A Phase 2, Multicenter, Randomised, Double-Blind, Placebo-Controlled-Parallel-Group study to determine how safe, effective and tolerable MT-7117 is in subjects with Diffuse Cutaneous Systemic Sclerosis

Phase 1

• Conditions: Diffuse Cutaneous Systemic Sclerosis; Therapeutic area: Diseases [C] - Immune System Diseases [C20]; MedDRA version: 21.0Level: LLTClassification code 10012977Term: Diffuse systemic sclerosisSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

• Registration Number: EUCTR2020-000134-17-DE
• Lead Sponsor: Mitsubishi Tanabe Pharma America (MTPA), Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2020-10-06
• Last Update Posted: 18 June 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 76

Inclusion Criteria

Subjects who meet all the following criteria will be considered eligible to participate in the study:
1.Must provide signed and dated informed consent form (ICF) to participate in the study. Subjects must be able to (in the judgment of the Investigator) understand the nature of the study and all risks involved with participation in the study. Subjects must be willing to cooperate and comply with all protocol restrictions and procedures including study visits.
2.Male or female age = 18 at screening with documented diagnosis of systemic sclerosis (SSc), as defined using the 2013 ACR/European League Against Rheumatism (EULAR) criteria ( Appendix 1).
3.Has diffuse cutaneous form of SSc according to Leroy and Medsger's criteria ( Appendix 1).
4.Disease duration = 5 years from the first non-Raynaud's phenomenon manifestation.
5.Has an modified Rodnan Skin Score (mRSS) of 15 to 45 units at screening and have clinical skin involvement proximal and distal to the elbows, knees, or both or any truncal involvement, with or without face involvement.
6.If disease duration is > 24 months defined as time from the first non-Raynaud phenomenon manifestation, subject must fulfill at least 1 of the criteria listed below that are indicatives of active disease at screening:
a.A documentation of new skin involvement that occurred within the past 9 months, or
b.Increase in mRSS = 3 units within the past 9 months, or
c.Presence of tendon friction rubs (TFRs) or,
d.C- reactive protein = 6 mg/L, or
e.Erythrocyte sedimentation rate = 28 mm/hr, or
f.Platelet count = 330 x 10^9/L (330,000/microliter).
7.Willing to follow restrictions regarding concomitant medications that are described in Appendix 2.
8.Female subjects who are non-lactating and have a negative urine pregnancy test at baseline visit prior to receiving the first dose of study drug.
9.Female subjects of childbearing potential and male subjects with partner of child-bearing potential currently using/willing to use two effective methods of contraception including barrier method as described in Appendix 3.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 63
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 13

Exclusion Criteria

1.Has a history or presence of rheumatic autoimmune diseases other than dcSSc unless the dominant features of the current disease are from dcSSc, as determined by the Investigator after discussion with the Medical Monitor.
2.Has a pulmonary disease with FVC = 50% of predicted at time of screening.
3.Has a diagnosis of clinically significant resting pulmonary hypertension (if exceeding estimated right ventricular systolic pressure of > 40 mmHg estimated by transthoracic echocardiography [unless the right heart catheterization is normal within the last 6 months] or mean pulmonary artery pressure > 30 mmHg as measured by right heart catheterization) and requires treatment with more than one oral medication.
4.Has a cardiac abnormality such as left ventricular failure with ejection fraction < 45%,significant arrhythmia, congestive heart failure (New York Heart Association Class II-IV), unstable angina, uncontrolled hypertension, or symptomatic pericardial effusion at screening.
5.Has a history of myocardial infarction in the last 26 weeks prior to screening.
6.Has a history of renal crisis within the past 52 weeks prior to screening.
7.Has a documented history of chronic kidney disease [stage 4-5, an estimated glomerular filtration rate (eGFR) < 30 ml/min at screening]
8.Presence or history of hepatobiliary disease at screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor
9. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP) = 2.0 × upper limit of normal (ULN), or total bilirubin>1.5 × ULN at screening
10. Has a history or presence of clinically significant disease not relatedto SSc [neurologic, renal, endocrine, gastrointestinal cardiovascular, hepatic, dermatologic, hematological, musculoskeletal, genitourinary, thromboembolic, advanced arteriosclerosis, hyperthyroidism, moderate to severe hypertension, immunologic disease, pulmonary (e.g., uncontrolled asthma, emphysema, chronic obstructive pulmonary disease) or any other disorder] as determined by the Investigator at screening
11. Has a history or presence of serious or clinically significant (as judged by the Investigator) psychiatric disorder including but not limited to, anxiety disorder, depression, and bipolar disorder that may make a subject unlikely or unable to complete the study or comply with study procedures and requirements, impact the subject's ability to participate in the study and/or interfere with the study evaluation and/or safety of the subject
12. Has any clinically significant disease or laboratory abnormality judged to be clinically significant by the Investigator and which may interfere with the study evaluation and/or safety of the subject at screening. Laboratory abnormalities include but not limited to any of the followings: Hemoglobin < 9 g/dL; WBC < 3,000/mm3 (< 3 x 10^9/L); platelets < 100,000/mm3 (<100 x 10^9/L)
13. Has a history of positive hepatitis B surface antigen, hepatitis C antibody, except for documented cure for the hepatitis B virus (HBV), defined as sustained, undetectable HBsAg and HBV DNA in serum and adequately treated hepatitis C virus (HCV) with documentation of sustained virologic response defined as undetectable HCV RNA at least 12 weeks after the EOT
14. Has a history of positive human immunodeficiency virus (HIV)
15. Has a history of melanoma, familial melanoma (defined as having 2 or more first-degree relatives

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified