Azithromycin a Treatment for Pulmonary Sarcoidosis

NCT04020380 | Completed Phase 2

• 1 other identifier: SARC2019
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Sarcoidosis, Pulmonary

Outcome Measures

Primary Outcomes (1)

• change in 24 hr cough counts from baseline

  24n hr cough monitoring with ambulatory cough monitor will be performed at baseline and at 1mth and 3 months post treatment

  3 months

Secondary Outcomes (4)

• Mean Change in severity of cough from baseline

  3 months

• Mean Change in urge to cough from baseline

  3 months

• Mean Change in Leicester cough questionnaire total score from baseline

  3 months

• Mean Change in Kings sarcoidosis questionnaire total score from baseline

  3 months

Study Arms (1)

Azithromycin 250 mg

EXPERIMENTAL

Azithromycin, 250 mg capsules once a day for a total of 3 months

Drug: Azithromycin 250Mg Capsule

Interventions

Azithromycin 250Mg Capsule DRUG

250 mg OD

Azithromycin 250 mg

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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.

Sponsors & Collaborators

• Hull University Teaching Hospitals NHS Trust: lead

Study Sites (1)

Respiratory Medicine Clinical trials Unit

Cottingham, East Yorkshire, HU16 5JQ, United Kingdom

Location

MeSH Terms

Conditions

Sarcoidosis, Pulmonary

Interventions

Azithromycin

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Sarcoidosis; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases

Intervention Hierarchy (Ancestors)

Erythromycin; Macrolides; Polyketides; Lactones; Organic Chemicals

Study Officials

• Simon Hart, MD

  Hull University Teaching Hospitals NHS Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Model Details: single centre, open label study

Study Record Dates

• First Submitted: July 11, 2019
• First Posted: July 16, 2019
• Study Start: September 6, 2019
• Primary Completion: June 20, 2020
• Study Completion: June 20, 2020
• Last Updated: July 16, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)