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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03421431




Registration number
NCT03421431
Ethics application status
Date submitted
29/01/2018
Date registered
5/02/2018
Date last updated
20/03/2019

Titles & IDs
Public title
A Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of EDP-305 in Subjects With Non-Alcoholic Steatohepatitis
Scientific title
A Phase 2 Dose Ranging, Randomized, Double Blind, and Placebo-Controlled Study Evaluating the Safety, Tolerability, Pharmacokinetics and Efficacy of EDP-305 in Subjects With Non-Alcoholic Steatohepatitis (NASH)
Secondary ID [1] 0 0
EDP 305-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Non-Alcoholic Steatohepatitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Diet and Nutrition 0 0 0 0
Obesity

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - EDP-305 Dose 1
Treatment: Drugs - EDP-305 Dose 2
Treatment: Drugs - Placebo

Experimental: EDP-305 Dose 1 - Subjects will take 2 tablets once a day orally for 12 weeks

Experimental: EDP-305 Dose 2 - Subjects will take 2 tablets once a day orally for 12 weeks

Placebo Comparator: Placebo - Subjects will take 2 tablets once a day orally for 12 weeks


Treatment: Drugs: EDP-305 Dose 1
Two tablets daily for 12 weeks

Treatment: Drugs: EDP-305 Dose 2
Two tablets daily for 12 weeks

Treatment: Drugs: Placebo
Two tablets daily for 12 weeks

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baseline in ALT levels at Week 12
Timepoint [1] 0 0
Measurement at Week 12
Primary outcome [2] 0 0
Safety as measured by frequency of adverse events (AEs), serious AEs, and AEs leading to discontinuation through Week 12
Timepoint [2] 0 0
Up to 12 weeks

Eligibility
Key inclusion criteria
- An informed consent document must be 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 presence of NASH by:

- Histologic evidence on a historical liver biopsy within 24 months of Screening
consistent with NASH with fibrosis (no cirrhosis), and elevated ALT at Screening
AND Screening MRI PDFF with >8 % steatosis OR

- Phenotypic diagnosis of NASH based on elevated ALT and diagnosis of T2DM or
pre-diabetes AND Screening MRI PDFF with >8 % steatosis

- Body mass index (BMI) >25 kg/m2; for Asian-Americans, BMI >23 kg/m2

- 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.

- Subject must be willing and able to adhere to the assessments, visit schedules,
prohibitions and restrictions, as described in this protocol
Minimum age
18 Years
Maximum age
75 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Laboratory Screening Results:

- Total bilirubin > ULN (normal range 0.2-1.2 mg/dL)

- Total white blood cells (WBC) <3,000 cells/mm3

- Absolute neutrophil count (ANC) <1,500 cells/mm3

- Platelet count <140,000/mm3

- Prothrombin time (international normalized ratio, INR) > 1.2

- Creatine kinase above the upper limit of normal (ULN) except when in relation
with intense exercise

- Serum creatinine >2 mg/dL or creatinine clearance <60 ml/min (based on Cockroft
Gault method)

- Known history of alpha-1-antitrypsin deficiency

- Use of an experimental treatment for NASH within the past 6 months

- Use of immunosuppressant (eg, corticosteroids) for more than 2 weeks in duration
within 1 year prior to Screening and during the course of the study

- Use of experimental or unapproved drugs within a year of Screening

- Any other condition(s) (including cardiovascular diseases) that would compromise the
safety of the subject or compromise the quality of the clinical study, as judged by
the Principal Investigator (PI)

- Pregnant or nursing females

- Recipients of liver or other organ transplantation or anticipated need for orthotropic
organ transplantation in one year as determined by a Model for End-Stage Liver Disease
(MELD) Score = 15

- Clinical suspicion of advanced liver disease or cirrhosis

- Coexisting liver or biliary diseases, such as primary sclerosing cholangitis (PSC),
choledocholithiasis, acute or chronic hepatitis, autoimmune hepatitis, alcoholic liver
disease, acute infection of bile duct system or gall bladder, history of
gastrointestinal bleeding (secondary to portal hypertension), cirrhosis

- Suspicion of cancer (eg, liver cancer) with the exception of basal cell carcinoma that
has been resected

- Cirrhosis with or without complications, including history or presence of: spontaneous
bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2xULN

- 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)

- Any condition possibly affecting drug absorption (eg, gastrectomy <3 years prior to
Screening)

- Subject has received an investigational agent or vaccine within 30 days, or a
biological product within 3 months or 5 elimination half-lives (whichever is longer)
prior to the planned intake of study drug. NOTE: Flu vaccine will be allowed upon
Medical Monitor's approval

- Use of a new statin regimen from Screening and throughout study duration. NOTE:
Subjects on a stable dose of statins for at least three months prior to Screening are
allowed. No dose modification during the study will be allowed.

- Current use of fibrates. Note: Subjects who discontinued fibrates for at least 3
months before Screening can participate

- Clinically significant history of drug sensitivity or allergy, as determined by the PI

- Uncontrolled diabetes mellitus (ie, HbA1c =9% or higher) 60 days prior to Day 1

- Subjects with contraindications to MRI imaging, or not being able to have the MRI
performed

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Florida
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United States of America
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Georgia
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United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Indiana
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Louisiana
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United States of America
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Maryland
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United States of America
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Michigan
Country [12] 0 0
United States of America
State/province [12] 0 0
Missouri
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United States of America
State/province [13] 0 0
New Jersey
Country [14] 0 0
United States of America
State/province [14] 0 0
New York
Country [15] 0 0
United States of America
State/province [15] 0 0
North Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Ohio
Country [17] 0 0
United States of America
State/province [17] 0 0
Pennsylvania
Country [18] 0 0
United States of America
State/province [18] 0 0
Tennessee
Country [19] 0 0
United States of America
State/province [19] 0 0
Texas
Country [20] 0 0
United States of America
State/province [20] 0 0
Utah
Country [21] 0 0
United States of America
State/province [21] 0 0
Virginia
Country [22] 0 0
United States of America
State/province [22] 0 0
Washington
Country [23] 0 0
United States of America
State/province [23] 0 0
Wisconsin
Country [24] 0 0
Canada
State/province [24] 0 0
Ontario
Country [25] 0 0
Canada
State/province [25] 0 0
Quebec
Country [26] 0 0
France
State/province [26] 0 0
Paris
Country [27] 0 0
France
State/province [27] 0 0
Pessac
Country [28] 0 0
France
State/province [28] 0 0
Strasbourg
Country [29] 0 0
New Zealand
State/province [29] 0 0
Auckland
Country [30] 0 0
Puerto Rico
State/province [30] 0 0
San Juan
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Cambridgeshire
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Greater London
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Nottinghamshire

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Enanta Pharmaceuticals
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
ICON Clinical Research
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Triangle Biostatistics
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
A randomized, double-blind study to assess the safety, tolerability, PK and efficacy of
EDP-305 in subjects with Non-Alcoholic Steatohepatitis
Trial website
https://clinicaltrials.gov/show/NCT03421431
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Nathalie Adda, MD
Address 0 0
Enanta Pharmaceuticals Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications