Dose Finding Study to Evaluate Safety and Efficacy of 3 Dosages of SAP 001.

NCT ID: NCT05690204

Last Updated: 2024-11-14

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 87 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-12-12
• Study Completion Date: 2025-02-28

Brief Summary

The aim of this study is to confirm the safety and pharmacological characteristics of SAP-001, evaluate its efficacy in lowering sUA and tophus burden, and identify the appropriate dose regimen for future studies in adult subjects with gout, with or without tophi, and hyperuricemia refractory to SoC XOI therapy.

Detailed Description

A Phase 2B, multicenter, randomized, double-blind, placebo-controlled, dose-finding study to assess the safety, PK, PD, and efficacy of 3 orally administered dosages of SAP-001 compared to placebo QD in adult subjects with gout, with or without tophi, and hyperuricemia refractory to standard-of-care (SoC) XOI therapy. In the completed Phase 1 and Phase 2 studies, SAP-001 was well tolerated at single doses up to 120 mg and at dosages up to 60 mg QD for 28-days in subjects with gout and hyperuricemia and demonstrated statistically significant reductions in sUA levels compared to placebo.

The aim of this study is to confirm the safety and pharmacological characteristics of SAP-001.

Conditions

Gout

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Placebo versus SAP-001

Placebo arm

Group Type: EXPERIMENTAL

SAP-001

Intervention Type: DRUG

Test the efficacy and safety of SAP-001 versus placebo

SAP-001 low dose

SAP-001 low dose

Group Type: EXPERIMENTAL

SAP-001

Intervention Type: DRUG

Test the efficacy and safety of SAP-001 versus placebo

SAP-001 middle dose

SAP-001 middle dose

Group Type: EXPERIMENTAL

SAP-001

Intervention Type: DRUG

Test the efficacy and safety of SAP-001 versus placebo

SAP-001 high dose

SAP-001 high dose

Group Type: EXPERIMENTAL

SAP-001

Intervention Type: DRUG

Test the efficacy and safety of SAP-001 versus placebo

Interventions

SAP-001

Test the efficacy and safety of SAP-001 versus placebo

Intervention Type: DRUG

Other Intervention Names

Xanthine Oxidase Inhibitor; Colchicine

Eligibility Criteria

Inclusion Criteria

1. Male or female, ≥18 and ≤75 years of age, willing and able to provide informed consent and to adhere to the requirements and guidelines of the protocol.

2. Body mass index ≥19 and ≤40 kg/m2 at the Screening Visit (Visit 1).

3. Already have been diagnosed with gout according to the current American College of Rheumatology (ACR) scoring criteria for the classification of primary gout; or has symptoms of gout with at least 1 of the following: i. 3 gout flares in the previous 18 months prior to screening; or ii. Presence of at least 1 gout tophus; or iii. Current diagnosis of gouty arthritis; Subject must be refractory to SoC XOI therapy, or in whom XOI is contraindicated. Refractory to SOC XOI is defined by a medical history of failure to normalize sUA to <6 mg/dL (the ACR target for gout) with at least 3 months of SoC XOI treatment at the maximum medically appropriate dose. XOI contraindication can be self-reported medical contraindication to SoC XOI therapy or in whom SoC XOI therapy is not considered medically appropriate treatment for symptomatic gout. Subject can still participate in the clinical trial if SOC XOI therapy is considered medically not appropriate or contraindicated.

4. Subject must have been on SoC XOI therapy for gout and hyperuricemia for at least 4 weeks immediately before the Randomization Visit (Day 1, Visit 4) unless SoC XOI therapy is contraindicated or not medically appropriate. Subjects who stopped SoC XOI therapy within 4 weeks of the Screening Visit are eligible for the study but must be restarted on SoC XOI therapy and confirmed resistant to XOI therapy (sUA levels ≥7.0 mg/dL) after at least 4 weeks of treatment.

5. Subject must have sUA levels ≥7.0 mg/dL by central laboratory results at the Screening Visit (Visit 1) and prior to randomization at the Randomization Visit (Day 1, Visit 4).

Exclusion Criteria

1. Subjects not previously diagnosed as having gout before the Screening Visit.

2. Female subject is pregnant, planning to get pregnant, lactating/breastfeeding, or has a positive urine pregnancy test at the Screening Visit or prior to randomization at the Randomization Visit (Day 1, Visit 4).

3. Subject has used any prescription drugs (eg, losartan, pegloticase, URAT1 inhibitors), OTC medications, herbal medications or products, vitamins, or minerals that are known to lower sUA levels (except SoC XOI therapies) within 14 days prior to the Randomization Visit (Day 1, Visit 4). Exceptions may be made on a case-by-case basis (such as chronic use of low dose aspirin) following discussion and agreement between the investigator and sponsor. Subjects who are already taking losartan for blood-pressure control are allowed to enroll in the study and continue taking losartan if they have been on a stable dose for at least 6 months.

4. Subject was not compliant with taking placebo during the Run-in Period (defined as taking <80% or >120% of planned placebo doses) or the investigator determines that the subject was not compliant with SoC XOI gout medications (unless SoC XOI therapy is contraindicated or not medically appropriate) during the Run-in Period as assessed prior to randomization at the Randomization Visit (Day 1, Visit 4).

5. Subject had an acute gout flare (exclusive of symptomology associated with chronic synovitis/arthritis) that did not resolve at least 14 days prior to the Randomization Visit (Day 1, Visit 4). If an acute gout flare occurs during the Screening or Run-in Periods, the subject may be rescreened after a period of at least 14 days has passed following resolution of the flare.

6. Serum creatinine level >1.5 mg/dL and/or eGFR ≤60 mL/min/1.73 m2 calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation23 by central laboratory results at the Screening Visit (Visit 1) or prior to randomization at the Randomization Visit (Day 1, Visit 4).

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shanton Pharma Pte. Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Carmen Arencibia

Role: STUDY_DIRECTOR

Study Official

Locations

California Site

Sacramento, California, United States

California Site

San Diego, California, United States

Denver Site

Denver, Colorado, United States

Florida Site

DeBary, Florida, United States

Florida Site

Miami, Florida, United States

Florida Site

Miami Lakes, Florida, United States

Florida Site

Miami Lakes, Florida, United States

Florida Site

Winter Park, Florida, United States

Idaho Site

Boise, Idaho, United States

Maryland Site

Oxon Hill, Maryland, United States

Mississippi Site

Jackson, Mississippi, United States

North Carolina Site

Raleigh, North Carolina, United States

Texas Site

Mesquite, Texas, United States

Texas Site

Plano, Texas, United States

Texas Site

The Woodlands, Texas, United States

Puerto Rico Site

San Juan, , Puerto Rico

Countries

United States; Puerto Rico

Other Identifiers

SAP-001-202

Identifier Type: -

Identifier Source: org_study_id