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Primary Biliary CholangitisPhase 2NCT03394924

A Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of EDP-305 in Subjects With Primary Biliary Cholangitis

A Phase 2 study testing EDP-305 1 mg and EDP-305 2.5 mg in people with Primary Biliary Cholangitis, run by Enanta Pharmaceuticals, Inc at 86 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

A randomized, double-blind study to assess the safety, tolerability, PK and efficacy of EDP-305 in subjects with primary biliary cholangitis Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Primary Biliary Cholangitis
What is being tested?
EDP-305 1 mg and EDP-305 2.5 mg
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
68 participants
Where
86 sitesin 10 countries
Study dates
27 Dec 2017to 16 Jan 2020
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • An informed consent document signed and dated by the subject.
  • Male and female subjects of any ethnic origin between the ages of 18 and 75 years, inclusive
  • Male or female with a diagnosis of PBC by at least two of the following criteria:
  • History of ALP above ULN for at least six months
  • Positive Anti-Mitochondrial Antibodies (AMA) titers (>1/40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies)
  • For subjects with no documented liver biopsy performed within 2 years, subjects must undergo a transient elastography (Fibroscan) showing liver stiffness < 14.0 kPA
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  • Must be on a stable dose of UDCA12-20 mg/kg/day for at least 6 months prior to Screening or intolerant of UDCA in the opinion of the Investigator (no UDCA for at least 12 weeks prior to Screening)
  • Alkaline Phosphatase (ALP) ≥ 1.67 × ULN and/or total bilirubin >ULN but < 2×ULN (<2.4 mg/dL)
  • Subjects must have Screening laboratory values for Hepatitis B surface antigen (HBsAg), anti-HCV antibodies and HCV RNA negative and Human Immunodeficiency Virus (HIV) 1 and 2 antibodies (Ab) as seronegative. Note: subjects previously infected by chronic hepatitis C and treated with direct acting antivirals (DAAs) with sustained virologic response (SVR) for at least 3 years will be allowed.
  • Female subjects of childbearing potential must agree to use two effective methods of contraception from the date of Screening until 90 days after the last dose of EDP-305.
  • All male participants who have not had a vasectomy must use effective contraception from Day -1 to 90 days after their last dose of study drug.
  • Male subjects must agree to refrain from sperm donation from the date of Screening until 90 days after their last dose of study drug
  • Screening body mass index (BMI) of ≥18 kg/m2
  • Subject must be willing and able to adhere to the assessments, visit schedule, prohibitions and restrictions, as described in this protocol

Who cannot join

  • Laboratory Screening Results:
  • AST >5 x ULN
  • ALT >5 x ULN
  • Patients with Gilbert's syndrome will not be allowed due to interpretability of bilirubin levels
  • Total white blood cells (WBC) <3000 cells/mm3
  • Absolute neutrophil count (ANC) <1500 cells/mm3
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  • Platelet count <140,000/mm3
  • Prothrombin time (international normalized ratio, INR) >1.2
  • Serum creatinine >2 mg/dL or creatinine clearance <60 mL/min (based on Cockroft-Gault Method)
  • Suspected to have relevant nonalcoholic fatty liver disease (NAFLD) as based on the judgment of the Investigator at Screening
  • Use of immunosuppressants known to have an effect on the liver of patients with PBC (eg, colchicine, methotrexate, azathioprine, or systemic steroids) in the three months preceding screening
  • Current use of fibrates, including fenofibrates. Note: Subjects who discontinued fibrates for at least 3 months before Screening can participate
  • Use of an experimental treatment for PBC within the past 6 months
  • Co-existing liver or biliary diseases, such as primary sclerosing cholangitis, choledocholithiasis, acute or chronic hepatitis, autoimmune hepatitis, alcoholic liver disease, nonalcoholic steatohepatitis (NASH), acute infection of bile duct system or gall bladder, history of gastrointestinal bleeding (secondary to portal hypertension), cirrhosis, cholangiocarcinoma diagnosed or suspected liver cancers
  • Cirrhosis with or without complications, including history or presence of: spontaneous bacterial peritonitis, hepatocellular carcinoma
  • Hepatorenal syndrome (type I or II) or Screening serum creatinine > 2 mg/dL (178 μmol/L)
  • Prior variceal hemorrhage, uncontrolled encephalopathy, Child-Pugh Class A, B and C, esophageal varices, or refractory ascites within the previous 6 months of Screening (defined as date informed consent signed)
  • Medical conditions that may cause nonhepatic increases in ALP (e.g., Paget's disease)
  • Use of a new statin regimen from Screening and throughout study duration. NOTE: Subjects on a stable dose of statins for at least 3 months prior to Screening are allowed. No dose modification during the study will be allowed.
  • Use of immunosuppressants (eg, systemic corticosteroids) for more than 2 consecutive weeks in duration within 1 year prior to Screening.

Trial locations and contacts

86 sites in 10 countries.

86 sites in 10 countries

United States40 sites

  • Consultative Gastroenterology

    Atlanta, Georgia, 30312

  • Mercy Medical Center-McAuley Plaza

    Baltimore, Maryland, 21202

  • Johns Hopkins University

    Baltimore, Maryland, 21287

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

  • Digestive Disease Associates

    Catonsville, Maryland, 21228

  • Northwestern University

    Chicago, Illinois, 60611

  • Gastroenterology Consultants of Clearwater

    Clearwater, Florida, 33756-3839

  • Southern California Research Center

    Coronado, California, 92118

  • The Liver Institute at Methodist Dallas Medical Center

    Dallas, Texas, 75203

  • Liver Consultants of Texas

    Dallas, Texas, 76104

  • Henry Ford Hospital

    Detroit, Michigan, 48377

  • Digestive Health Specialists of the Southeast

    Dothan, Alabama, 36305

  • South Denver Gastroenterology - Swedish Medical Center Office

    Englewood, Colorado, 80113

  • Baylor Saint Luke's Medical Center

    Houston, Texas, 77030

  • Nature Coast Clinical Research

    Inverness, Florida, 34452

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • Dartmouth-Hitchcock Medical Center

    Lebanon, New Hampshire, 03756

  • Arkansas Diagnostic Center

    Little Rock, Arkansas, 72205

  • Texas Clinical Research Institute

    Little Rock, Arkansas, 76012

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

  • Northwell Health

    Manhasset, New York, 11030

  • University of Miami Leonard M. Miller School of Medicine

    Miami, Florida, 33136

  • Yale School of Medicine

    New Haven, Connecticut, 06520

  • Concorde Medical Group

    New York, New York, 10016

  • Mount Sinai Beth Isreal

    New York, New York, 10016

  • Weill Cornell Medical College

    New York, New York, 10024

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • CHI Health

    Omaha, Nebraska, 68124

  • California Liver Research Institue

    Pasadena, California, 91105

  • Pasadena Liver Center

    Pasadena, California, 91105

  • University of Pittsburgh Medical Center - Center for Liver Disease

    Pittsburgh, Pennsylvania, 15213

  • Inland Empire Liver Foundation

    Rialto, California, 92377

  • Liver Institute of Virginia-Bremo

    Richmond, Virginia, 23602

  • American Research Corporation at the Texas Liver Institute

    San Antonio, Texas, 78215

  • California Pacific Medical Center

    San Francisco, California, 94115

  • Swedish First Hill Campus

    Seattle, Washington, 98104

  • University of Washington

    Seattle, Washington, 98195-6460

  • Louisiana Research Center

    Shreveport, Louisiana, 71105

  • Montefiore Medical Center - Bronx

    The Bronx, New York, 10461-1925

Other trials in United States

France8 sites

  • Centre Hospitalier Universitaire Amines-Picardie Hôpital Sud

    Amiens, Picardie, 80054

  • Centre Hospitalier Régional Universitaire de Lille

    Lille, Hauts-de-France, 59037

  • Hôpital de la Croix Rousse

    Lyon, Auvergne-Rhône-Alpes, 69317

  • Hôpital Saint-Eloi

    Montpellier, Languedoc-roussillon, 34295

  • Hôpital Saint-Antoine

    Paris, Île-de-France Region, 75012

  • Hôpital Haut-Lévêque

    Pessac, Aquitaine, 33600

  • Nouvel Hôpital Civil

    Strasbourg, Alsace, 67091

  • Hôpital Paul Brousse

    Villejuif, Île-de-France Region, 94800

Other trials in France

Spain8 sites

  • Hospital Universitari Vall d'Hebrón

    Barcelona, 08029

  • Hospital Universitario Donostia

    Donostia / San Sebastian, Guipuzcoa, 20014

  • Hospital Universitario Virgen de la Arrixaca

    El Palmar, Murcia, 30120

  • Hospital Universitario Ramón Y Cajal

    Madrid, 28034

  • Hospital Universitario Marqués de Valdecilla

    Santander, 39008

  • Hospital Universitario Virgen del Rocio

    Seville, 41013

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026

  • Universidad de Valladolid - Hospital Universitario Rio Hortega

    Valladolid, 47010

Other trials in Spain

United Kingdom8 sites

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, England, B15 2TT

  • Cambridge University Hospitals NHS Foundation Trust

    Cambridge, England, CB2 0QQ

  • NHS Lothian

    Edinburgh, Scotland, EH16 4SA

  • The Leeds Teaching Hospitals NHS Trust

    Leeds, England, LS9 7TF

  • King's College Hospital NHS Foundation Trust

    London, England, SE5 9RS

  • Norfolk and Norwich University Hospitals NHS Foundation Trust

    Norwich, England, NR4 7UY

  • Queen's Medical Centre - Nottingham

    Nottingham, England, NG7 2UH

  • Portsmouth Hospitals NHS Trust

    Portsmouth, England, PO6 3LY

Other trials in United Kingdom

Germany7 sites

  • Charité Universitätsmedizin Berlin

    Berlin, 13353

  • Universitätsklinikum Bonn

    Bonn, North Rhine-Westphalia, 53127

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, 45122

  • Universitätsklinikum Frankfurt

    Frankfurt am Main, Hesse, 60590

  • Universitätsklinikum Leipzig

    Leipzig, Saxony, 04103

  • Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz

    Mainz, Rhineland-Palatinate, 55131

  • Universitätsklinikum Würzburg

    Würzburg, Bavaria, 97080

Other trials in Germany

Australia3 sites

  • Monash Medical Centre

    Clayton, Victoria, 3168

  • Nepean Hospital

    Kingswood, New South Wales, 2747

  • Linear Clinical Research

    Perth, Western Australia, 6000

Other trials in Australia

Austria3 sites

  • Medizinische Universität Innsbruck

    Innsbruck, Tyrol, 6020

  • Klinikum Klagenfurt Am Wörthersee

    Klagenfurt, Carinthia, 9020

  • Klinikum Wels-Grieskirchen

    Wels, Upper Austria, 4600

Other trials in Austria

Belgium3 sites

  • Ziekenhuis Oost-Limburg

    Genk, Limburg, 3600

  • Universitair Ziekenhuis Gent

    Ghent, Oost-vlaanderen, 9000

  • CHU de Liège, Cardiology Dept.

    Liège, Liege, 4000

Other trials in Belgium

Canada3 sites

  • London Health Sciences Centre University Hospital

    London, Ontario, N6A 5A5

  • Toronto General Hospital

    Toronto, Ontario, M5G 2C4

  • Toronto Liver Center

    Toronto, Ontario, M6H 3M1

Other trials in Canada

Netherlands3 sites

  • Vrije Universiteit Medisch Centrum

    Amsterdam, North Holland, 1081 HV

  • Leiden Universitair Medisch Centrum

    Leiden, South Holland, 2333 ZA

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX

Other trials in Netherlands

About this study

Official title: A Phase 2 Dose Ranging, Randomized, Double Blind, Placebo-Controlled Study Evaluating the Safety, Tolerability, Pharmacokinetics and Efficacy of EDP-305 in Subjects With Primary Biliary Cholangitis (PBC) With or Without an Inadequate Response to Ursodeoxycholic Acid (UDCA)

A randomized, double-blind study to assess the safety, tolerability, PK and efficacy of EDP-305 in subjects with primary biliary cholangitis

Being tested
EDP-305 1 mg, EDP-305 2.5 mg, Placebo
Keywords
PBC, Primary Biliary Cholangitis (PBC)
Medical subject headings
Liver Cirrhosis, Biliary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EDP-305 1 mgDrug

    Two tablets daily for 12 weeks

  • EDP-305 2.5 mgDrug

    Two tablets daily for 12 weeks

  • PlaceboDrug

    Two tablets daily for 12 weeks

Study groups in NCT03394924
Study groupRoleWhat they receive
EDP-305 1 mgExperimentalSubjects will take 2 tablets once a day orally for 12 weeks
Receives: Drug: EDP-305 1 mg
EDP-305 2.5 mgExperimentalSubjects will take 2 tablets once a day orally for 12 weeks
Receives: Drug: EDP-305 2.5 mg
PlaceboPlacebo comparatorSubjects will take two tablets once a day orally for 12 weeks
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants With At Least a 20% Reduction in Alkaline Phosphatase (ALP) or Normalization of ALP at Week 12 Compared to Baseline

    Percent change was calculated as [(ALP at Week 12 - ALP at Baseline)/ALP at Baseline] *100. The participant was considered to have successfully achieved a 20% reduction in ALP if the result was ≤-20. The participant was considered to have successfully achieved ALP normalization if ALP was abnormal at Baseline and normal at Week 12.

    Measured: Baseline and Week 12

Secondary outcomes (20)

  1. Percentage of Participants With a Treatment-Emergent Adverse Event (TEAE) During On-Treatment Period

    An adverse event (AE) was defined as any event, side effect, or untoward medical occurrence in a subject enrolled in a clinical trial whether or not it is considered to have a causal relationship to the study drug. A TEAE was an AE that first occurred or began previous to and worsened on or after the first dose date and before the last dose date +7 days.

    Measured: Up to approximately Week 12

  2. Percentage of Participants With a Treatment-Emergent Serious Adverse Event (SAE) During On-Treatment Period

    A SAE is any untoward medical occurrence at any dose that results in death, is a life-threatening event, requires inpatient hospitalization or prolonged hospitalization of an existing hospitalization, results in permanent or prolonged disability or incapacity, is a congenital anomaly or birth defect in the offspring of a study subjects, or is a medically important event.

    Measured: Up to approximately Week 12

  3. Percentage of Participants Who Stopped Study Treatment Due to a Treatment-Emergent Adverse Event (TEAE) During On-Treatment Period

    An adverse event (AE) was defined as any event, side effect, or untoward medical occurrence in a subject enrolled in a clinical trial whether or not it is considered to have a causal relationship to the study drug. A TEAE was an AE that first occurred or began previous to and worsened on or after the first dose date and before the last dose date +7 days.

    Measured: Up to approximately Week 12

  4. Change From Baseline to Week 12 in Total, Conjugated and Unconjugated Bilirubin

    The data presented below was measured using least square mean change from baseline.

    Measured: Baseline and Week 12

  5. Change From Baseline to Week 12 in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), and Gamma Glutamyl Transferase (GGT)

    Measured: Baseline and Week 12

  6. Change From Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: Enhanced Liver Fibrosis (ELF) Panel and N-terminal Type III Collagen Propeptide (PRO C3)

    The ELF panel included hyaluronic acid (HA), procollagen III amino terminal peptide (PIIINP), and tissue inhibitor of metalloproteinase 1 (TIMP 1). This endpoint also presents PRO C3 results.

    Measured: Baseline and Week 12

  7. Change From Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: AST to Platelet Ratio Index (APRI) Score

    APRI was calculated as ([AST level/AST upper limit of normal]/[Platelet count 1^09/L])×100. AST is aspartate aminotransferase. The aspartate transaminase to platelet ratio index (APRI) is used to assess liver fibrosis in participants with chronic liver disease. Scores range from 0 to ≥ 2.0, with scores < 0.5 predictive of no liver fibrosis; scores >1.5 significant fibrosis; and scores > 2.0 indicative of cirrhosis. A negative change from baseline indicates a decrease in fibrosis.

    Measured: Baseline and Week 12

  8. Change From Baseline to Week 12 in Noninvasive Liver Fibrosis Markers: Fibrosis-4 (FIB-4) Score

    Fibrosis-4 is the ratio of age in years and aminotransferase to platelet count. It is a non-invasive hepatic fibrosis index score that is calculated using formula: FIB-4 = (Age [years] x AST [U/L]) / (platelets [10^9/L] x (square root of ALT [U/L])). A FIB-4 index of < 1.45 indicates no or moderate fibrosis and an index of > 3.25 indicates extensive fibrosis/cirrhosis. A positive change from Baseline indicates increased fibrosis.

    Measured: Baseline and Week 12

  9. Change From Baseline to Week 12 in Fibrinogen and C Reactive Protein (CRP) Levels

    Measured: Baseline and Week 12

  10. Change From Baseline to Week 12 in Interleukin (IL) and Tumor Necrosis Factor (TNF) Levels

    For IL, both IL6 and IL1β variants were analysed. For TNF, both TNF α and TNF β (also known as lymphotoxin alpha) variants were analyzed.

    Measured: Baseline and Week 12

  11. Change From Baseline to Week 12 in Haptoglobin and Alpha2 Macroglobulin Levels

    Measured: Baseline and Week 12

  12. Change From Baseline to Week 12 in Triglycerides (TG), Total Cholesterol (TC), High Density Lipoprotein Cholesterol (HDL-C), Low Density Lipoprotein Cholesterol (LDL-C)

    Measured: Baseline and Week 12

  13. Change From Baseline to Week 12 in Domain and Total Scores on the 5D-Itch Scale

    The 5D-Itch scale is a multidimensional questionnaire completed by participants to quantify the magnitude of pruritus, assessed considering the past 2 weeks. Scale range is 1 to 5 covering five dimensions: duration (1=Less than 6 hrs/day to 5=All day), degree (1=Not present to 5=Unbearable), direction (1=Completely resolved to 5=Getting worse), disability (for Sleep rated as 1=Never affects sleep to 5=Delays falling asleep and frequently wakes me up at night; for Leisure/Social, Housework/Errands and Work/School rated as 1=Never affects activity to 5=Always affects activity), and distribution (assess if itching is present in 16 body locations, scored as 1=present at 0-2 locations to 5=present at 14-16 locations). Total scores (including highest disability score obtained from any of the daily activities) ranged between 5 and 25 where higher scores indicated more severe itching. Negative change scores indicate improvement from the baseline score.

    Measured: Baseline and Week 12

  14. Change From Baseline to Week 12 in Visual Analog Score (VAS) for Itching

    An itch VAS (0-100mm) was used to record the intensity of the event. Participants drew a line on a scale corresponding to the maximum intensity of itch. Lines drawn towards the right of the line indicated greater itching and higher scores indicated more severe itching. Negative change from baseline indicates decrease in itching.

    Measured: Baseline to Week 12

  15. Change From Baseline to Week 12 in Domain Scores on the Primary Biliary Cholangitis-40 (PBC-40) Quality of Life (QoL) Assessment

    The PBC-40 is a survey measuring health related quality of life in participants with PBC. The 40 questions from the PBC-40 questionnaire are scored from 1-5, with 5 representing the highest impact and 1 the lowest impact of PBC on the quality of life. Six domains were computed from the 40 questions: symptoms (score range 7-35), itch (0-15), fatigue (11-55), cognition (6-30), social (8-50) and emotional (1-15). Higher scores indicate worse quality of life and negative change scores indicate improvement from the baseline score.

    Measured: Baseline and Week 12

  16. Maximum Plasma Concentration (Cmax) of EDP-305 and Its Metabolites

    Metabolites of EDP-305 are EP-022571, EP-022572, and EP-022679.

    Measured: Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose

  17. Time to Maximum Plasma Concentration (Tmax) of EDP-305 and Its Metabolites

    Metabolites of EDP-305 are EP-022571, EP-022572, and EP-022679.

    Measured: Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose

  18. Area Under the Plasma Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) of EDP-305 and Its Metabolites

    Metabolites of EDP-305 are EP-022571, EP-022572, and EP-022679.

    Measured: Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose

  19. Percentage Change From Baseline to Week 12 in Fibroblast Growth Factor 19 (FGF19), 7α-OH-4-cholesten-3-one (C4) and Bile Acid (BA) Concentrations

    FGF19 was measured in plasma. BA was measured in serum. C4 was measured in serum.

    Measured: Baseline and Week 12

  20. Percentage Change From Baseline to Week 12 in AUC0-8 and AUC2-8 of Fibroblast Growth Factor 19 (FGF19), 7α-OH-4-cholesten-3-one (C4) and Bile Acid (BA)

    AUC0-8 is area under the biomarker concentration-time curve from time zero to 8 hours. AUC2-8 is area under the biomarker concentration-time curve from 2 hours to 8 hours. FGF19 was measured in plasma. BA was measured in serum. C4 was measured in serum.

    Measured: Day 1 and Week 12: Pre-dose and 2, 6 and 8 hours post-dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Jan 2018
  2. Study start27 Dec 2017
  3. Primary completion19 Dec 2019
  4. Study completion16 Jan 2020

Resources

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
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
68 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03394924
Sponsor's protocol ID
EDP 305-201
Status last verified
Jun 2020
First submitted
23 Dec 2017
Last updated
18 May 2021
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing EDP-305 1 mg and EDP-305 2.5 mg in people with Primary Biliary Cholangitis, run by Enanta Pharmaceuticals, Inc at 86 sites in 10 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 20 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 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 86 sites in United States, France, Spain, United Kingdom, Germany and 5 more countries. 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 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?

Yes. Results are posted in the registry (first posted 18 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Percentage of Participants With At Least a 20% Reduction in Alkaline Phosphatase (ALP) or Normalization of ALP at Week 12 Compared to Baseline (time frame: Baseline and Week 12). 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 NCT03394924.

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 May 2021.

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