Alagille SyndromeNCT02131623
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
An observational study of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, run by Mirum Pharmaceuticals, Inc. at 5 sites in 2 countries.
Trial summary
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
- How many people
- 23 participants
- Where
- 5 sitesin 2 countries
- Study dates
- Jul 2014to Mar 2015
- 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Subjects (children) with ALGS or PFIC and/or caregivers of subjects with ALGS or PFIC
Who may be able to join
- The subject has voluntarily provided verbal assent or written assent in accordance with the site's Institutional Review Board (IRB) guidelines;
- The subject's legal guardian has voluntarily provided permission for study participation and authorization for access to personal health information in conjunction with HIPPA;
- The subject has a diagnosis of ALGS or PFIC;
- The subject is able to speak and comprehend US English (applicable only for children age 5 and older);
- The subject is willing and able to participate in the two-week validation study (applicable only for children age 5 and older); and
- The subject is able to understand the nature, scope, and possible consequences of the study, and/or does not present evidence of an uncooperative attitude (applicable only for children age 5 and older).
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- Inclusion Criteria for caregivers of subjects with ALGS or PFIC:
- The caregiver voluntarily provided written informed consent;
- The caregiver is 18 years of age or older;
- The caregiver has served as a consistent caregiver for the subject with ALGS or PFIC;
- The caregiver's child or subject with ALGS or PFIC meets all of the study inclusion criteria for subjects with ALGS or PFIC;
- The caregiver is able to speak, read, write, and comprehend US English;
- The caregiver is willing and able to participate in the two-week validation study; and
- The caregiver is able to understand the nature, scope, and possible consequences of the study, and/or does not present evidence of an uncooperative attitude.
Who cannot join
None listed.
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
United States4 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Childrens Hospital
Phoenix, Arizona, 85016-7710
St. Louis Children's Hospital
St Louis, Missouri, 63110
About this study
Official title: Validation of the ItchRO Diaries in Pediatric Cholestatic Liver Disease
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.
Read the detailed description
This research will examine the psychometric performance of the electronic diaries for the itch-reported outcome (ItchRO) measure for both patients and caregivers in a stand-alone 2-week validation study in the target patient population. The study is non-interventional in nature; the purpose of the study is to validate the ItchRO prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials. Following training and initial completion of the electronic diaries, families will return home and complete the diaries twice daily over the course of two weeks. Additional assessments include the numerical rating scale to assess itching, the patient impression of change (PIC), the caregiver impression of change (CIC), PedsQL (pediatric quality of life), children's sleep habits questionnaire (CSHQ), and clinical scratch scale.
Longitudinal data will be used to follow the natural (non-interventional) course of the disease and the quality of the data. Test-retest reliability will be assessed using diary data from Day 1 to Day 14 in stable subjects as measured by the PIC for the child-completed measure and the CIC for the caregiver measure. Convergent validity (correlations between the ItchRO and similar concepts on the other measures included in the study) will also be explored (such as the correlation between sleep and morning ItchRO diary scores). Clinical validity (correlation between the ItchRO and clinician reports) and preliminary responsiveness of those who changed in their itching status over the two week period will also be assessed if sample sizes allow.
- Medical subject headings
- Alagille Syndrome, Cholestasis, progressive familial intrahepatic 1
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 |
|---|---|---|
| Validation Group | Subjects with ALGS or PFIC and/or their caregivers |
What is being measured
The primary outcome is the main question the study is designed to answer.
Validation of ItchRO measure
Validate the ItchRO measure with subjects with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) and/or their caregivers through the completion of the electronic diary twice daily over the course of two weeks.
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2014 (estimated)
- Study startJul 2014
- Primary completionMar 2015
- Study completionMar 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- More trials run by Mirum Pharmaceuticals, Inc.The 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Mirum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02131623
- Sponsor's protocol ID
- LUM1038
- Status last verified
- Mar 2019
- First submitted
- 1 May 2014
- Last updated
- 18 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of the ItchRO Diaries in Pediatric Cholestatic Liver Disease
- ClinicalTrials.gov ID
- NCT02131623
- Acronym / sponsor ID
- LUM1038
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2014
- Primary completion
- Mar 2015 (actual)
- Study completion
- Mar 2015 (actual)
- First posted
- 6 May 2014 (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.
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.
Detailed description
This research will examine the psychometric performance of the electronic diaries for the itch-reported outcome (ItchRO) measure for both patients and caregivers in a stand-alone 2-week validation study in the target patient population. The study is non-interventional in nature; the purpose of the study is to validate the ItchRO prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials. Following training and initial completion of the electronic diaries, families will return home and complete the diaries twice daily over the course of two weeks. Additional assessments include the numerical rating scale to assess itching, the patient impression of change (PIC), the caregiver impression of change (CIC), PedsQL (pediatric quality of life), children's sleep habits questionnaire (CSHQ), and clinical scratch scale.
Longitudinal data will be used to follow the natural (non-interventional) course of the disease and the quality of the data. Test-retest reliability will be assessed using diary data from Day 1 to Day 14 in stable subjects as measured by the PIC for the child-completed measure and the CIC for the caregiver measure. Convergent validity (correlations between the ItchRO and similar concepts on the other measures included in the study) will also be explored (such as the correlation between sleep and morning ItchRO diary scores). Clinical validity (correlation between the ItchRO and clinician reports) and preliminary responsiveness of those who changed in their itching status over the two week period will also be assessed if sample sizes allow.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Validation Group | – | –Subjects with ALGS or PFIC and/or their caregivers |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Subjects (children) with ALGS or PFIC and/or caregivers of subjects with ALGS or PFIC
Key inclusion criteria (14 listed)
- The subject has voluntarily provided verbal assent or written assent in accordance with the site's Institutional Review Board (IRB) guidelines;
- The subject's legal guardian has voluntarily provided permission for study participation and authorization for access to personal health information in conjunction with HIPPA;
- The subject has a diagnosis of ALGS or PFIC;
- The subject is able to speak and comprehend US English (applicable only for children age 5 and older);
- The subject is willing and able to participate in the two-week validation study (applicable only for children age 5 and older); and
Key exclusion criteria (0 listed)
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
Validation of ItchRO measure
Time frame: 2 weeks
Validate the ItchRO measure with subjects with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC) and/or their caregivers through the completion of the electronic diary twice daily over the course of two weeks.
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 18 Mar 2019.
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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.
Study Director study director
Mirum
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, run by Mirum Pharmaceuticals, Inc. at 5 sites in 2 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 14 inclusion and 0 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 18 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Canada. 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 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?
Validation of ItchRO measure (time frame: 2 weeks). 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 18 Mar 2019.