Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia

Launched by EDIMER PHARMACEUTICALS · Mar 26, 2014

Keywords

ClinConnect Summary

Important to the development and regulatory approval of therapies for XLHED will be the collection of data on the clinical history and prospective health of those affected by XLHED. The proposed natural history study will enroll male and female patients, ages 36 months and younger, who have a diagnosis of XLHED based on genetic testing and who have not received an investigational study drug. The study protocol will include collection of all relevant medical history and documentation of clinical outcomes using age-appropriate, minimally invasive technologies. Data will be collected both retr...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• Subjects must meet all of the following criteria to be enrolled in this study:
• • 1. Confirmed genetic diagnosis of XLHED
• • 2. Written informed consent of both parents (if reasonably available)
• Exclusion Criteria:
• Subjects who meet any of the following criteria cannot be enrolled in this study:
• • 1. Medically-significant complications or congenital anomalies outside of those considered to be associated with the diagnosis or status of XLHED
• • 2. Having received an investigational study drug prior to enrollment. For subjects less than 6 months of age, the mother cannot have taken an investigational drug during her pregnancy.
• • 3. Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists
• • 4. Presence of pacemakers