg/kg, EDI200, 3mg/kg |
Primary outcome
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
8 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2016.
- Sponsor's websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenneth Huttner, MD, PhD study director
Edimer Pharmaceuticals
7 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EDI200 in people with X-Linked Hypohidrotic Ectodermal Dysplasia, run by Edimer Pharmaceuticals at 7 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, France, Germany, Italy, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (time frame: Up to 6 months after dosing). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2016.