Primary Sclerosing CholangitisPhase 3NCT03872921
norUrsodeoxycholic Acid vs Placebo in PSC
A Phase 3 study testing norUrsodeoxycholic Acid in people with Primary Sclerosing Cholangitis, run by Dr. Falk Pharma GmbH at 2 sites in 2 countries.
Trial summary
Double-blind, randomized, multi-center, placebo-controlled, comparative, phase III trial. The study will be conducted with two treatment groups in the form of a parallel group comparison and will serve to compare oral treatment with either 1500 mg/d norursodeoxycholic acid capsules or placebo capsules for the… Read more about this study
- Who is it for?
- Ages 16 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis
- What is being tested?
- norUrsodeoxycholic Acid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 303 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 8 Feb 2018to Jun 2026
- Phase
- Phase 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 16 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent
- verified PSC
- Liver Biopsy available for Review
- If pre-treated with UDCA Patient must be on stable dose not exceeding 20mg/kg/bw
- Patients with or without concomittant IBD
Who cannot join
- History or presence of other concomitant liver diseases
- Presence of Cholangiocarcinoma
- Secondary causes of Sclerosing Cholangitis
- Small Duct Cholangitis in the absence of large duct disease
- Any known relevant infectious disease
- Abnormal renal function
- Any active malignant disease
- Known intolerance/hypersensitivity to study drug
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
Austria1 site
Medical University of Vienna, Department of Internal Medicine III
Vienna, 1090
About this study
Official title: Double-blind, Randomized, Placebo-controlled, Phase III Study Comparing norUrsodeoxycholic Acid Capsules With Placebo in the Treatment of Primary Sclerosing Cholangitis
Double-blind, randomized, multi-center, placebo-controlled, comparative, phase III trial. The study will be conducted with two treatment groups in the form of a parallel group comparison and will serve to compare oral treatment with either 1500 mg/d norursodeoxycholic acid capsules or placebo capsules for the treatment of Primary Sclerosing Cholangitis.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- norUrsodeoxycholic Acid
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
norUrsodeoxycholic AcidDrug
Also called NUC
oral treatment
| Study group | Role | What they receive |
|---|---|---|
| norUrsodeoxycholic acid | Experimental | norUrsodeoxycholic acid 250mg capsules, 6 capsules/day for 2 years Receives: Drug: norUrsodeoxycholic Acid |
| Placebo to norUrsodeoxycholic acid | Placebo comparator | norUrsodeoxycholic acid 250mg Placebo-capsules, 6 capsules/day for 2 years Receives: Drug: norUrsodeoxycholic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC)
prevention of disease progression assessed by partial normalization of serum Alkaline Phosphatase (s-ALP) levels in patients with PSC
Measured: 2 years
Show superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC)
prevention of disease progression assessed by liver histology
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2019
- Study start8 Feb 2018
- Primary completionJan 2025 (estimated)
- Study completionJun 2026 (estimated)
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 Primary Sclerosing CholangitisStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 303 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dr. Falk Pharma GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael Trauner, MD, Principal investigator, Medical University of Vienna, Department of Internal Medicine III
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03872921
- Sponsor's protocol ID
- NUC-5/PSC
- Status last verified
- Aug 2024
- First submitted
- 8 Mar 2019
- Last updated
- 6 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: It is not yet decided which data will be made available. Neither a time Frame can be indicated yet.
Publications
Li T, Chiang JYL. Bile Acid Signaling in Metabolic and Inflammatory Diseases and Drug Development. Pharmacol Rev. 2024 Oct 16;76(6):1221-1253. doi: 10.1124/pharmrev.124.000978.
PubMed 38977324
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-blind, Randomized, Placebo-controlled, Phase III Study Comparing norUrsodeoxycholic Acid Capsules With Placebo in the Treatment of Primary Sclerosing Cholangitis
- ClinicalTrials.gov ID
- NCT03872921
- Acronym / sponsor ID
- NUC-5/PSC
- Conditions
- Primary Sclerosing Cholangitis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 303 participants (actual)
- Lead sponsor
- Dr. Falk Pharma GmbHIndustry (pharma, biotech, devices)
- Study start
- 8 Feb 2018 (actual)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Jun 2026 (estimated)
- First posted
- 13 Mar 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.
Double-blind, randomized, multi-center, placebo-controlled, comparative, phase III trial. The study will be conducted with two treatment groups in the form of a parallel group comparison and will serve to compare oral treatment with either 1500 mg/d norursodeoxycholic acid capsules or placebo capsules for the treatment of Primary Sclerosing Cholangitis.
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 |
|---|---|---|
| norUrsodeoxycholic acid | Experimental | Drug: norUrsodeoxycholic AcidnorUrsodeoxycholic acid 250mg capsules, 6 capsules/day for 2 years |
| Placebo to norUrsodeoxycholic acid | Placebo comparator | Drug: norUrsodeoxycholic AcidnorUrsodeoxycholic acid 250mg Placebo-capsules, 6 capsules/day for 2 years |
Who may qualify
- Ages
- 16 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Signed informed consent
- verified PSC
- Liver Biopsy available for Review
- If pre-treated with UDCA Patient must be on stable dose not exceeding 20mg/kg/bw
- Patients with or without concomittant IBD
Key exclusion criteria (8 listed)
- History or presence of other concomitant liver diseases
- Presence of Cholangiocarcinoma
- Secondary causes of Sclerosing Cholangitis
- Small Duct Cholangitis in the absence of large duct disease
- Any known relevant infectious disease
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 |
|---|---|---|
| norUrsodeoxycholic AcidAlso: NUCoral treatment | Drug | Placebo to norUrsodeoxycholic acid, norUrsodeoxycholic acid |
Primary outcome
superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC)
Time frame: 2 years
prevention of disease progression assessed by partial normalization of serum Alkaline Phosphatase (s-ALP) levels in patients with PSC
Show superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC)
Time frame: 2 years
prevention of disease progression assessed by liver histology
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2024.
- Derived from this study: PubMed 38977324Li T, Chiang JYL. Bile Acid Signaling in Metabolic and Inflammatory Diseases and Drug Development. Pharmacol Rev. 2024 Oct 16;76(6):1221-1253. doi: 10.1124/pharmrev.124.000978.
- Individual participant dataSharing planned: No. It is not yet decided which data will be made available. Neither a time Frame can be indicated yet.
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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.
Michael Trauner, MD principal investigator
Medical University of Vienna, Department of Internal Medicine III
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing norUrsodeoxycholic Acid in people with Primary Sclerosing Cholangitis, run by Dr. Falk Pharma GmbH at 2 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 5 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 6 Aug 2024. 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 Austria, Germany. 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 phase mean?
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?
Results are not available in the source record.
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?
superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC) (time frame: 2 years). The study lists 2 primary outcomes in all. 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 NCT03872921, 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 6 Aug 2024.