Chronic CholestasisPhase 2NCT03586674
Fibrates in Pediatric Cholestasis
A Phase 2 study testing Lipanthyl and Ursogal in people with Chronic Cholestasis, run by Hoda A. Atta at 2 sites in Egypt.
Trial summary
A study conducted to assess the effect of fibrates on pruritus and biochemical picture in pediatric patients with cholestatic liver diseases. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Chronic Cholestasis
- What is being tested?
- Lipanthyl and Ursogal
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 2 sitesEgypt
- Study dates
- 1 Nov 2017to 20 Jun 2018
- 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 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient with chronic cholestatic liver disease defined as any condition in which substances normally excreted into bile are retained for more than 6 months.
Who cannot join
- Patients with anatomical or mechanical obstructive causes for cholestasis.
- Cholestatic patients who were suffering from another liver disease.
- Cholestatic patients who were receiving drugs affecting lipid profile.
- Patients receiving drugs that interact with Fenofibrate (FF) e.g statins and warfarin
- Patients with non obstructive gall bladder stones were excluded from T gp.
Trial locations and contacts
2 sites in 1 country.
Egypt2 sites
Dr. Yassin Abdel Ghaffar Charity Center For Liver Diseases & Researches
Cairo, Nasr City
National liver istitute
Shibīn al Kawm
About this study
Official title: Fibrates: An Adjuvant Therapy for Cholestasis In Pediatric Age Group
A study conducted to assess the effect of fibrates on pruritus and biochemical picture in pediatric patients with cholestatic liver diseases.
Read the detailed description
Cholestatic liver disorders include a spectrum of hepatobiliary diseases of diverse etiologies that are characterized by impaired hepatocellular secretion of bile, resulting in accumulation of bile acids, bilirubin and cholesterol.This could result in different clinical features including pruritus, malabsorption and vitamin deficiencies with subsequent coagulation disorders and bone disease. Persistence of cholestasis leads to biliary fibrosis which can progress to liver cirrhosis and end-stage liver disease.
Nuclear receptors (NRs) regulate ligand-activated transcription factor networks of genes for the elimination and detoxification of potentially toxic biliary constituents accumulating in cholestasis. Activation of several NRs also modulates fibrogenesis, inflammation, and carcinogenesis as sequelae of cholestasis. Hence, It represent attractive targets for pharmacotherapy of cholestatic disorders.
Several already available drugs may exert their beneficial effects in cholestasis via NR activation eg, ursodeoxycholic acid via glucocorticoid receptor and pregnane X receptor, and rifampicin via pregnane X receptor. Unfortunately, Some patients may not respond to these medications.
Fibrates, serum Lipid lowering medication, has a stimulation action on proliferator activated receptor alpha. It is a nuclear receptor with an integral role in bile homeostasis. Several case reports and pilot studies have demonstrated the efficacy of fibrates in reducing serum biomarkers of cholestasis and liver function abnormalities in patients with incomplete response to ursodeoxycholic acid monotherapy. These results are of interest, because fibrates are attracting increased attention as adjunct therapy for chronic cholestatic liver diseases.
- Conditions
- Chronic Cholestasis
- Being tested
- Lipanthyl, Ursogal
- Keywords
- pediatric cholestasis, Fibrate
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LipanthylDrug
Also called ursogal
Tablet
UrsogalDrug
Also called non
suspension
| Study group | Role | What they receive |
|---|---|---|
| Ursogal | Active comparator | Control group : Ursogal 10-20 mg/kg/d on 2 divided dose for four months with regular follow up. Receives: Drug: Ursogal |
| Lipanthyl + Ursogal | Experimental | Therapy group: Ursogal 10-20 mg/kg/d by mouth, on 2 divided dose, and lipanthyl 10-20 mg/kg/d by mouth,once per day, for four months with regular follow up. Receives: Drug: Lipanthyl; Drug: Ursogal |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the pruritus grading score
The pruritus grading score includes four areas each has its score: distribution score 1-3;1= single site and 3 generalized, Severity score 1-5 ; 1= rubbing,5 = general excoriation, Frequency score 1-5; 1= episodic,5= continuous, and Sleep disturbance score 0-6 ; 0= no effect on sleep, 6= total restless.
Measured: four months
Secondary outcomes (1)
Changes in the liver function test and lipid profile
investigate the effect on Alanine Aminotransferase (ALT),Aspartate Aminotransferase (AST) ,Albumin,Bilirubin, Bile acid, lipid profile
Measured: four months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2018
- Study start1 Nov 2017
- Primary completion1 Jun 2018
- Study completion20 Jun 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic CholestasisStudies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoda A. Atta
- Sponsor type
- Other government
- Responsible party
- Sponsor-investigator, Hoda A. Atta, Ministry of Health and Population, Egypt
- Investigators
- Tawhida Y Abdel Ghaffar, MD, Study director, ASU
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03586674
- Sponsor's protocol ID
- 1984
- Status last verified
- Jul 2018
- First submitted
- 2 Jul 2018
- Last updated
- 17 Jul 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Only overall results will be shared.
Publications
Ghonem NS, Boyer JL. Fibrates as adjuvant therapy for chronic cholestatic liver disease: its time has come. Hepatology. 2013 May;57(5):1691-3. doi: 10.1002/hep.26155. Epub 2013 Apr 5. No abstract available.
PubMed 23174993
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fibrates: An Adjuvant Therapy for Cholestasis In Pediatric Age Group
- ClinicalTrials.gov ID
- NCT03586674
- Acronym / sponsor ID
- 1984
- Conditions
- Chronic Cholestasis
- Keywords
- pediatric cholestasis, Fibrate
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Hoda A. AttaOther government
- Study start
- 1 Nov 2017 (actual)
- Primary completion
- 1 Jun 2018 (actual)
- Study completion
- 20 Jun 2018 (actual)
- First posted
- 13 Jul 2018 (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.
A study conducted to assess the effect of fibrates on pruritus and biochemical picture in pediatric patients with cholestatic liver diseases.
Detailed description
Cholestatic liver disorders include a spectrum of hepatobiliary diseases of diverse etiologies that are characterized by impaired hepatocellular secretion of bile, resulting in accumulation of bile acids, bilirubin and cholesterol.This could result in different clinical features including pruritus, malabsorption and vitamin deficiencies with subsequent coagulation disorders and bone disease. Persistence of cholestasis leads to biliary fibrosis which can progress to liver cirrhosis and end-stage liver disease.
Nuclear receptors (NRs) regulate ligand-activated transcription factor networks of genes for the elimination and detoxification of potentially toxic biliary constituents accumulating in cholestasis. Activation of several NRs also modulates fibrogenesis, inflammation, and carcinogenesis as sequelae of cholestasis. Hence, It represent attractive targets for pharmacotherapy of cholestatic disorders.
Several already available drugs may exert their beneficial effects in cholestasis via NR activation eg, ursodeoxycholic acid via glucocorticoid receptor and pregnane X receptor, and rifampicin via pregnane X receptor. Unfortunately, Some patients may not respond to these medications.
Fibrates, serum Lipid lowering medication, has a stimulation action on proliferator activated receptor alpha. It is a nuclear receptor with an integral role in bile homeostasis. Several case reports and pilot studies have demonstrated the efficacy of fibrates in reducing serum biomarkers of cholestasis and liver function abnormalities in patients with incomplete response to ursodeoxycholic acid monotherapy. These results are of interest, because fibrates are attracting increased attention as adjunct therapy for chronic cholestatic liver diseases.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ursogal | Active comparator | Drug: UrsogalControl group : Ursogal 10-20 mg/kg/d on 2 divided dose for four months with regular follow up. |
| Lipanthyl + Ursogal | Experimental | Drug: Lipanthyl, Drug: UrsogalTherapy group: Ursogal 10-20 mg/kg/d by mouth, on 2 divided dose, and lipanthyl 10-20 mg/kg/d by mouth,once per day, for four months with regular follow up. |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Patient with chronic cholestatic liver disease defined as any condition in which substances normally excreted into bile are retained for more than 6 months.
Key exclusion criteria (5 listed)
- Patients with anatomical or mechanical obstructive causes for cholestasis.
- Cholestatic patients who were suffering from another liver disease.
- Cholestatic patients who were receiving drugs affecting lipid profile.
- Patients receiving drugs that interact with Fenofibrate (FF) e.g statins and warfarin
- Patients with non obstructive gall bladder stones were excluded from T gp.
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 |
|---|---|---|
| LipanthylAlso: ursogalTablet | Drug | Lipanthyl + Ursogal |
| UrsogalAlso: nonsuspension | Drug | Lipanthyl + Ursogal, Ursogal |
Primary outcome
Change in the pruritus grading score
Time frame: four months
The pruritus grading score includes four areas each has its score: distribution score 1-3;1= single site and 3 generalized, Severity score 1-5 ; 1= rubbing,5 = general excoriation, Frequency score 1-5; 1= episodic,5= continuous, and Sleep disturbance score 0-6 ; 0= no effect on sleep, 6= total restless.
1 secondary outcome
Changes in the liver function test and lipid profile
Time frame: four months
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 17 Jul 2018.
- Background: PubMed 23174993Ghonem NS, Boyer JL. Fibrates as adjuvant therapy for chronic cholestatic liver disease: its time has come. Hepatology. 2013 May;57(5):1691-3. doi: 10.1002/hep.26155. Epub 2013 Apr 5. No abstract available.
- Review articleLink provided by the sponsor.
- Individual participant dataSharing planned: No. Only overall results will be shared.
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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.
Tawhida Y Abdel Ghaffar, MD study director
ASU
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Lipanthyl and Ursogal in people with Chronic Cholestasis, run by Hoda A. Atta at 2 sites in Egypt.
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 1 inclusion and 5 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 17 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Egypt. 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.
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 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?
Change in the pruritus grading score (time frame: four months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03586674, which also lists 1 linked publication.
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 17 Jul 2018.