A Clinical Study to Evaluate the Efficacy and Safety of REGEND001 Cell Therapy on Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic Pulmonary Fibrosis Clinical Trial

Official title:

A Multi-center, Randomized, Double-Blinded, Parallel and Placebo-Controlled Phase II Clinical Study to Evaluate the Efficacy and Safety of REGEND001 Cell Therapy in Idiopathic Pulmonary Fibrosis (IPF) Patients

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06081621
• Other study ID #: REGEND001-IPF-231-V1.1
• Status: Recruiting
• Phase: Phase 2
• Start date: June 30, 2023
• Est. completion date: July 2025

Study information

• Verified date: May 2024
• Source: Regend Therapeutics
• Contact: Zuojun Xu, M.D.
• Phone: +86-10-69156114
• Email: xuzj[@]hotmail.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Idiopathic pulmonary fibrosis (IPF) is a serious chronic (long term) disease with injury of lung tissues. REGEND001 is a cell therapy product, made from bronchial basal cells with ability to regenerate lung tissue, is promising to IPF treatment. This is a multi-center, randomized, double-blinded, parallel and placebo-controlled phase II clinical study to evaluate the efficacy and safety of REGEND001 in IPF patients.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: July 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 40 Years to 75 Years

Eligibility

Inclusion Criteria:

• Male or female, aged between 40 to 75;

• Subjects diagnosed with IPF according to guidelines for the diagnosis of idiopathic pulmonary fibrosis 2022 edition;

• Subjects tolerant to bronchofiberscope;

• Subjects tolerant to test of lung function;

• Subjects able to voluntarily sign the informed consent and cooperate with the completion of pulmonary function tests;

Exclusion Criteria:

• Female subject who is pregnant, nursing, or planning to be pregnant in half a year after using this product (or male subjects planning to have a pregnant spouse);

• At the time of screening, subject who is positive in each of treponema pallidum antibody (TP-Ab), human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody test, except the following: (1) Hepatitis B virus carriers (only HBsAg positive, no hepatitis symptoms and signs, all liver function tests are normal); (2) Cured hepatitis C patients with negative result in HCV ribonucleic acid (RNA) test.

• Subject with malignant tumors or a history of malignant tumors;

• Subject with severe anemia, poorly controlled agranulocytosis and thrombocytopenia at screening;

• Subject at risk of suicide or has a history of mental illness or epilepsy at the time of screening;

• Subject with severe arrhythmias or atrioventricular block of degree II or above, shown by 12-lead Electrocardiogram (ECG);

• Subject who participated in other interventional clinical trials in the past 3 months;

• Subject assessed as inappropriate to participate in this clinical trial by investigators.

Related Conditions & MeSH terms

• Fibrosis
• Idiopathic Pulmonary Fibrosis
• Pulmonary Fibrosis

Intervention

Biological:
• REGEND001
REGEND001: 1-1.5×10^6 bronchial basal cells/kg administrated by bronchoscopy.
• Placebo
Placebo: Sodium chloride injection administrated by bronchoscopy.

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital, Chinese Academy of Medical Sciences	Beijing	Beijing
China	Renji Hospital, Shanghai Jiaotong University School Of Medicine	Shanghai	Shanghai
China	Ruijin Hospital, Shanghai Jiaotong University School Of Medicine	Shanghai	Shanghai

Sponsors (4)

Lead Sponsor	Collaborator
Regend Therapeutics	Peking Union Medical College Hospital, RenJi Hospital, Ruijin Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change from baseline in lung ventilatory capacity	Forced vital capacity (FVC) and forced expiratory volume in one second (FEV1) will be used to evaluate the lung ventilatory capacity. FVC indicates the volume of air that can forcibly be blown out after full inspiration. FEV1 is the volume of breath exhaled with effort in one second.	12 and 24 weeks after treatment
Other	Progression-free survival (PFS)	PFS refers to the time from randomization or initiation of treatment to the occurrence of disease progression or death.	Within 24 weeks after treatment
Other	Change from baseline in St. George's respiratory questionnaire (SGRQ) scale	Quality of life was assessed by St. George's respiratory questionnaire (SGRQ) scale. Total score, ranged from 0 to 100, is the sum of points from all items. A higher value represents a worse outcome.	12 and 24 weeks after treatment
Other	Change from baseline in 6-minute-walk test (6MWT)	The 6MWT is a commonly used test for the objective assessment of functional exercise capacity by testing the distance patients can walk at the fastest speed within 6 minutes.	12 and 24 weeks after treatment
Other	Time from enrollment to all-cause death	Time from enrollment to all-cause death is used to evaluate the overall survival of the population.	up to 24 weeks(first period), 5 years (second period).
Other	Blood oxygen saturation	Blood oxygen saturation is the measure of how much oxygen is traveling through body in red blood cells.	12 and 24 weeks after treatment
Other	Change from baseline in images of lung by high resolution computed tomography (HR-CT)	HR-CT images of lung will be analyzed to indicate the change of pulmonary structure.	12 and 24 weeks after treatment
Other	Change from baseline in C-reactive protein (CRP)	The level of CRP increases when there's inflammation in the body.	12 and 24 weeks after treatment
Other	Time to acute exacerbations of idiopathic pulmonary fibrosis (AE-IPF)	AE-IPF is an often deadly complication of IPF, which is defined as an acute, clinically significant respiratory deterioration characterized by evidence of new widespread alveolar abnormality.	Within 24 weeks after treatment
Other	Temperature	Number of cases with abnormal body temperature.	Within 24 weeks after treatment
Other	Breathing	Number of cases with abnormal breathing.	Within 24 weeks after treatment
Other	Pulse	Number of cases with abnormal pulse.	Within 24 weeks after treatment
Other	Blood pressure	Number of cases with abnormal blood pressure.	Within 24 weeks after treatment
Other	Symptoms, physical examination	Number of cases with abnormal physical examination	Within 24 weeks after treatment
Other	12-lead ECG	Number of cases with abnormal 12-lead Electrocardiogram (ECG).	Within 24 weeks after treatment
Other	Blood routine	Number of cases with abnormal laboratory test results	Within 24 weeks after treatment
Other	Liver & Kidney function check	Number of cases with abnormal results in Liver & Kidney function check	Within 24 weeks after treatment
Other	Blood sugar	Number of cases with abnormal results	Within 24 weeks after treatment
Other	Function of blood clotting	Number of cases with abnormal function of blood clotting.	Within 24 weeks after treatment
Other	Antibody testing for autoimmune diseases	Antibodies related to autoimmune diseases are tested for safety assessment	Within 24 weeks after treatment
Other	Carcinoembryonic antigen (CEA)	CEA is a tumor marker used for early diagnosis of lung cancer.Clinically significant changes of this markers will be assessed.	Within 24 weeks after treatment
Other	Neuron-specific enolase (NSE)	NSE is a tumor marker significantly elevated in small cell lung cancer. Clinically significant changes of this marker will be assessed	Within 24 weeks after treatment
Other	Cytokeratin-19-fragment (CYFRA21-1)	CYFRA21-1 is a tumor marker which is valuable for the pathological classification and prognosis evaluation of lung cancer. Clinically significant changes of this marker will be assessed	Within 24 weeks after treatment
Other	Squamous cell carcinoma antigen (SCC)	SCC is a specific marker for lung squamous cell carcinoma. Clinically significant changes of this marker will be assessed	Within 24 weeks after treatment
Other	Other cases of adverse effects	Other cases of adverse effects will be recorded and compared.	Within 24 weeks after treatment
Primary	The ratio of subjects with improvement of lung carbon oxide diffusion function (DLCO)	DLCO is measured by the single-breath method. Improvement is defined as an increase in DLCO from baseline.	12 and 24 weeks after treatment
Secondary	Change from baseline in lung diffusing capacity	DLCO will be used to evaluate the lung diffusing capacity. DLCO test refers to the diffusing capacity for carbon monoxide in the lungs. It's a type of pulmonary function test that helps to assess how well gas is exchanged between the lungs and the bloodstream.	12 and 24 weeks after treatment