Safety, Efficacy and Pharmacokinetics of C21 in Subjects With IPF

NCT04533022 | Completed Phase 2 Results DMC

• 2 other identifiers: VP-C21-0052020-000822-24
• Study Type: interventional
• Target: 52
• Locations: 4 countries
• Sites: 21

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

Keywords

Idiopathic Pulmonary Fibrosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events Occurring Over the Trial Period

  Number of participants with adverse events occurring over the trial period incl. number of participants with any TEAE, serious TEAEs, TEAEs leading to withdrawal from trial, TEAEs leading to discontinuation of treatment, treatment-related TEAEs leading to discontinuation of treatment, TEAEs leading to death, and serious treatment-related TEAEs. Adverse events were recorded from signing of informed consent until end of trial. Nature and frequency of adverse events are presented in details in the adverse events section.

  Trial period of 36 weeks

Secondary Outcomes (11)

• Change From Baseline in Forced Vital Capacity (Non-imputed Data)

  12, 24, and 36 weeks

• Change From Baseline in Forced Vital Capacity (Imputed Data)

  12, 24, and 36 weeks

• Rate of Forced Vital Capacity Decline Over Time, FAS

  12, 24, and 36 weeks

• Plasma Concentration of C21 Evaluated in a Sub-set of Subjects, Day 1

  Prior to dosing, 30 min post dose, 1 h post dose, 2 h post dose, 3 h post dose, and 4 h post dose on Day 1

• Plasma Concentration of C21 Evaluated in a Sub-set of Subjects, Week 12

  Prior to dosing, 30 min post dose, 1 h post dose, 2 h post dose, 3 h post dose, and 4 h post dose at Week 12

Study Arms (1)

C21

EXPERIMENTAL

C21 100 mg BID (twice daily)

Drug: C21

Interventions

C21 DRUG

C21 100 mg BID (twice daily)

Also known as: Buloxibutid, Compound 21

C21

Eligibility Criteria

• Age: 40 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent, consistent with ICH-GCP R2 and local laws, obtained before the initiation of any trial related procedure
• A diagnosis of IPF within 5 years prior to Visit 1, as per either ATS/ERS/JRS/ATLAT/Fleischner guidelines
• Age ≥40 years
• Forced vital capacity (FVC) ≥60% predicted at Visit 1 (specifically for UK: FVC ≥80% predicted at Visit 1)
• Forced expiratory volume in the first sec (FEV1)/FVC ratio ≥0.7 prebronchodilator at Visit 1
• Oxygen saturation (SpO2) \>85% by pulse oximetry while breathing ambient air at rest at Visit 1
• 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
• 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

You may not qualify if:

• Previous use of antifibrotic treatment for an interstitial lung disease (e.g. nintedanib or pirfenidone) for \> 6 months
• Smoking (including e-cigarettes) within 6 months prior to Visit 1
• Body mass index (BMI) \>35 or \<18
• 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
• 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
• Malignancy within the past 5 years with the exception of in situ removal of basal cell carcinoma and cervical intraepithelial neoplasia grade I
• 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:

Sponsors & Collaborators

• Vicore Pharma AB: lead
• Orphan Reach Ltd.: collaborator

Study Sites (21)

AMCMET Medical College and Sheth LG General Hospital

Ahmedabad, Gujarat, 380008, India

Location

Unity Hospital

Surat, Gujarat, 395010, India

Location

The Bhatia Hospital

Mumbai, Maharashtra, 400007, India

Location

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

Mumbai, Maharashtra, 400008, India

Location

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

Nagpur, Maharashtra, 440019, India

Location

Ace Hospital & Research Center

Pune, Maharashtra, 411004, India

Location

Oyster & Pearl Hospitals

Pune, Maharashtra, 411005, India

Location

Hindusthan Hospital

Coimbatore, Tamil Nadu, 641028, India

Location

Jawaharlal Nehru Medical College - Aligarh Muslim University

Aligarh, Uttar Pradesh, 202002, India

Location

Midland Healthcare and Research Centre

Lucknow, Uttar Pradesh, India

Location

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

Kanpur, 208001, India

Location

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

Yaroslavl, Russia

Location

MNCE City Clinical Hospital

Kharkiv, 61124, Ukraine

Location

Lviv National Medical University

Lviv, 79010, Ukraine

Location

Odessa Regional Hospital

Odesa, 65025, Ukraine

Location

Private Small Scale Enterprise Medical Center 'PULSE'

Vinnytsia, 21001, Ukraine

Location

University Hospital Birmingham

Birmingham, B15 2TH, United Kingdom

Location

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

Location

University College Hospital

London, United Kingdom

Location

University College London Hospitals

London, United Kingdom

Location

Medicines Evaluation Unit

Manchester, M23 9QZ, United Kingdom

Location

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis

Interventions

compound 21

Condition Hierarchy (Ancestors)

Pulmonary Fibrosis; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases

Results Point of Contact

• Title: Chief medical officer Bertil Lindmark
• Organization: Vicore Pharma AB

info@vicorepharma.com

+46 707661505

Study Officials

• Joanna Porter, MD

  Respiratory Medicine, University College Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

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

Study Record Dates

• First Submitted: August 26, 2020
• First Posted: August 31, 2020
• Study Start: November 13, 2020
• Primary Completion: March 30, 2024
• Study Completion: March 30, 2024
• Last Updated: May 23, 2025
• Results First Posted: May 23, 2025

Record last verified: 2025-05

Locations

UA (4); IN (11); RU (1); GB (5)