Investigating the efficacy and tolerability of nintedanib therapy in idiopathic-inflammatory-myopathy-related interstitial lung disease
| ISRCTN | ISRCTN10507540 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN10507540 |
- Submission date
- 24/07/2020
- Registration date
- 30/07/2020
- Last edited
- 24/05/2021
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Respiratory
Plain English summary of protocol
Background and study aims
Interstitial lung disease (ILD) is a group of lung conditions that affects the network of tissue (interstitium) that supports the air sacs of the lungs. ILD can cause stiffness in the lungs and lead to shortness of breath and death.
Idiopathic inflammatory myopathy (IIM) is a group of autoimmune disorders that cause inflammation of the muscles used for movement and lead to progressive muscle weakness. IIM can also affect non-muscular areas and the lung interstitium is the ILD is the most common non-muscular area affected. It is reported that 78% of IIM patients will develop ILD.
Idiopathic-inflammatory-myopathy-related interstitial lung disease (IIM-ILD) is frequently aggressive and may not respond to conventional therapies including glucocorticoids and immunosuppressive drugs. Meanwhile, the rapid progression of interstitial lung disease (RP-ILD) is a major cause of death in IIM patients. It is therefore necessary to search for more successful treatment for IIM-ILD.
Nintedanib has been proven effective and relatively safe in idiopathic pulmonary fibrosis and systemic-sclerosis-associated interstitial lung disease, however, its efficacy and tolerability are not known in adult idiopathic-inflammatory-myopathy-related interstitial lung disease (IIM-ILD). This study aims to assess how effective and well-tolerated nintedanib is in patients with IIM-ILD.
Who can participate?
Adult patients who regularly attend the outpatient or inpatient department of the study center with a diagnosis of IIM-ILD.
What does the study involve?
Patients who agree to participate will receive nintedanib therapy (150 mg, twice daily by mouth) in addition to standard treatment. Those who do not agree will receive standard immunosuppressive medication only.
What are the possible benefits and risks of participating?
Patients who received the nintedanib therapy may benefit from a therapeutic effect of nintedanib of slowing the progress of interstitial lung disease, reducing the chance of rapid progression, and improved survival. However, participants might experience side effects such as diarrhea and hepatic (liver) insufficiency as a result of this medication.
Where is the study run from?
The First Affiliated Hospital, College of Medicine, Zhejiang University (China)
When is the study starting and how long is it expected to run for?
From December 2017 to April 2020
Who is funding the study?
The National Natural Science Foundation of China (81701602) and Natural Science Foundation of Zhejiang Province (LQ20H100003) (China)
Who is the main contact?
Dr Junyu Liang
collinliangzju@zju.edu.cn
Contact information
Public
79 Qingchun Road
Hangzhou
310003
China
 ORCID ID |
0000-0003-1050-1274 |
|---|---|
| Phone | +86 15168302715 |
| collinliangzju@zju.edu.cn |
Study information
| Study design | Single-center interventional non-randomized real-world analysis pilot study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised study |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details below to request a participant information sheet |
| Scientific title | A real-world analysis of Nintedanib therapy in Idiopathic-inflammatory-myopathy-related Interstitial Lung Disease (NIILD): an efficacy and tolerability pilot study |
| Study acronym | NIILD |
| Study objectives | Nintedanib is efficient and relatively safe in adult idiopathic-inflammatory-myopathy-related interstitial lung disease. |
| Ethics approval(s) | Approved 28/05/2020, the Research Ethics Committee of the First Affiliated Hospital of Zhejiang University (FAHZJU) (#79 Qingchun Road, Hangzhou, Zhejiang Province, P.R.China, 310003; +86 (0)571-87236629; zyiitlunli@163.com; kjkzyyy@163.com), ref: 2020-200, 2018-224 |
| Health condition(s) or problem(s) studied | Interstitial lung disease in idiopathic inflammatory myopathy |
| Intervention | Participants who agree to participate will receive nintedanib (150 mg, twice daily, orally) in addition to traditional immunosuppressive therapy. Patients who do not agree to participate will only receive traditional immunosuprresive medications. The duration of treatment and follow-up should be at least 6 months. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Nintedanib |
| Primary outcome measure | 1. Occurrence of rapid progression of interstitial lung disease (RP-ILD), measured by the number of participants meeting the criteria of RP-ILD, assessed at the end of the follow-up. Patients with RP-ILD are defined as those presenting with progressive dyspnea and progressive hypoxemia, a worsening of interstitial change on the chest radiograph within 1 month after the initial visit or onset of respiratory symptoms. |
| Secondary outcome measures | 1. Time to death from any cause, measured through recording of survival or not and the length of follow-up, at the end of the follow-up. Cause of death will also be recorded in the follow-up such as exacerbation of interstitial lung disease, cardiopulmonary failure of unknown origin, pulmonary artery hypertension, etc. 2. Complications of pulmonary infection, measured and recorded in the process of follow-up, at the end of follow-up. Pulmonary infection will be identified based on International Statistical Classification of Diseases, 10th revision (ICD-10)-coded discharge diagnosis of community-acquired pneumonia (CAP), hospital-acquired pneumonia (HAP), pulmonary fungal infection or pulmonary infection. Responsible pathogens were recognized based on repeated cultures/smears of bronchoalveolar lavage fluid (BALF) or sputum before related treatment. 3. Difference in immunosuppressive regimen such as dose of steroid, whether the potent and expensive intravenous immunoglobulin was used, etc. measured and recorded in the process of follow-up, at the end of follow-up 4. Tolerability of nintedanib will be measured as the incidence of adverse events, the incidence of dose reduction, or discontinuation due to adverse events, at the end of follow-up |
| Overall study start date | 01/12/2017 |
| Completion date | 30/04/2020 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | over 100 |
| Total final enrolment | 151 |
| Key inclusion criteria | 1. Aged ≥18 years 2. Diagnosis of dermatomyositis, polymyositis or amyopathic dermatomyositis that meets the 2017 ACR/EULAR classification criteria 3. Attending a regular outpatient visit or hospitalization in the First Affiliated Hospital, College of Medicine, Zhejiang University |
| Key exclusion criteria | 1. Overlap syndromes with other connective tissue diseases (CTDs) 2. Attending outpatient visit or hospitalization for reasons unrelated to myositis and its complications, such as fracture, pregnancy, acquired immunodeficiency syndrome, cataract, and etc. 3. Previous use of nintedanib, or previous/present use of pirfenidone 4. Loss to follow-up without death from any cause within 6 months after the initial outpatient visit or hospitalization |
| Date of first enrolment | 01/01/2018 |
| Date of final enrolment | 31/10/2019 |
Locations
Countries of recruitment
- China
Study participating centre
79 Qingchun Road
Hangzhou
310003
China
Sponsor information
Government
83 Shuangqing Road
Haidian District
Beijing
100085
China
| Phone | +86 (0)10-62328991 |
|---|---|
| yxzhc-1@nsfc.gov.cn | |
| Website | http://www.nsfc.gov.cn/ |
"ROR" |
https://ror.org/01h0zpd94 |
Government
Provincial Natural Science Foundation Office
3rd floor
Computing Institute
Building 4
Provincial Government
33 Huancheng West Road
Hangzhou City
Hangzhou
310006
China
| Phone | +86 (0)571-88212780 |
|---|---|
| cy@zjnsf.gov.cn | |
| Website | http://www.zjnsf.gov.cn/ |
|
"ROR" |
https://ror.org/05m1p5x56 |
Funders
Funder type
Government
Government organisation / National government
- Alternative name(s)
- Chinese National Science Foundation, Natural Science Foundation of China, National Science Foundation of China, NNSF of China, NSF of China, 国家自然科学基金委员会, National Nature Science Foundation of China, Guójiā Zìrán Kēxué Jījīn Wěiyuánhuì, NSFC, NNSF, NNSFC
- Location
- China
Government organisation / Local government
- Alternative name(s)
- Zhejiang Natural Science Foundation, 浙江省自然科学基金根据国, 浙江省自然科学基金根据国, Zhejiang Provincial Natural Science Foundation, Zhejiang Provincial Natural Science Fund, ZJNSF
- Location
- China
Results and Publications
| Intention to publish date | 01/10/2020 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Other |
| Publication and dissemination plan | Planned publication in a high-impact peer-reviewed journal. |
| IPD sharing plan | Data including therapy duration, doses, adverse events, age, and sex of patients receiving nintedanib therapy will be available as supplements of the future publication. Other parts of the data in this study will be provided upon request. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | 03/02/2021 | 24/05/2021 | Yes | No |
Editorial Notes
24/05/2021: The following changes have been made:
1. Publication reference added.
2. The final enrolment number has been added from the reference.
29/07/2020: Trial’s existence confirmed by the Research Ethics Committee of the First Affiliated Hospital of Zhejiang University.
