Syndromic IchthyosesPhase 2NCT01110642
Novel Treatment for Syndromic Ichthyoses
A Phase 2 study testing Lovastatin in people with Syndromic Ichthyoses and CHILD Syndrome, run by Northwestern University.
Trial summary
This is an open label-pilot study to assess the efficacy and safety of a novel cholesterol-lovastatin topical solution in children with rare syndromic ichthyoses. Often times, these children have difficulty in finding easily applied treatments to make their psoriasiform and ichthyotic plaques more manageable. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Syndromic Ichthyoses, CHILD Syndrome, Smith Lemli Opitz Syndrome
- What is being tested?
- Lovastatin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jul 2011to not given
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Informed Consent form signed by the subject or subject's legal representative; also, if the subject is under the age of majority but capable of providing assent, signed assent from the subject.
- Diagnosis of a syndromic ichthyoses (verified by Dr Amy S. Paller).
- Children and adults 12 months of age and above
Who cannot join
- Use of any investigational drug within the 30 days before enrollment.
- Current malignancy.
- Pregnancy or breastfeeding during the study. (All female subjects of childbearing potential will be assessed for pregnancy at all visits.)
About this study
Official title: Open-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic Ichthyoses
This is an open label-pilot study to assess the efficacy and safety of a novel cholesterol-lovastatin topical solution in children with rare syndromic ichthyoses. Often times, these children have difficulty in finding easily applied treatments to make their psoriasiform and ichthyotic plaques more manageable. We propose the use of a cholesterol-lovastatin topical solution as a treatment option with the hypothesis that it will lead to regression of involved areas and decreased erythema and warty-like appearance of the plaques. We plan to enroll children with syndromic ichthyoses over the age of 1 year for a 12 month study with a total of 5 visits and 5 phone calls.
- Being tested
- Lovastatin
- Medical subject headings
- Congenital Hemidysplasia with Ichthyosiform Erythroderma and Limb Defects, Smith-Lemli-Opitz Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LovastatinDrug
Topical lovastatin applied to red, rashy areas two times daily for 12 months
| Study group | Role | What they receive |
|---|---|---|
| Lovastatin solution | Experimental | All patients will receive lovastatin solution Receives: Drug: Lovastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Physician global assessment of severity (PGAS)
Difference in physician global assessment of severity at baseline compared to month 12
Measured: 12 months
Secondary outcomes (1)
Total body surface area (TBSA)
Percent change in TBSA affected at baseline compared to at month 12
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2010 (estimated)
- Study startJul 2011
Why it stopped: Study was withdrawn due to lack of eligible population for study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Syndromic IchthyosesStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amy Paller, Northwestern University
- Investigators
- Amy S Paller, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01110642
- Sponsor's protocol ID
- 2010-04-15
- Status last verified
- May 2015
- First submitted
- 22 Apr 2010
- Last updated
- 19 May 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic Ichthyoses
- ClinicalTrials.gov ID
- NCT01110642
- Acronym / sponsor ID
- 2010-04-15
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study was withdrawn due to lack of eligible population for study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Study start
- Jul 2011
- First posted
- 26 Apr 2010 (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 is an open label-pilot study to assess the efficacy and safety of a novel cholesterol-lovastatin topical solution in children with rare syndromic ichthyoses. Often times, these children have difficulty in finding easily applied treatments to make their psoriasiform and ichthyotic plaques more manageable. We propose the use of a cholesterol-lovastatin topical solution as a treatment option with the hypothesis that it will lead to regression of involved areas and decreased erythema and warty-like appearance of the plaques. We plan to enroll children with syndromic ichthyoses over the age of 1 year for a 12 month study with a total of 5 visits and 5 phone calls.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lovastatin solution | Experimental | Drug: LovastatinAll patients will receive lovastatin solution |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Informed Consent form signed by the subject or subject's legal representative; also, if the subject is under the age of majority but capable of providing assent, signed assent from the subject.
- Diagnosis of a syndromic ichthyoses (verified by Dr Amy S. Paller).
- Children and adults 12 months of age and above
Key exclusion criteria (3 listed)
- Use of any investigational drug within the 30 days before enrollment.
- Current malignancy.
- Pregnancy or breastfeeding during the study. (All female subjects of childbearing potential will be assessed for pregnancy at all visits.)
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 |
|---|---|---|
| LovastatinTopical lovastatin applied to red, rashy areas two times daily for 12 months | Drug | Lovastatin solution |
Primary outcome
Physician global assessment of severity (PGAS)
Time frame: 12 months
Difference in physician global assessment of severity at baseline compared to month 12
1 secondary outcome
Total body surface area (TBSA)
Time frame: 12 months
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 19 May 2015.
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.
Amy S Paller, MD principal investigator
Northwestern University
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lovastatin in people with Syndromic Ichthyoses and CHILD Syndrome, run by Northwestern University.
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 3 inclusion and 3 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 19 May 2015. Status can change between updates, so confirm with the study contact before making plans.
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 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?
Physician global assessment of severity (PGAS) (time frame: 12 months). 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 19 May 2015.