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An investigator-initiated clinical trial of TM5614 for SSc-ILD

Phase 2
Recruiting
Conditions
Interstitial lung disease associated with systemic sclerosis
D012595
Registration Number
JPRN-jRCT2021230022
Lead Sponsor
Asano Yoshihide
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
Not specified
Target Recruitment
50
Inclusion Criteria

1. Patients 18 years of age or older with written consent
2. Patients with systemic sclerosis who meet the 2013 ACR/EULAR classification criteria for systemic sclerosis
3. Within 7 years of the onset of systemic sclerosis (first appearance of systemic sclerosis-related symptoms other than Raynaud's phenomenon)
4. Interstitial lung disease associated with systemic sclerosis confirmed by high-resolution CT performed within 12 months, and lung involvement greater than 10% in extent
5. %FVC of 40% or more
6. %DLco of 30% or more but less than 90%
7. No oral administration of PSL 10 mg/day or more within 2 weeks prior to obtaining consent (less than 10 mg/day during the study period, dose cannot be increased)
8. No treatment with antifibrotic drugs or tocilizumab within 4 weeks
9. Not receiving rituximab treatment within 6 months
10. Receiving immunosuppressive drugs (azathioprine, cyclophosphamide, or mycophenolate mofetil) at least 6 months prior to the date of consent, mycophenolate mofetil at least 3 months prior to the date of consent, and no dose change of mycophenolate mofetil within 3 months

Exclusion Criteria

1. Patients with an estimated right ventricular systolic pressure greater than 35 mmHg on echocardiography performed within 12 months prior to enrollment, or with pulmonary arterial hypertension requiring treatment
2. Patients with FEV1/FVC less than 0.7
3. Patients with AST, ALT, or bilirubin exceeding 1.5 times the upper limit of the institutional reference value
4. Patients with creatinine clearance (Cockcroft-Gault method) less than 30 mL/min
5. Patients with poorly controlled hypertension (systolic pressure of 160 mmHg or more, or diastolic pressure of 100 mmHg or more)
6. Patients with a history of myocardial infarction or unstable angina pectoris within 6 months prior to obtaining consent
7. Patients with chronic liver failure
8. Patients with a history of serious trauma or surgery within 3 months prior to consent
9. Patients on anticoagulation therapy
10. Patients receiving more than 3 antiplatelet agents
11. Patients with a history of bleeding within 6 months prior to obtaining consent
12. Patients with a PT-INR greater than 2 and an APTT greater than 1.5 times the upper limit of the institutional standard
13. Patients with a history of thromboembolism within 1 year prior to obtaining consent
14. Patients with clinical signs of malabsorption or need for central venous nutrition
15. Patients who have participated in another clinical trial or clinical study within the past 6 months and have received any drug, or are currently participating in another clinical trial or clinical study
16. Patients who are pregnant, lactating, or planning to become pregnant
17. Female patients of childbearing age who are unable to use effective contraception from the date of consent until 3 months after completion of the study
18. Patients with a history of scleroderma renal crisis
19. Other patients who are judged by the investigator (subinvestigator) to be ineligible for this clinical trial.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Change in % forced vital capacity (%FVC) (%) at 48 weeks compared to pre-observation period
Secondary Outcome Measures
NameTimeMethod
Efficacy<br>1. Absolute change in %FVC (mL) at 48 weeks compared to pre-observation period<br>2. Percentage of inhibition of %FVC decline at 48 weeks (a decrease of less than 10% compared to pre-observation period is defined as a reduction in decline).<br>3. Percentage of decrease in %FVC at 48 weeks (percentage of %FVC based on %FVC in pre-observation period)<br>4. Percentage of patients with improved %FVC at 48 weeks (an increase in %FVC greater than 3.3% is defined as an improvement)<br>5. Absolute change in %DLco (mL) at 48 weeks<br>6. Absolute change in mRSS at 48 weeks<br>7. Percentage of patients with improvement of 3 or more in mRSS at 48 weeks<br>8. Absolute change in net burden of digital ulcers at 48 weeks<br>9. Absolute change in SHAQ total score at 48 weeks<br>10. Proportion of patients requiring additional treatment for interstitial lung disease at 48 weeks*.<br>11. Mortality (all causes of death)<br>Safety<br>Adverse Events
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