A Trial to Evaluate the Safety, Tolerability and Preliminary Efficacy of NCR101 in the Treatment of Subjects With Interstitial Lung Disease

A Phase I/II, Open Label, Single Arm, Dose Escalation and Dose Expansion Clinical Trial to Evaluate the Safety, Tolerability and Efficacy of Human Induced Pluripotent Stem Cell Derived Mesenchymal Stromal Cells (NCR101) Injection in the Treatment of Subjects With Interstitial Lung Disease

The target of this trial is to evaluate the safety, tolerability and preliminary efficacy of NCR101 in the treatment of subjects with interstitial lung disease. The trial contains Single ascending dose（SAD） and Multiple ascending dose（MAD）. Subjects will receive at least 1 dose of NCR101.

Study Overview

• Status: Not yet recruiting
• Conditions: Interstitial Lung Disease (ILD)
• Intervention / Treatment: Biological: NCR101 injection

Detailed Description

Interstitial lung disease (ILD) is a group of heterogeneous diseases, including idiopathic pulmonary fibrosis(IPF), hypersensitivity pneumonia, sarcoidosis, and connective tissue-associated interstitial lung disease (CTD-ILD), which is characterized by alveolar unit inflammation and/or fibrinization, leading to the destruction of lung structure and loss of function. In the absence of effective treatment, most ILD may develop diffuse pulmonary fibrosis, leading to structural destruction of lung tissue, diffusion dysfunction, and progressive respiratory failure and death. ILD causes a heavy disease and socio-economic burden, and has become a major public health problem. The target of treatment for interstitial lung disease depends on the type of disease and its clinical manifestations. At present, the existing drugs and treatments such as Pirfenidone and Nintedanib can only alleviate the symptoms of IPF or delay the progression of the disease, and the survival improvement is not obvious, and there is no treatment on the market can cure IPF, Patients with connective tissue have a high burden of lung complications, are prone to ILD complications, and the diagnosis and treatment of ILD are difficult for different CTDs. The evidence to guide the optimization of treatment is limited. Therefore, novel drugs with great therapeutic potential are urgently needed for ILD patients.

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Tao Ren, M.Pharm; Phone Number: 021-64369181; Email: yqli@nuwacell.com
• Study Contact Backup: Name: Tao Ren, MD; Phone Number: 021-64369181

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age>18 years old (including threshold), gender not limited;
• Meets the diagnosis of interstitial lung disease;
• Screening for interstitial lung disease diagnosed by pulmonary imaging within the first 6 months;
• For patients undergoing glucocorticoid therapy at the time of screening: the background treatment remained stable during the study period;
• If the patient is undergoing anti-fibrotic therapy (pirfenidone or nintedanib) at the time of screening, they must have received a stable dose for at least 4 weeks at the time of screening and plan to continue receiving this background therapy steadily after entering this study;
• Patients of childbearing age (male and female) must agree to take effective non pharmacological contraceptive measures during the trial period and within 6 months after the last use of medication:
• Voluntarily sign an informed consent form and be able to cooperate in completing research related procedures and checks.

Exclusion Criteria:

• Individuals with a history of severe allergies or allergies to the main active ingredients or excipients of the investigational drug;
• Individuals who have been hospitalized for three or more times due to acute exacerbation of ILD or other respiratory diseases within the previous year prior to screening;
• Individuals with a history of mechanical ventilation (invasive or non-invasive) within the past month prior to screening, or those currently requiring oxygen therapy (oxygen therapy duration>15 hours/day);
• Individuals who have contracted infectious pneumonia or require intravenous anti-infective treatment within the previous month; Previous history of tuberculosis
• Patients with other serious respiratory diseases, such as asthma, those with airway obstruction (FEV1/FVC<0.7 before using bronchodilators), and those with other clinically significant abnormalities in the lungs;
• Within the first 3 days of enrollment, use high-dose corticosteroids (equivalent to methylprednisolone>240 mg/day) or irregularly use systemic corticosteroids;

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NCR101 injection; Cohort1:Low dose NCR101 injection; Cohort2:High dose NCR101 injection	Biological: NCR101 injection; Subjects will receive one NCR101 injection in Single Ascending Dose（SAD）and receive 4 NCR101 injections in Multiple Ascending Dose(SAD).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Event(AE) or Serious Adverse Event(SAE)	Number of participants with treatment-related adverse events or serious adverse events as assessed by CTCAE v5.0	4 weeks after administration of SAD and multiple ascending dose (MAD)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Diffusion capacity of the lungs for carbon monoxide(DLCO)	Change from baseline in the result of DLCO	4 weeks, 12 weeks, 24 weeks, 48 weeks
Forced vital capacity (FVC)	Change from baseline in the result of FVC	4 weeks, 12 weeks, 24 weeks, 48 weeks
Forced expiratory volume in 1 second (FEV1)	Change from baseline in the result of FEV1	4 weeks, 12 weeks, 24 weeks, 48 weeks
6-min walk test (6MWT)	Change from baseline in the result of 6MWT	4 weeks, 12 weeks, 24 weeks, 48 weeks
St George's Respiratory Questionnaire (SGRQ)	Change from baseline in the result of SGRQ	4 weeks, 12 weeks, 24 weeks, 48 weeks
Dyspnea index	Change from baseline in the result of Dyspnea index	4 weeks, 12 weeks, 24 weeks, 48 weeks
high-resolution computed tomography (HRCT) Score	Change from baseline in the result of	24 weeks, 48 weeks

Collaborators and Investigators

• Sponsor: Nuwacell Biotechnologies Co., Ltd.
• Investigators: Principal Investigator: Tao Ren, MD, Shanghai 6th People's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): March 1, 2025
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): September 1, 2028

Study Registration Dates

• First Submitted: February 9, 2025
• First Submitted That Met QC Criteria: February 9, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 9, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Lung Diseases, Interstitial
• Other Study ID Numbers: NCR101-1001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No