XLHEDNCT03912792
A Cross-Sectional Natural History Study to Evaluate Sweat Volume and Other Phenotypic and Genetic Characteristics in Patients Affected by XLHED
An observational study of XLHED, run by Dermelix Biotherapeutics, LLC. at 1 site in United States.
Trial summary
The proposed natural history study will enroll male patients with a diagnosis of XLHED, female carriers of XLHED and healthy volunteers. The study protocol will include collection of XLHED questionnaires and clinical outcomes using minimally invasive technologies. Read more about this study
- Who is it for?
- Ages Up to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- XLHED
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Jun 2019to Sep 2019
- Phase
- Observational
- 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 Up to 80 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Males with XLHED and female carriers of XLHED Male and female healthy volunteers
Who may be able to join
- Informed Consent
- Males with XLHED, female carriers of XLHED and healthy volunteers
Who cannot join
- Treatment with an investigational study drug for XLHED
- Any major medical problems that would prevent her/him from participating in this study
- Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists (Examples: Urecholine, Salagen, Pilocar, and Provocholine)
- Pacemaker
Trial locations and contacts
1 site in 1 country.
United States1 site
Chicago Marriott Lincolnshire Resort
Lincolnshire, Illinois, 60069
About this study
Official title: A Cross-Sectional Natural History Study to Evaluate Sweat Volume and Other Phenotypic and Genetic Characteristics in Patients Affected by X-Linked Hypohidrotic XLHED Ectodermal Dysplasia (XLHED)
The proposed natural history study will enroll male patients with a diagnosis of XLHED, female carriers of XLHED and healthy volunteers. The study protocol will include collection of XLHED questionnaires and clinical outcomes using minimally invasive technologies. Data will be collected both retrospectively and prospectively.
Clinical outcome assessments will be performed at the NFED Family Conference on July 11-12, 2019. Study participants will be able to complete XLHED questionnaires electronically ahead of the conference.
- Conditions
- XLHED
- Keywords
- Natural History Study, X-Linked Hypohidrotic Ectodermal Dysplasia
- 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.
| Study group | Role | What they receive |
|---|---|---|
| XLHED Patients | Not described | |
| Healthy Controls | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
The volume of sweat from a pilocarpine-induced sweat test
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2019
- Study start28 Jun 2019
- Primary completionSep 2019 (estimated)
- Study completionSep 2019 (estimated)
Why it stopped: Company decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for XLHEDStudies 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
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dermelix Biotherapeutics, LLC.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dorothy K Grange, MD, Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03912792
- Sponsor's protocol ID
- DMX101-0001
- Status last verified
- Jun 2019
- First submitted
- 10 Apr 2019
- Last updated
- 2 Jul 2019
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Cross-Sectional Natural History Study to Evaluate Sweat Volume and Other Phenotypic and Genetic Characteristics in Patients Affected by X-Linked Hypohidrotic XLHED Ectodermal Dysplasia (XLHED)
- ClinicalTrials.gov ID
- NCT03912792
- Acronym / sponsor ID
- DMX101-0001
- Conditions
- XLHED
- Keywords
- Natural History Study, X-Linked Hypohidrotic Ectodermal Dysplasia
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Company decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Dermelix Biotherapeutics, LLC.Industry (pharma, biotech, devices)
- Study start
- 28 Jun 2019 (actual)
- Primary completion
- Sep 2019 (estimated)
- Study completion
- Sep 2019 (estimated)
- First posted
- 11 Apr 2019 (actual)
- 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.
The proposed natural history study will enroll male patients with a diagnosis of XLHED, female carriers of XLHED and healthy volunteers. The study protocol will include collection of XLHED questionnaires and clinical outcomes using minimally invasive technologies. Data will be collected both retrospectively and prospectively.
Clinical outcome assessments will be performed at the NFED Family Conference on July 11-12, 2019. Study participants will be able to complete XLHED questionnaires electronically ahead of the conference.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| XLHED Patients | – | – |
| Healthy Controls | – | – |
Who may qualify
- Ages
- Up to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Males with XLHED and female carriers of XLHED Male and female healthy volunteers
Key inclusion criteria (2 listed)
- Informed Consent
- Males with XLHED, female carriers of XLHED and healthy volunteers
Key exclusion criteria (4 listed)
- Treatment with an investigational study drug for XLHED
- Any major medical problems that would prevent her/him from participating in this study
- Known hypersensitivity to pilocarpine or pilocarpine-like muscarinic agonists (Examples: Urecholine, Salagen, Pilocar, and Provocholine)
- Pacemaker
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The volume of sweat from a pilocarpine-induced sweat test
Time frame: Day 1
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2019.
Related trials for XLHED
RecruitingPhase 2
Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
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.
Dorothy K Grange, MD principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of XLHED, run by Dermelix Biotherapeutics, LLC. at 1 site in United States.
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 2 inclusion and 4 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: withdrawn, as of the sponsor's last update on 2 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
The volume of sweat from a pilocarpine-induced sweat test (time frame: Day 1). 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 2 Jul 2019.