What is the mechanism of action of this treatment?

Common treatments for Cystic Fibrosis (CF) include antibiotics like Cephalexin, which target and kill specific bacteria such as MSSA, and inhaled antibiotics like tobramycin and aztreonam, which target Pseudomonas aeruginosa. These antibiotics disrupt bacterial cell walls or protein synthesis, leading to bacterial death. Additionally, inhaled hypertonic saline and DNase help to hydrate and break down mucus in the lungs, improving airway clearance. These treatments are essential for CF patients as they manage chronic lung infections, reduce inflammation, and improve lung function, thereby enhancing quality of life and survival.

What side effects are associated with this type of intervention?

Common side effects of antibiotics used in the treatment of Cystic Fibrosis, including Cephalexin, often involve gastrointestinal issues such as nausea, vomiting, and diarrhea. Allergic reactions can also occur. Inhaled antibiotics like tobramycin and aztreonam may cause cough, throat irritation, and bronchospasm. Systemic antibiotics, particularly aminoglycosides like tobramycin, can lead to more severe side effects such as nephrotoxicity and ototoxicity, which require careful monitoring.

What prior FDA approvals exist?

The FDA has approved several antibiotics for the treatment of Cystic Fibrosis, particularly targeting common pathogens such as MSSA and Pseudomonas aeruginosa. Cephalexin, an oral antibiotic targeting MSSA, is being studied for its efficacy in CF patients. Inhaled tobramycin and aztreonam lysine have been approved for chronic Pseudomonas aeruginosa infections, showing significant benefits in lung function and infection control. These treatments are part of a broader strategy to manage bacterial infections in CF patients, aiming to improve lung function and reduce exacerbations.

What other types of research exist?

Promising alternatives to Cephalexin for treating MSSA in CF patients include inhaled tobramycin, which has shown efficacy in eradicating P. aeruginosa and may be beneficial for MSSA as well. Another option is inhaled aztreonam, which has demonstrated similar outcomes to tobramycin. Oral fluoroquinolones like ciprofloxacin or levofloxacin could also be considered, especially for mild exacerbations. These alternatives offer targeted action and have been evaluated in CF contexts, making them viable options for 2024.

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Anti-bacterial

Antibiotics for Cystic Fibrosis (ASAP-CF Trial)

Phase 3

Recruiting

Led By Jonathan Rayment, MDCM

Research Sponsored by University of British Columbia

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 12 months

Awards & highlights

Pivotal Trial

Summary

This trial tests if the antibiotic Cephalexin can help children with cystic fibrosis and a specific bacterial infection (MSSA) by improving their lung function. Cephalexin has been used in previous studies for antistaphylococcal prophylaxis in infants and young children with cystic fibrosis.

Who is the study for?

This trial is for children with cystic fibrosis who have had a Staphylococcus aureus infection in the past two years, are between 3 years and under 17, weigh at least 10 kg, and haven't changed their respiratory meds recently. They can't join if they have chronic infections with certain bacteria or use daily antibiotics.

What is being tested?

The study tests whether Cephalexin (an oral antibiotic) improves lung function in stable cystic fibrosis patients compared to a placebo. It's randomized and double-blinded, meaning neither the researchers nor participants know who gets the real medicine during the two-week trial.

What are the potential side effects?

Cephalexin may cause allergic reactions, digestive issues like diarrhea or nausea, skin rashes, or rarely more serious effects like kidney problems or severe allergic reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The relative change in LCI2.5 between day 0 and day 14 (relative change = [LCI2.5 at day 14-LCI2.5 at day 0]/LCI2.5 at day 0).

Secondary study objectives

Absolute change in the CFQ-R(R) between day 0 and day 14.

Relative change in LCI5 between day 0 and day 14.

Side effects data

From 2013 Phase 2 & 3 trial • 2265 Patients • NCT00729937

21%

Nausea

16%

Headache

15%

Diarrhoea

14%

Abscess

10%

Vomiting

Dizziness

Abdominal pain upper

Constipation

Pyrexia

Rash

Cellulitis

100%

80%

60%

40%

20%

Study treatment Arm

Abscess, Placebo

Abscess, TMP/SMX

Infected Wound, TMP/SMX

Infected Wound, Clindamycin

Cellulitis, Cephalexin and TMP/SMX

Cellulitis, Cephalexin

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: CephalexinExperimental Treatment1 Intervention

Oral cephalexin (available in capsule or suspension format) dosed at 150 mg/kg/day. Doses will be administered 3 times a day for 2 weeks.

Group II: PlaceboPlacebo Group1 Intervention

The placebo will be available in both capsule and suspension format. Doses will be administered 3 times a day for 2 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cephalexin

2012

Completed Phase 4

~4070

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Cystic Fibrosis (CF) include antibiotics like Cephalexin, which target and kill specific bacteria such as MSSA, and inhaled antibiotics like tobramycin and aztreonam, which target Pseudomonas aeruginosa. These antibiotics disrupt bacterial cell walls or protein synthesis, leading to bacterial death. Additionally, inhaled hypertonic saline and DNase help to hydrate and break down mucus in the lungs, improving airway clearance. These treatments are essential for CF patients as they manage chronic lung infections, reduce inflammation, and improve lung function, thereby enhancing quality of life and survival.

Drugs, Drugs, Drugs: Current Treatment Paradigms in Cystic Fibrosis Airway Infections.Cystic fibrosis: a clinical view.Targeting the Root Cause of Cystic Fibrosis.