Safety, Efficacy and Pharmacokinetics of C21 in Subjects With IPF

NCT ID: NCT04533022

Last Updated: 2025-05-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 52 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-11-13
• Study Completion Date: 2024-03-30

Brief Summary

This trial is a multi-centre, open-label, single-arm phase 2 trial investigating the safety, efficacy and pharmacokinetics of C21 in subjects with idiopathic pulmonary fibrosis.

Conditions

Idiopathic Pulmonary Fibrosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

C21

C21 100 mg BID (twice daily)

Group Type: EXPERIMENTAL

C21

Intervention Type: DRUG

C21 100 mg BID (twice daily)

Interventions

C21

C21 100 mg BID (twice daily)

Intervention Type: DRUG

Other Intervention Names

Buloxibutid, Compound 21

Eligibility Criteria

Inclusion Criteria

1. Written informed consent, consistent with ICH-GCP R2 and local laws, obtained before the initiation of any trial related procedure

2. A diagnosis of IPF within 5 years prior to Visit 1, as per either ATS/ERS/JRS/ATLAT/Fleischner guidelines

3. Age ≥40 years

4. Forced vital capacity (FVC) ≥60% predicted at Visit 1 (specifically for UK: FVC ≥80% predicted at Visit 1)

5. Forced expiratory volume in the first sec (FEV1)/FVC ratio ≥0.7 prebronchodilator at Visit 1

6. Oxygen saturation (SpO2) >85% by pulse oximetry while breathing ambient air at rest at Visit 1

7. High-resolution computed tomography (HRCT) within 36 months prior to Visit 1 with central reading demonstrating either a or b, and c:

a. A pattern consistent with usual interstitial pneumonitis (UIP) according to ATS/ERS/JRS/ALAT or Fleischner guidelines i. UIP ii. Probable UIP or b. A pattern indeterminate for UIP according to either ATS/ERS/JRS/ALAT or Fleischner guidelines and a historical biopsy consistent with IPF c. Extent of fibrosis > extent of emphysema

8. Fully vaccinated against COVID-19 prior to screening (Visit 1). Subjects are considered fully vaccinated for COVID-19 ≥14 days after they have received vaccination dose(s) according to local label

Exclusion Criteria

1. Previous use of antifibrotic treatment for an interstitial lung disease (e.g. nintedanib or pirfenidone) for > 6 months

2. Smoking (including e-cigarettes) within 6 months prior to Visit 1

3. Body mass index (BMI) >35 or <18

4. IPF exacerbation within 3 months prior to Visit 1:

• Acute worsening or development of dyspnoea typically <1 month duration
• Computed tomography with new bilateral ground-glass opacity and/or consolidation superimposed on a background pattern consistent with usual interstitial pneumonia pattern (if no previous computed tomography is available, the qualifier "new" can be dropped)
• Deterioration not fully explained by cardiac failure or fluid overload

5. Concurrent serious medical condition with special attention to cardiac or ophthalmic conditions (e.g. contraindications to cataract surgery) which in the opinion of the investigator makes the subject inappropriate for this trial

6. Malignancy within the past 5 years with the exception of in situ removal of basal cell carcinoma and cervical intraepithelial neoplasia grade I

7. Treatment with any of the medications listed below within 4 weeks prior to Visit 1:

• Cytochrome p450 (CYP) 3A4 inducers (e.g. rifampicin, phenytoin, St. John's Wort)
• CYP3A4 inhibitors (e.g. clarithromycin, ketoconazole, nefazodone, itraconazole, ritonavir)
• Medicines that are substrates of CYP1A2, CYP3A4 or CYP2C9 with a narrow therapeutic range
• Experimental drugs
• Any systemic immunosuppressive therapies other than:
• Inhaled corticosteroids which can be used throughout the trial period provided the dose is kept stable
• Corticosteroids for the treatment of acute exacerbations
• The continuation of stable doses of ≤15 mg daily doses of prednisolone

8. Treatment with any of the medications listed below within 2 weeks prior to Visit 1:

• Proton pump inhibitors (PPI's) more than once daily
• Histamine H2 receptor antagonists (H2RA's)
• Sulphasalazine and rosuvastatin
• High dose breast cancer resistance protein sensitive substrates (other than sulphasalazine or rosuvastatin)

9. Any of the following findings at Visit 1:

• Prolonged QTcF (QT interval with Fridericia's correction) (>450 ms), cardiac arrhythmias or any clinically significant abnormalities in the resting ECG, as judged by the Investigator
• Positive results for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCVAb) or human immunodeficiency virus 1+2 antigen/antibody (HIV 1+2 Ag/Ab)
• Positive serum pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin)

10. Inability to generate lung function data at Visit 1 meeting the minimum standards of the ATS/ERS 2005 guideline, as determined by central review

11. Clinically significant abnormal laboratory value at Visit 1 indicating a potential risk for the subject if enrolled in the trial as evaluated by the investigator

12. Pregnant or breast-feeding female subjects

13. Female subjects of childbearing potential not willing to use contraceptive methods

14. Male subjects not willing to use contraceptive methods

15. Subjects not willing to adhere to dietary restrictions during the trial period

16. Participation in any other interventional trial during the trial period

17. Subjects known or suspected of not being able to comply with this trial protocol (e.g. due to alcoholism, drug dependency or psychological disorder)

18. Discontinuation or change of previous antifibrotic treatment (e.g. nintedanib or pirfenidone) due to disease progression

• Minimum Eligible Age: 40 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Orphan Reach Ltd.

INDUSTRY

Sponsor Role: collaborator

Vicore Pharma AB

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Joanna Porter, MD

Role: PRINCIPAL_INVESTIGATOR

Respiratory Medicine, University College Hospital

Locations

AMCMET Medical College and Sheth LG General Hospital

Ahmedabad, Gujarat, India

Unity Hospital

Surat, Gujarat, India

The Bhatia Hospital

Mumbai, Maharashtra, India

Grant Government Medical Collage and Sir J.J. Group of Hospitals

Mumbai, Maharashtra, India

N. K. P. Salve Institute of Medical Sciences & Research Centre and Lata Mangeshkar Hospital

Nagpur, Maharashtra, India

Ace Hospital & Research Center

Pune, Maharashtra, India

Oyster & Pearl Hospitals

Pune, Maharashtra, India

Hindusthan Hospital

Coimbatore, Tamil Nadu, India

Jawaharlal Nehru Medical College - Aligarh Muslim University

Aligarh, Uttar Pradesh, India

Midland Healthcare and Research Centre

Lucknow, Uttar Pradesh, India

Apollo Spectra Hospitals (Apollo Speciality Hospital Pvt. Ltd.)

Kanpur, , India

Clinical Hospital for Emergency Medical Care n.a. N.V. Solovyev

Yaroslavl, , Russia

MNCE City Clinical Hospital

Kharkiv, , Ukraine

Lviv National Medical University

Lviv, , Ukraine

Odessa Regional Hospital

Odesa, , Ukraine

Private Small Scale Enterprise Medical Center 'PULSE'

Vinnytsia, , Ukraine

University Hospital Birmingham

Birmingham, , United Kingdom

Royal Brompton Hospital

London, , United Kingdom

University College Hospital

London, , United Kingdom

University College London Hospitals

London, , United Kingdom

Medicines Evaluation Unit

Manchester, , United Kingdom

Countries

India; Russia; Ukraine; United Kingdom

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2020-000822-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VP-C21-005

Identifier Type: -

Identifier Source: org_study_id