Genetic Data Collection in Adult Participants to Identify Genetic Variants of Known Importance in Non-alcoholic Steatohepatitis (NASH)

Regeneron Pharmaceuticals

Status: Terminated

Conditions: Non-alcoholic Steatohepatitis (NASH)

Treatments: Procedure: No Intervention

Study type: Interventional

Funder types: Industry

Identifiers

NCT05423327

R0000-NASH-CES-2117

Details and patient eligibility

About

Collection of clinical and genetic data to help identify individuals that carry genetic variants of known importance in Non-alcoholic Steatohepatitis (NASH)

Enrollment: 5,311 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

One or more of the following:

1. A prior diagnosis of NASH with a Clinical Research Network (CRN) fibrosis score of F1 to F4 based on liver biopsy

2. Evidence of NAFLD by imaging or liver histology as described in the protocol

   • The possible imaging modalities to assess historic evidence of NAFLD may be based on thresholds as described in the protocol

3. Known high-risk genotype for HSD17B13 (T/T or T/TA) and/or PNPLA3 (C/G,or G/G)

4. A clinical suspicion of NASH based on presence of 2 or more elements of the metabolic syndrome defined by:

   1. Waistline that measures >35 inches (89 centimeters) for women or >40 inches (102 centimeters) for men
   2. Historic fasting triglycerides >150 mg/dL within the prior 6 months
   3. Historic fasting HDL cholesterol <40 mg/dL in men or <50 mg/dL in women, or on cholesterol-lowering medication within the prior 6 months
   4. Historic fasting blood glucose >100 mg/dL or on diabetes medication within the prior 6 months
   5. Historic blood pressure >130/85 mmHg, or on anti-hypertensive medication within the prior 6 months

Key Exclusion Criteria:

1. Known history or clinical evidence of drug abuse, within the 12 months before screening. Drug abuse is defined as compulsive, repetitive, and/or chronic use of drugs or other substances with problems related to their use and/or where stopping or a reduction in dose will lead to withdrawal symptoms.
2. Excessive alcohol intake for ≥3 months during the past year prior to screening (>3 units/day for males and >2 units/day for females is generally considered excessive (unit: 1 glass of wine [approximately 125 mL]=1 measure of spirits [approximately 1 fluid ounce]=½ pint of beer [approximately 284 mL]).
3. History of liver transplant, current placement on a liver transplant list, or Model for End-stage Liver Disease (MELD) score >12.
4. History of viral and resolved hepatitis or human immunodeficiency virus (HIV).
5. Any malignancy within the past 5 years except for basal cell or squamous epithelial cell carcinoma of the skin, or any carcinoma in situ.

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Trial design

Primary purpose: Screening

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5,311 participants in 1 patient group

Single Blood Draw

Other group

Treatment:

Procedure: No Intervention