Azithromycin a Treatment for Pulmonary Sarcoidosis

• Status: Completed
• Trial end date: 2020-06-20
• Target enrollment: 0
• Participant gender: All

Summary

Patients with sarcoidosis need treatment options that effectively control their disease without causing undesirable side effects. An appealing strategy is to repurpose existing drugs which possess beneficial immune modulating activity and are safe for long-term use. Recently, increased activity of the mTOR intracellular signalling pathway in inflammatory cells has emerged as a key driver of granulomatous inflammation in mouse models and patients with sarcoidosis. The macrolide antibiotic azithromycin directly inhibits mTOR activity in inflammatory cells, making it a prime target for drug repurposing in sarcoidosis. Azithromycin has an acceptable tolerability profile when used for long-term treatment of other chronic respiratory disease Single centre open label clinical trial of oral azithromycin 250 mg once daily for 3 months in 20-30 patients with pulmonary sarcoidosis.

The Investigator have opted for an open label study because this will be the first study of azithromycin in sarcoidosis. Trial assessments will be performed according to standards of Good Clinical Practice with assessments at baseline, 1, and 3 months. All other clinical care, investigations, and treatment (if indicated) will remain the responsibility of the treating physician and based on clinical MDT consensus decisions.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hull and East Yorkshire Hospitals NHS Trust; Hull University Teaching Hospitals NHS Trust
• Treatments: Azithromycin

Criteria

Inclusion Criteria:

• • Males or females, of any race, between 18 and 80 years of age, inclusive;

• Able to speak, read, and understand English;

• Able to provide written informed consent;

• Able to communicate effectively with the Investigator and other study centre personnel and agree to comply with the study procedures and restrictions.

• Clinician diagnosis of pulmonary sarcoidosis;

• If a female of child-bearing potential (i.e., have not undergone a hysterectomy or bilateral oophorectomy) or not post-menopausal (defined as no menses for at least 12 months), agree to use acceptable birth control (defined in Section 6.3) from screening through to the follow up visit;

Exclusion Criteria:

• • Hypersensitivity to azithromycin or another macrolide antibiotic (e.g. erythromycin, clarithromycin) or excipients (see 7.4)

• History of signficant cardiac arrhythmia

• Personal or family history of congenital long QT syndrome;

• Prolonged QTc interval on 12-lead ECG

• Signficant liver disease

• Evidence of acute bacterial infection

• Clinically significant bronchiectasis

• Requiring concomitant therapy with prohibited medications (see Section 7.5)

• Pregnant or breastfeeding;

• Treatment with an investigational drug or biologic within 30 days preceding the first dose of study medication or plans to take another investigational drug or biologic within 30 days of study completion;

• Other severe, acute, or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the Investigator or Sponsor, would make the subject inappropriate for entry into this trial.