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

Withdrawn before enrolling · Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis, Cystic Fibrosis Pulmonary Exacerbation

In brief

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).

Key facts

Study ID

NCT05641298

Run by

Aceragen

People needed

0

Starts

2023-02-10

Expected to finish

2023-09-01

Last updated by the study team

2023-07-19

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

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).
• Known hepatitis C virus (HCV) or infection and currently receiving HCV-specific antiviral therapy. HCV infection alone, and in the absence of decompensated liver disease, is not exclusionary.
• 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)
• Moderate/strong CYP3A4 inhibitors (eg, azole antifungals, erythromycin, clarithromycin)
• P-gp substrates with narrow therapeutic windows (eg, digoxin and colchicine)
• Prior treatment with a CYP3A4 inducer (such as lumacaftor, dexamethasone, phenytoin, carbamazepine, rifampin, phenobarbital and nafcillin) within 7 days prior to enrollment.
• Dietary use of large amounts of grapefruit juice and/or Seville oranges or other products containing these fruits (eg, grapefruit juice or marmalade) during the study.
• Participant requiring warfarin therapy.
• Seizure disorder requiring current therapy with an anticonvulsant.
• Female participant who is pregnant or lactating.
• History of /current chronic alcohol consumption and/or drug abuse (including cannabis use).
• Any study personnel or their immediate dependents, family, or household members.

Where it is running

• University of Florida — Gainesville, Florida, United States
• Central Florida Pulmonary Group — Orlando, Florida, United States
• Nemours Children's Health - Pensacola — Pensacola, Florida, United States
• Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States
• Cystic Fibrosis Center of Chicago — Chicago, Illinois, United States
• Cystic Fibrosis Center of Chicago — Northfield, Illinois, United States
• Riley Hospital for Children — Indianapolis, Indiana, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• University Of Louisville — Louisville, Kentucky, United States
• Maine Medical Center — Portland, Maine, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• University of Michigan, Michigan Medicine — Ann Arbor, Michigan, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• UNMC-Nebraska CF Pediatric Center — Omaha, Nebraska, United States
• Gunnar H. Esiason Adult Cystic Fibrosis Center — Morristown, New Jersey, United States
• New York Medical College — Hawthorne, New York, United States
• University of Rochester Medical Center Strong Memorial — Rochester, New York, United States
• Rainbow Babies and Children's Hospital/Cleveland Medical Center — Cleveland, Ohio, United States
• University of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Cook Children's Health Care System — Fort Worth, Texas, United States
• The University of Health Science Center at Tyler — Tyler, Texas, United States
• Providence Medical Research Center — Spokane, Washington, United States

Full record on ClinicalTrials.gov

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