Ceftaroline for Cystic Fibrosis

Age: < 65

Sex: Any

Trial Phase: Phase 4

Sponsor: Children's Hospital Medical Center, Cincinnati

Must be taking: Ceftaroline

Disqualifiers: Age, Weight, Liver function, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 hours

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how ceftaroline, an antibiotic, can more effectively treat children with cystic fibrosis who have a history of MRSA, a challenging bacterial infection. The study aims to fine-tune dosing strategies by analyzing the drug's movement and action in the body (pharmacokinetics and pharmacodynamics). Children with cystic fibrosis who are currently hospitalized and have been selected by their doctor to receive ceftaroline may be suitable for this trial. The goal is to improve treatment plans specifically for these young patients. As a Phase 4 trial, this research focuses on understanding how this FDA-approved and effective treatment can benefit more patients.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What is the safety track record for ceftaroline?

A study focusing on children with cystic fibrosis (CF) found ceftaroline to be safe and effective for treating MRSA, a difficult-to-treat bacterium. Another study showed that ceftaroline's safety profile in children mirrors that in adults, with no new safety concerns identified. This indicates the treatment is generally well-tolerated. Additionally, the FDA has already approved ceftaroline for treating other infections, confirming its thorough safety testing. For those considering joining a trial involving ceftaroline, existing research suggests it is a safe option. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Ceftaroline is unique because, unlike traditional antibiotics used for cystic fibrosis, it has a strong action against drug-resistant bacteria. Most treatments for cystic fibrosis target common bacterial infections, but ceftaroline is effective against more stubborn strains like MRSA, which are harder to treat. Researchers are excited about ceftaroline because it could offer a new option for patients who have infections that don't respond well to current therapies, potentially improving their quality of life.

What is the effectiveness track record for ceftaroline in treating cystic fibrosis?

Research has shown that ceftaroline effectively combats infections caused by methicillin-resistant Staphylococcus aureus (MRSA). Studies have found ceftaroline fosamil effective for treating skin infections and pneumonia, even in cases involving resistant bacteria like MRSA. In children, ceftaroline has led to successful treatment, resulting in recovery, no recurrence of infection, and noticeable improvement. This trial will evaluate ceftaroline for cystic fibrosis patients dealing with MRSA, suggesting it could be a helpful treatment option. ^{4 6 7 8 9}

Who Is on the Research Team?

Alexander Vinks, PhD

Principal Investigator

Children's Hospital Medical Center, Cincinnati

Are You a Good Fit for This Trial?

This trial is for children and teens with cystic fibrosis who have had infections caused by a tough-to-treat bacteria called MRSA. They must be in the hospital, weigh at least 15 kg, and their liver tests need to be within certain limits. The study excludes very young kids under 2 years old, adults over 22, those with severe liver issues or low platelets, patients without confirmed CF diagnosis, and non-English speaking families.

Inclusion Criteria

I am currently hospitalized.

My doctor has decided to treat me with intravenous ceftaroline.

I have been diagnosed with Cystic Fibrosis according to the foundation's guidelines.

Exclusion Criteria

Your liver enzymes (AST or ALT) are more than 5 times the normal level.

I am under 2 years old.

Your platelet count is less than 50,000.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravenous ceftaroline dosed according to current recommendations for community acquired pneumonia in pediatric patients

12 hours

Single visit

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of pulmonary exacerbations and FEV1 percentage change

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ceftaroline

Trial Overview

The study is testing how the drug Ceftaroline works in the bodies of kids and teens with cystic fibrosis when given through an IV. It looks at how much of the drug gets into the bloodstream and how it fights bacteria. This will help figure out the best doses that match up with disease severity and bacterial resistance.

How Is the Trial Designed?

Group I: PK/PD Experimental Treatment 1 Intervention

Open-label prospective study with participants receiving treating physician-approved intravenous ceftaroline, dosed according to current recommendations. Blood samples collected at baseline, 1 hour, 1.5 hours, 3 hours, and 6 hours after infusion.

Ceftaroline is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Teflaro for:

• Community-acquired bacterial pneumonia
• Acute bacterial skin and skin structure infections

Approved in European Union as Zinforo for:

• Complicated skin and soft tissue infections
• Community-acquired pneumonia

Approved in Canada as Teflaro for:

• Community-acquired pneumonia
• Acute bacterial skin and skin structure infections

Who Is Running the Clinical Trial?

Children's Hospital Medical Center, Cincinnati

Lead Sponsor

Trials

844

Recruited

6,566,000+

Published Research Related to This Trial

The combination therapy of elexacaftor, ivacaftor, and tezacaftor (Trikafta™) has shown significant improvements in lung function and quality of life for cystic fibrosis patients with the F508del mutation, based on multinational phase II and III studies.

In October 2019, this treatment was approved by the US FDA for patients aged 12 and older with at least one F508del mutation, marking a major advancement in cystic fibrosis therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Elexacaftor/Ivacaftor/Tezacaftor: First Approval. Hoy, SM.[2020]

In a review of safety data from 14 clinical trials involving 1,711 participants, the rates of depression-related adverse events in patients with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) were similar to those in the placebo group, indicating no increased risk associated with the treatment.

Postmarketing data from 61,499 patients showed low exposure-adjusted rates of depression-related events, and patterns of depression prevalence did not change after starting ELX/TEZ/IVA, suggesting that any reported depression symptoms are consistent with the background rates in the cystic fibrosis population rather than being caused by the medication.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Elexacaftor/Tezacaftor/Ivacaftor Treatment and Depression-related Events. Ramsey, B., Correll, CU., DeMaso, DR., et al.[2023]

Elexacaftor-tezacaftor-ivacaftor is a newly approved triple-combination therapy for cystic fibrosis that significantly improves lung function, quality of life, and reduces exacerbations in patients aged 12 and older with at least one F508del mutation in the CFTR gene, surpassing existing treatments.

While the therapy shows promising efficacy, common side effects include rash and headache, and liver function monitoring is advised; however, about 10% of the cystic fibrosis population remains ineligible for this or any CFTR modulation therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy. Ridley, K., Condren, M.[2020]

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/38979062/

Real-World Use and Treatment Outcomes of Ceftaroline ...

All-cause 30-day readmission occurred in 13 (9.8%) patients, and all-cause 30-day mortality in 7 (5.3%). Clinical response to ceftaroline was ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11230118/

Real-World Use and Treatment Outcomes of Ceftaroline ...

Ceftaroline fosamil has been demonstrated to be an effective treatment for patients hospitalized with cSSTI or CAP, including those at risk of ...

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S1569199317309475

Ceftaroline pharmacokinetics and pharmacodynamics in ...

The objective of this study was to determine the pharmacokinetic and pharmacodynamic profile of IV ceftaroline in patients with CF.

4.

mdpi.com

mdpi.com/2079-6382/14/9/864

Evaluation of Ceftaroline Use in Pediatric Patients

The primary outcome was clinical success, defined as a composite of 30-day survival, absence of microbiological recurrence within 30 days, and/or resolution of ...

5.

academic.oup.com

academic.oup.com/ofid/article/8/Supplement_1/S745/6451133

1315. Ceftaroline Versus Vancomycin for the Treatment of ...

There were no differences in baseline characteristics (Table 1). Eleven (50%) patients in the ceftaroline group and 19 (56%) in the vancomycin ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11128847/

Ceftaroline fosamil treatment patterns and outcomes in ...

Overall, clinical response to ceftaroline fosamil was associated with >60% shorter length of ICU stay (3.6 versus 9.2 days), and >30% lower ...

7.

sciencedirect.com

sciencedirect.com/science/article/pii/S2213716521002800

Ceftaroline versus vancomycin for treatment of acute ...

In a study of predominantly paediatric CF patients, ceftaroline was found to be safe and efficacious for MRSA-mediated APEs compared with vancomycin [9].

8.

fda.gov

fda.gov/media/113598/download

Teflaro® (ceftaroline fosamil) for injection

In summary, the safety findings were similar to those seen in the adult studies, and no safety concerns were identified beyond those already ...

9.

clinicaltrials.gov

clinicaltrials.gov/study/NCT03771313

Study Details | NCT03771313 | Pharmacokinetic and ...

This is an open-label, single center, prospective study of patients with a diagnosis of cystic fibrosis (CF) and a history of methicillin-resistant ...