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Cystic FibrosisPhase 2REPRIEVENCT05641298

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

A Phase 2 study testing Sodium Fusidate in people with Cystic Fibrosis and Cystic Fibrosis Pulmonary Exacerbation, run by Aceragen at 25 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 12 years and older
What is being tested?
Sodium Fusidate
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
25 sitesUnited States
Study dates
10 Feb 2023to Sep 2023
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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).
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  • 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.

Trial locations and contacts

25 sites in 1 country.

25 sites in 1 country

United States25 sites

  • University of Michigan, Michigan Medicine

    Ann Arbor, Michigan, 48109

  • Johns Hopkins University

    Baltimore, Maryland, 21287

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Cystic Fibrosis Center of Chicago

    Chicago, Illinois, 60093

  • Rainbow Babies and Children's Hospital/Cleveland Medical Center

    Cleveland, Ohio, 44106

  • Cook Children's Health Care System

    Fort Worth, Texas, 76104

  • University of Florida

    Gainesville, Florida, 32610

  • New York Medical College

    Hawthorne, New York, 10532

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • University Of Louisville

    Louisville, Kentucky, 40202

  • Gunnar H. Esiason Adult Cystic Fibrosis Center

    Morristown, New Jersey, 07960

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Cystic Fibrosis Center of Chicago

    Northfield, Illinois, 60093

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

  • UNMC-Nebraska CF Pediatric Center

    Omaha, Nebraska, 68114

  • Central Florida Pulmonary Group

    Orlando, Florida, 32803

  • Nemours Children's Health - Pensacola

    Pensacola, Florida, 32514

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15224

  • Maine Medical Center

    Portland, Maine, 04102

  • University of Rochester Medical Center Strong Memorial

    Rochester, New York, 14642

  • Providence Medical Research Center

    Spokane, Washington, 99204

  • Washington University School of Medicine

    St Louis, Missouri, 63114

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701

  • The University of Health Science Center at Tyler

    Tyler, Texas, 75708

Other trials in United States

About this study

Official title: A Phase 2, Randomized, DB, Placebo-controlled Study to Determine the Efficacy, Safety and PK Profile of ARV-1801 in Combination With Optimized Background Therapy for the Treatment of Pulmonary Exacerbations in Patients With Cystic Fibrosis

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).

Read the detailed description

This is a Phase 2, randomized, double-blind, multicenter study designed to evaluate the efficacy and safety of an oral ARV-1801/ACG-701 plus OBT compared to oral placebo plus OBT, each administered for 14 days, in the treatment of participants with CF-related PEx.

Participants who provide informed consent (plus informed assent, if applicable) and meet all study eligibility criteria will be enrolled in the study and randomized via an interactive response technology (IRT) in a 1:1 ratio to receive ARV-1801/ACG-701 or placebo in addition to OBT for 14 days. A participant may be hospitalized (inpatient) or treated as an outpatient. If admitted to the hospital for initial treatment, the participant may be discharged at the Investigator's discretion to complete study therapy as an outpatient. The target study population will comprise 80 participants.

The duration of participation in the study for an individual participant will be approximately 28 days and will involve up to 5 clinic visits as well as the requirement to complete an electronic symptom questionnaire every day for the duration of the study. Participation will include a Screening period of up to 24 hours prior to the first dose of study drug (Day 1), a 14 day treatment course of study drug, a Day 7 visit (±1 day), an end of treatment (EOT) visit (day of last dose of study drug +3 days), and end of study (EOS) visit on Day 28 (+3 days).

Being tested
Sodium Fusidate, Placebo
Keywords
Pulmonary Exacerbation, Antibiotic
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Sodium FusidateDrug

    Also called ACG-701, ARV-1801

    Tablets

  • PlaceboDrug

    Tablets

Study groups in NCT05641298
Study groupRoleWhat they receive
ARV-1801(ACG-701) Active GroupActive comparatorARV-1801(ACG-701) tablets by mouth twice a day for 14 days
Receives: Drug: Sodium Fusidate
Placebo GroupPlacebo comparatorPlacebo tablets by mouth twice a day for 14 days
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Day 7

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Dec 2022
  2. Study start10 Feb 2023
  3. Primary completionSep 2023 (estimated)
  4. Study completionSep 2023 (estimated)

Why it stopped: Corporate finances

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05641298
Sponsor's protocol ID
ACG-701-101
Acronym
REPRIEVE
Status last verified
Feb 2023
First submitted
29 Nov 2022
Last updated
19 Jul 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Hamasaki T, He Y, Wu Q, Howard-Anderson J, Boucher HW, Doernberg SB, Holland TL, Powers JH 3rd, Wang J, Diao G, van Duin D, Fowler VG Jr, Chambers HF, Evans SR. A patient-centric paradigm and tool for clinical research: the DOOR is open. Antimicrob Agents Chemother. 2026 Jan 7;70(1):e0147825. doi: 10.1128/aac.01478-25. Epub 2025 Nov 24.

    PubMed 41277847

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sodium Fusidate in people with Cystic Fibrosis and Cystic Fibrosis Pulmonary Exacerbation, run by Aceragen at 25 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 19 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Desirability in outcome ranking (DOOR) (time frame: Day 7). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05641298, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jul 2023.

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