CholestasisPhase 2/3URSONEONATNCT00846963
Ursodiol for Treating Parenteral Nutrition Associated Cholestasis in Neonates
A Phase 2/3 study testing Ursodiol in people with Cholestasis, run by Ibrahim Mohamed at 1 site in Canada.
Trial summary
The purpose of this study is to determine whether ursodiol is effective in the treatment of parenteral nutrition associated cholestasis in neonates. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cholestasis
- What is being tested?
- Ursodiol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 1 siteCanada
- Study dates
- Oct 2008to Mar 2013
- Phase
- Phase 2/3
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Preterm or in-term newborns hospitalized in neonatal care units at CHU Sainte-Justine between October 1st 2008 and October 1st 2011.
- Must be receiving parenteral nutrition (either partial or total) at the diagnosis of cholestasis.
- Parental Consent must be obtained.
Who cannot join
- Active urinary tract infection
- Presence of clinical signs(acholic stool) of or ultrasound evidence of biliary tract anomalies.
- Positive TORCH infections(Toxoplasmosis, Other infections, Rubella, Cytomegalovirus, Herpes simplex virus)
- Known short bowel syndrome
- Known congenital hypothyroidism
- Known genetic disorders associated with cholestasis like galactosemia, phenylcytonuria, antitrypsin 1 deficiency... etc
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this study is to determine whether ursodiol is effective in the treatment of parenteral nutrition associated cholestasis in neonates.
Read the detailed description
This is the first randomised controlled study that address the question of the role of ursodiol as treatment of cases of PNAC.
It includes all neonates with stratification of less than and equal to 32 weeks or more than 32 weeks of gestation.
- Conditions
- Cholestasis
- Being tested
- Ursodiol, placebo
- Keywords
- parenteral nutrition associated cholestasis in neonates, parenteral nutrition induced cholestasis in preterms, Bile duct obstruction, biliary stasis
- Medical subject headings
- Cholestasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UrsodiolDrug
Also called Urso, ursodeoxycholic acid
Ursodiol is given by mouth, three times a day from second value of elevated conjugated bilirubin (>33mmol/L) to the resolution of cholestasis (conjugated bilirubin <34mmol/L) If Nil per os, 3,3mg/kg/dose is given. If Nil per os is required (e.g. pre-surgery, or necrotizing enterocolitis), none is given.
If enteral feeding is under 100mL/kg/day, 6,7 mg/kg/day is given. If enteral feeding exceeds 100mL/kg/day, 10 mg/kg/day is given.
placeboDrug
Placebo given in the same amount that ursodiol would be given, depending on enteral feeding and weight. It is also given three times a day, until cholestasis resolution.
| Study group | Role | What they receive |
|---|---|---|
| Ursodiol | Experimental | Participants assigned in this arm receive an ursodiol suspension at 20mg/ml. Receives: Drug: Ursodiol |
| placebo | Placebo comparator | A placebo suspension that looks like the ursodiol suspension used. Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Length of parenteral nutrition associated cholestasis (in days)
Measured: at the end of cholestasis (when conjugated bilirubin < 34 mmol/L) average of 4 weeks.
Secondary outcomes (5)
Peak value of biomarkers associated with cholestasis (Gamma-glutamyl transpeptidase, Alkaline phosphatase, conjugated bilirubin)
Measured: at least once a week, during cholestasis
1- Other hepatic marker (Aspartate transaminase, alanine transaminase, albumin blood level)
Measured: at least once a week, during cholestasis
Length required to minimal enteral feeding (120mL/kg/day) measured in days.
Measured: From birth to outcome (usually less than 21 days)
Weight gain (in g/kg/day)
Measured: From birth to resolution of cholestasis (very varuiable but usually less than 3 months)
Adverse effects linked to ursodiol
Measured: From beginning to the end of the medication (average 4 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2009 (estimated)
- Study startOct 2008
- Primary completionDec 2012
- Study completionMar 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ibrahim Mohamed
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ibrahim Mohamed, St. Justine's Hospital
- Investigators
- Ibrahim Mohamed, MB ChB, DIS P, Principal investigator, St. Justine's HospitalJosianne Malo, B.Pharm, M.Sc., Study director, St. Justine's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00846963
- Sponsor's protocol ID
- RC:127
- Acronym
- URSONEONAT
- Status last verified
- Sep 2013
- First submitted
- 17 Feb 2009
- Last updated
- 17 Sep 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
Al-Hathlol K, Al-Madani A, Al-Saif S, Abulaimoun B, Al-Tawil K, El-Demerdash A. Ursodeoxycholic acid therapy for intractable total parenteral nutrition-associated cholestasis in surgical very low birth weight infants. Singapore Med J. 2006 Feb;47(2):147-51.
PubMed 16435058Arslanoglu S, Moro GE, Tauschel HD, Boehm G. Ursodeoxycholic acid treatment in preterm infants: a pilot study for the prevention of cholestasis associated with total parenteral nutrition. J Pediatr Gastroenterol Nutr. 2008 Feb;46(2):228-31. doi: 10.1097/MPG.0b013e3181560524.
PubMed 18223390Chen CY, Tsao PN, Chen HL, Chou HC, Hsieh WS, Chang MH. Ursodeoxycholic acid (UDCA) therapy in very-low-birth-weight infants with parenteral nutrition-associated cholestasis. J Pediatr. 2004 Sep;145(3):317-21. doi: 10.1016/j.jpeds.2004.05.038.
PubMed 15343182Venigalla S, Gourley GR. Neonatal cholestasis. Semin Perinatol. 2004 Oct;28(5):348-55. doi: 10.1053/j.semperi.2004.09.008.
PubMed 15686266McKiernan PJ. Neonatal cholestasis. Semin Neonatol. 2002 Apr;7(2):153-65. doi: 10.1053/siny.2002.0103.
PubMed 12208100
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ursodiol for Treating Parenteral Nutrition Associated Cholestasis in Neonates
- ClinicalTrials.gov ID
- NCT00846963
- Acronym / sponsor ID
- URSONEONAT / RC:127
- Conditions
- Cholestasis
- Keywords
- parenteral nutrition associated cholestasis in neonates, parenteral nutrition induced cholestasis in preterms, Bile duct obstruction, biliary stasis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Ibrahim MohamedUniversity, hospital or other
- Study start
- Oct 2008
- Primary completion
- Dec 2012 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 19 Feb 2009 (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 this study is to determine whether ursodiol is effective in the treatment of parenteral nutrition associated cholestasis in neonates.
Detailed description
This is the first randomised controlled study that address the question of the role of ursodiol as treatment of cases of PNAC.
It includes all neonates with stratification of less than and equal to 32 weeks or more than 32 weeks of gestation.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ursodiol | Experimental | Drug: UrsodiolParticipants assigned in this arm receive an ursodiol suspension at 20mg/ml. |
| placebo | Placebo comparator | Drug: placeboA placebo suspension that looks like the ursodiol suspension used. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Preterm or in-term newborns hospitalized in neonatal care units at CHU Sainte-Justine between October 1st 2008 and October 1st 2011.
- Must be receiving parenteral nutrition (either partial or total) at the diagnosis of cholestasis.
- Parental Consent must be obtained.
Key exclusion criteria (6 listed)
- Active urinary tract infection
- Presence of clinical signs(acholic stool) of or ultrasound evidence of biliary tract anomalies.
- Positive TORCH infections(Toxoplasmosis, Other infections, Rubella, Cytomegalovirus, Herpes simplex virus)
- Known short bowel syndrome
- Known congenital hypothyroidism
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 |
|---|---|---|
| UrsodiolAlso: Urso, ursodeoxycholic acidUrsodiol is given by mouth, three times a day from second value of elevated conjugated bilirubin (>33mmol/L) to the resolution of cholestasis (conjugated bilirubin <34mmol/L) If Nil per os, 3,3mg/kg/dose is given. If Nil per os is required (e.g. pre-surgery, or necrotizing enterocolitis), none is given. If enteral feeding is under 100mL/kg/day, 6,7 mg/kg/day is given. If enteral feeding exceeds 100mL/kg/day, 10 mg/kg/day is given. | Drug | Ursodiol |
| placeboPlacebo given in the same amount that ursodiol would be given, depending on enteral feeding and weight. It is also given three times a day, until cholestasis resolution. | Drug | placebo |
Primary outcome
Length of parenteral nutrition associated cholestasis (in days)
Time frame: at the end of cholestasis (when conjugated bilirubin < 34 mmol/L) average of 4 weeks.
5 secondary outcomes
Peak value of biomarkers associated with cholestasis (Gamma-glutamyl transpeptidase, Alkaline phosphatase, conjugated bilirubin)
Time frame: at least once a week, during cholestasis
1- Other hepatic marker (Aspartate transaminase, alanine transaminase, albumin blood level)
Time frame: at least once a week, during cholestasis
Length required to minimal enteral feeding (120mL/kg/day) measured in days.
Time frame: From birth to outcome (usually less than 21 days)
Weight gain (in g/kg/day)
Time frame: From birth to resolution of cholestasis (very varuiable but usually less than 3 months)
Adverse effects linked to ursodiol
Time frame: From beginning to the end of the medication (average 4 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 17 Sep 2013.
- Background: PubMed 16435058Al-Hathlol K, Al-Madani A, Al-Saif S, Abulaimoun B, Al-Tawil K, El-Demerdash A. Ursodeoxycholic acid therapy for intractable total parenteral nutrition-associated cholestasis in surgical very low birth weight infants. Singapore Med J. 2006 Feb;47(2):147-51.
- Background: PubMed 18223390Arslanoglu S, Moro GE, Tauschel HD, Boehm G. Ursodeoxycholic acid treatment in preterm infants: a pilot study for the prevention of cholestasis associated with total parenteral nutrition. J Pediatr Gastroenterol Nutr. 2008 Feb;46(2):228-31. doi: 10.1097/MPG.0b013e3181560524.
- Background: PubMed 15343182Chen CY, Tsao PN, Chen HL, Chou HC, Hsieh WS, Chang MH. Ursodeoxycholic acid (UDCA) therapy in very-low-birth-weight infants with parenteral nutrition-associated cholestasis. J Pediatr. 2004 Sep;145(3):317-21. doi: 10.1016/j.jpeds.2004.05.038.
- Background: PubMed 15686266Venigalla S, Gourley GR. Neonatal cholestasis. Semin Perinatol. 2004 Oct;28(5):348-55. doi: 10.1053/j.semperi.2004.09.008.
- Background: PubMed 12208100McKiernan PJ. Neonatal cholestasis. Semin Neonatol. 2002 Apr;7(2):153-65. doi: 10.1053/siny.2002.0103.
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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.
Ibrahim Mohamed, MB ChB, DIS P principal investigator
St. Justine's Hospital
Josianne Malo, B.Pharm, M.Sc. study director
St. Justine's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Ursodiol in people with Cholestasis, run by Ibrahim Mohamed at 1 site in Canada.
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 6 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 Sep 2013. 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 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Length of parenteral nutrition associated cholestasis (in days) (time frame: at the end of cholestasis (when conjugated bilirubin < 34 mmol/L) average of 4 weeks.). 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 NCT00846963, which also lists 5 linked publications.
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 Sep 2013.