A Long-term Multicenter Open-label Study to Evaluate the Long-term Safety and Durability of Effect of XTMAB-16 in Patients With Pulmonary Sarcoidosis With or Without Extra pulmonary Involvement

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Xentria Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Decision date (initial)

2025-01-16

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Xentria Inc.

External identifiers

EU CT number

2023-507590-18-00

ClinicalTrials.gov

NCT06169397

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Efficacy

To evaluate the long-term safety and tolerability of XTMAB-16 in participants with pulmonary sarcoidosis with or without extra-pulmonary manifestations

Secondary objectives 4

1. 1. To evaluate the durability/maintenance of effect of XTMAB-16 as characterized by corticosteroid dosing (maintain or lower steroid dosing)
2. 2. To evaluate the durability/maintenance of effect of XTMAB-16 as characterized by changes in quality of life or pulmonary tests
3. 3. To evaluate the immunogenicity of XTMAB-16 in the context of long-term dosing
4. 4. To evaluate the long-term effect of XTMAB-16 on serum biomarkers important to cytokine pathway and granuloma formation in sarcoidosis

Conditions and MedDRA coding

Pulmonary sarcoidosis with or without extrapulmonary manifestations

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1. Participants are eligible to be included in the study if the following criteria apply: • Completion of XTMAB-16-201 study: completion of Week 12 (Part A) or Week 24 (Part B) assessments. • Participants from XTMAB-16-201 Part A and Part B should be on a stable steroid dose for at least 2 weeks prior to informed consent/Day 1 of XTMAB-16-202 (Day 1 should occur at the next scheduled dosing visit on the assigned dose frequency cohort of XTMAB-16-201 ± 2 weeks).

Exclusion criteria 1

1. 1. Participants are excluded from the study if any of the following criteria apply: • Evidence of treatment-related AEs requiring treatment discontinuation per XTMAB-16-201 protocol observed in XTMAB-16-201 study. • Evidence of treatment failure observed in XTMAB-16-201 study per protocol definition. • Pregnant or breastfeeding women or women who are planning to be pregnant during the course of the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 1. Rate of Adverse Events (AEs), including Serious Adverse Events (SAEs), and Adverse Events of Special Interests (AESIs) throughout the study duration

Secondary endpoints 7

1. 1. Total corticosteroid dose: evaluation of accumulated dose over the first 12 weeks of the study o Average daily dose o Percentage of time in study with dose ≤ 5 mg
2. 2. Proportion of participants with treatment failure and time to treatment failure, as defined in Section 5.4
3. 3. Change in quality-of-life measures from baseline to assessment timepoints throughout the study duration: o King’s Sarcoidosis Questionnaire (KSQ) – Lung Module o KSQ – General Health Status Module o Leicester Cough Questionnaire (LCQ) o Steroid toxicity questionnaire (STQ)
4. 4. Change in pulmonary function tests from baseline to assessment timepoints throughout the study duration: o Forced vital capacity (FVC)
5. 5. Number and percentage of participants who test positive for XTMAB-16 anti-drug antibody (ADA)
6. Number and percentage of participants who test positive for XTMAB-16 neutralizing antibodies (nAb)
7. 7. Absolute and percent change in biomarkers from baseline to assessment timepoints throughout the study duration: • Angiotensin converting enzyme (ACE) • Interleukin-6 (IL-6) • Soluble interleukin-2 receptor (sIL-2R) • Soluble tumor necrosis factor alpha (sTNFα) •C-reactive protein (CRP) • Interleukin-1b (IL-1b) •Calcitriol (Vitamin D1, 25)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Xentria Inc.

Sponsor organisation

Xentria Inc.

Address

2032 North Clybourn Avenue Suite 100

City

Chicago

Postcode

60614-4051

Country

United States

Scientific contact point

Organisation

Xentria Inc.

Contact name

Tom Matthews

Public contact point

Organisation

Xentria Inc.

Contact name

Noopur Singh

Third parties 4

Locations

5 EU/EEA countries · 15 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 56 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications