Study to Determine the Efficacy&Safety of ARV-1801(ACG-701) for the Treatment of Cystic Fibrosis Pulmonary Exacerbations

NCT05641298 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: ACG-701-101
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 25

Brief Summary

This study will evaluate the efficacy and safety of an oral ARV-1801(ACG-701) plus optimized background therapy (OBT) compared to oral placebo plus OBT, each administered for 14 days, in the treatment of participants with Cystic Fibrosis-related pulmonary exacerbations (PEx).

Conditions

Cystic Fibrosis; Cystic Fibrosis Pulmonary Exacerbation

Keywords

Pulmonary Exacerbation; Antibiotic

Outcome Measures

Primary Outcomes (1)

• Desirability in outcome ranking (DOOR)

  To demonstrate that the addition of oral ARV-1801(ACG-701) to OBT is superior to placebo plus OBT based on DOOR in cystic fibrosis pulmonary exacerbations.

  Day 7

Study Arms (2)

ARV-1801(ACG-701) Active Group

ACTIVE COMPARATOR

ARV-1801(ACG-701) tablets by mouth twice a day for 14 days

Drug: Sodium Fusidate

Placebo Group

PLACEBO COMPARATOR

Placebo tablets by mouth twice a day for 14 days

Drug: Placebo

Interventions

Sodium Fusidate DRUG

Tablets

Also known as: ACG-701, ARV-1801

ARV-1801(ACG-701) Active Group

Placebo DRUG

Tablets

Placebo Group

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females of 12 years of age and older
• Participants must have a confirmed diagnosis of Cystic Fibrosis with a diagnosis of an acute pulmonary exacerbation as defined as:
• Deterioration in 3 or more of the following symptoms for at least 48 hrs (cough, sputum volume and/or consistency, sputum purulence, breathlessness and/or exercise tolerance, fatigue and/or malaise, or hemoptysis) And
• a clinician determines that a change in CF treatment is required
• Participants must have a CFRSD-CRISS score of \>/= 35
• Participants must have a moderate or Severe Patient Global Impression of Severity
• Participants must have a negative pregnancy test and agree to use a highly effective method of contraception during the study and 30 days after last dose
• Participants must agree not to smoke during any part of the clinical trial
• Participants must voluntarily sign the informed consent for the study

You may not qualify if:

• Participants cannot have any changes in any antimicrobial, bronchodilator, anti-inflammatory, CFTR modulator or corticosteroid medications from 28 - 3 days prior to the Screening visit.
• Participants cannot be receiving treatment for non-tuberculosis mycobacteria and/or Aspergillus infection.
• History of hypersensitivity or allergic reaction to sodium fusidate, fusidic acid (Fucidin®) or its excipients.
• Abnormal laboratory findings or other findings or medical history at Screening that, in the Investigator's opinion, would compromise the safety of the participant or the quality of the study data.
• The use of an investigational drug or device (ie, a drug or device without the FDA approved indication) within 30 days prior to the Screening visit
• Known severe renal impairment, as indicated by estimated creatinine clearance (CrCl) \<30 mL/min (by Cockcroft-Gault calculation).
• Evidence of significant liver disease: ALT \>3×ULN, or direct bilirubin \>ULN, or total bilirubin \>1.5 mg/dL; known cirrhosis with decompensation (ie, Child-Pugh Class B or C disease).
• Neutropenia (absolute neutrophil count \<500/µL); thrombocytopenia (\<60,000 platelets/mm3).
• Known human immunodeficiency virus (HIV) infection and currently receiving antiretroviral therapy, or current CD4 count ≤200 cells/mm3 (documented within 3 months prior to enrollment); if CD4 count is unknown, participant may not enroll.
• Changes to or initiation of immunosuppressant agents (ie, prednisone \[≥15mg/day\], cyclosporine, tumor necrosis factor alpha \[TNFα\] antagonist) within 30 days of study medication administration through the EOS visit.
• Malignancy requiring ongoing cytotoxic chemotherapy or radiation therapy.
• Requires concomitant treatment with (washout period prior to randomization allowed):
• OATP1B1 and OATP1B3 substrates (eg, HMG-CoA reductase inhibitors \[statins\])
• CYP2C8 substrates, namely glitazones (eg, repaglinide)
• CYP3A4 inducers (eg, dexamethasone, phenytoin, carbamazepine, rifampin, phenobarbital, nafcillin)

Sponsors & Collaborators

• Aceragen: lead

Study Sites (25)

University of Florida

Gainesville, Florida, 32610, United States

Location

Central Florida Pulmonary Group

Orlando, Florida, 32803, United States

Location

Nemours Children's Health - Pensacola

Pensacola, Florida, 32514, United States

Location

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Cystic Fibrosis Center of Chicago

Chicago, Illinois, 60093, United States

Location

Cystic Fibrosis Center of Chicago

Northfield, Illinois, 60093, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University Of Louisville

Louisville, Kentucky, 40202, United States

Location

Maine Medical Center

Portland, Maine, 04102, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

University of Michigan, Michigan Medicine

Ann Arbor, Michigan, 48109, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63114, United States

Location

UNMC-Nebraska CF Pediatric Center

Omaha, Nebraska, 68114, United States

Location

Gunnar H. Esiason Adult Cystic Fibrosis Center

Morristown, New Jersey, 07960, United States

Location

New York Medical College

Hawthorne, New York, 10532, United States

Location

University of Rochester Medical Center Strong Memorial

Rochester, New York, 14642, United States

Location

Rainbow Babies and Children's Hospital/Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15224, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Cook Children's Health Care System

Fort Worth, Texas, 76104, United States

Location

The University of Health Science Center at Tyler

Tyler, Texas, 75708, United States

Location

Providence Medical Research Center

Spokane, Washington, 99204, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Fusidic Acid

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Cholestadienols; Cholestadienes; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Membrane Lipids; Lipids

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 29, 2022
• First Posted: December 7, 2022
• Study Start: February 10, 2023
• Primary Completion: September 1, 2023
• Study Completion: September 1, 2023
• Last Updated: July 19, 2023

Record last verified: 2023-02

Locations

US (25)