Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (ECP-002)
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ClinicalTrials.gov Identifier: NCT01775462 |
Recruitment Status :
Completed
First Posted : January 25, 2013
Last Update Posted : January 20, 2016
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
X-Linked Hypohidrotic Ectodermal Dysplasia | Drug: EDI200 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 6 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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) |
Study Start Date : | April 2013 |
Actual Primary Completion Date : | December 2015 |
Actual Study Completion Date : | December 2015 |

Arm | Intervention/treatment |
---|---|
Experimental: EDI200, 3mg/kg
Five doses of EDI200 given at 3 mg/kg twice weekly
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Drug: EDI200
3 or 10 mg/kg of EDI200
Other Name: APO200 |
Experimental: EDI200, 10 mg/kg
Five doses of EDI200 given at 10 mg/kg twice weekly
|
Drug: EDI200
3 or 10 mg/kg of EDI200
Other Name: 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 ]

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Ages Eligible for Study: | 48 Hours to 14 Days (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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.
- 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.
Exclusion Criteria:
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01775462
United States, California | |
University of California, San Francisco | |
San Francisco, California, United States, 94143 | |
United States, District of Columbia | |
Children's National Medical Center | |
Washington, District of Columbia, United States, 20010 | |
United States, Missouri | |
Washington University School of Medicine | |
St. Louis, Missouri, United States, 63110 | |
France | |
Hôpital Necker-Enfants Malades | |
Paris, France, 75015 | |
Germany | |
University Hospital Erlangen | |
Erlangen, Bavaria, Germany, 91054 | |
Italy | |
Azienda Ospedaliera-Polo Universitario "Luigi Sacco" | |
Milan, Italy, 20157 | |
United Kingdom | |
University Hospital of Wales | |
Cardiff, United Kingdom, CF14 4XW |
Study Director: | Kenneth Huttner, MD, PhD | Edimer Pharmaceuticals |
Responsible Party: | Edimer Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT01775462 |
Other Study ID Numbers: |
ECP-002 |
First Posted: | January 25, 2013 Key Record Dates |
Last Update Posted: | January 20, 2016 |
Last Verified: | January 2016 |
Hypohidrotic Ectodermal Dysplasia XLHED |
Ectodermal Dysplasia Ectodermal Dysplasia 1, Anhidrotic Hyperplasia Pathologic Processes Abnormalities, Multiple Congenital Abnormalities |
Skin Abnormalities Skin Diseases, Genetic Genetic Diseases, Inborn Skin Diseases Genetic Diseases, X-Linked |