KU-12K-001

JRCT ID: jRCT2071220063

Registered date:21/10/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Systemic Sclerosis
• Date of first enrollment: 09/02/2023
• Target sample size: 15
• Study type: Interventional
• Intervention(s): Randomized oral administration of the invetigational drug(sirolimus 0.35-2.8mg) and placebo daily for 16 weeks

Outcome(s)

• Primary Outcome: Suspected adverse reaction rate

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

(1)Patients aged 20 years or older at the time of obtaining informed consent
(2)American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) classification criteria for systemic sclerosis satisfied.
(3)One or more digital ulcer excluding due to calcification at the screening point
(4)Performance Status of 0-2
(5)Patients who have not been able to improve digital ulcers by treatment including existing medical treatment or catheter vascular treatment for more than 4 weeks before administration of the investigational drug
(6)Patients who are able to give consent in writing

Exclude criteria

(1)Patients with one of the following lung disease findings
20% and more area of interstitial disease lesions in lung revealed by high-resolution computed tomography
Patients requiring treatment for interstitial lung disease
Respiratory test reveal following data
Vital capacity (%VC):75% and less,Forced vital capacity (%FVC):70% and less,Diffusing capacity of the lung for carbon monooxide (%DLCO):75% and less
(2)Patients with clinical symptoms due to interstitial lung disease such as cough, dyspnea, or fever
(3)Patients with a history of scleroderma renal crisis
(4)Patients with pulmonary hypertension or heart disease associated with pulmonary hypertension
(5)Patients with skin ulcers (wounds) requiring treatment for causes other than systemic scleroderma
(6)hematocrit < 30% or blood platelet count < 75,000/mm^3 or Absolute neutrophil count < 1,000/mm^3 or white blood cell count < 3,000/mm3
(7)AST or ALT elevated more than 3 times the upper limit of normal
(8)Child-Pugh class B or C
(9)Patients with organ failure (lungs, kidneys, etc.) and abnormal clinical laboratory values of any of the following
Urine protein>=3 , creatinine elevated more than 1.5 times the upper limit of normal , eGFR < 30mL/min/1.73m^2
(10)Patients with severe malabsorption(unintended weight loss of more than 15% in 6 months prior to investigational drug administration)
(11)Poorly controlled diabetes or poorly controlled hypertension or poorly controlled hyperlipidemia
(12)Patients with a history of hypersensitivity to sirolimus, sirolimus ingredients, or sirolimus derivatives (everolimus, temsirolimus)(History of allergy to sirolimus, or additive substance)
(13)Patients with immunodeficiency such as HIV, primary immunodeficiency, etc.
(14)HBsAg-positive patients, HBs antibody or HBc antibody-positive patients with detectable HBV-DNA, or patients with active hepatitis C (except for HCV antibody-positive patients who are inactive and have normal liver function)
(15)Patients with a history of tuberculosis or suspected tuberculosis infection(QFT/T-SPOT positive patients)
(16)Patients with severe infections requiring treatment or suspected infections
(17)Has a compromised host status
(18)Patients with a history of myocardial infarction or angina pectoris or stroke, patients with NYHA class II-IV congestive heart failure , patients with symptomatic arrhythmias requiring treatment
(19)Patients with a history of malignant tumors other than squamous or basal cell skin cancer or cervical cancer within 2 years prior to obtaining an informed consent.
(20)Patients who have operated within 8 weeks prior to the start of clinical trial administration or who are scheduled to undergo surgery(surgery requiring intrabody invasion or more than 3 stitches, including biopsy)
(21)Patients who have a history of drug or alcohol poisoning within 5 years prior to obtaining an informed consent, or who have a history of drug or alcohol abuse within one year prior to obtaining an informed consent
(22)Use of any investigational drug or device in other clinical studies within 30 days before obtaining an informed consent
(23)Patients who are pregnant, breastfeeding, or who are unable to provide adequate contraception from the time of obtaining informed consent to 12 weeks after the last dose of the investigational product
(24)Patients judged inappropriate by the investigators

Related Information

• Primary Sponsor: Goto Mizuki
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development