Efficacy and Safety of Belumosudil in Subjects With Diffuse Cutaneous Systemic Sclerosis

  • STATUS
    Recruiting
  • End date
    Oct 1, 2022
  • participants needed
    15
  • sponsor
    Kadmon Corporation, LLC
Updated on 31 July 2021

Summary

This is a phase 2, open-label, single-cohort, multicenter trial of belumosudil in subjects with Diffuse Cutaneous Systemic Sclerosis (dcSSc). An estimated total of 12 to 15 subjects will receive belumosudil 200 mg administered orally (PO) twice daily (BID) for 52 weeks. The primary analysis will be at 24 weeks.

Description

The primary objective of this phase 2, open-label, single-cohort, multicenter trial is to evaluate the efficacy of belumosudil 200 mg BID using the Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) after 24 weeks of therapy. The duration of the study will be approximately 14 months (4 weeks for screening, 52 weeks of dosing period, and 4 weeks of Follow-up)

Subjects who have signed an Institutional Review Board/Independent Ethics Committee-(IRB/IEC)-approved informed consent form (ICF) and met all of the inclusion/exclusion criteria will be enrolled. A total of 12 to 15 to subjects at approximately 5 to 6 sites will receive belumosudil 200 mg in tablet form administered PO BID for 52 weeks. The total duration of the study is approximately 14 months: a 4-week screening period, a 52-week treatment period, and a 4-week follow-up.

The primary endpoint will be analyzed using Week 24 data.

Efficacy will be assessed throughout the 52-week dosing period using:

  • Composite Response Index in Systemic Sclerosis (CRISS)
  • Modified Rodnan Skin Score (mRSS)
  • Pulmonary Function Tests (PFTs)
  • Physician Global Assessment
  • Patient Global Assessment

Safety will be assessed throughout the study and will include:.

  • Physical examinations (PEs)
  • Vital sign measurements
  • Weight measurements
  • Blood sample collection for hematology and chemistry; urinalysis
  • Electrocardiograms (ECGs)
  • Adverse event (AE) assessments
  • Concomitant medication assessments
  • Pregnancy testing for females of childbearing potential.

Reasons for discontinuation of treatment because of toxicity will be documented. Careful monitoring of all toxicities will be carried out. Dosing can be reduced 1 dose level. If the dose is not tolerated, then the subject will be discontinued from the study. If there is a interruption of dosing, after 14 days the subject will be discontinued from the study.

Subjects will be given a study drug diary to record the details of each dose of belumosudil 200 mg. Diaries will be dispense/collected on each visit. Compliance with dosing will be confirmed using subject diaries, which will be examined at each visit by site staff to determine if dosing is as instructed per protocol and follow-up.

A 4-Week Safety Follow-up Visit will occur 28 days ( 3 days) after the last dose of study drug.

Details
Condition Diffuse Scleroderma, diffuse cutaneous systemic sclerosis
Treatment Belumosudil
Clinical Study IdentifierNCT04680975
SponsorKadmon Corporation, LLC
Last Modified on31 July 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Male and female subjects 18 years old with the diagnosis of dcSSc according to the 2013 American College of Rheumatology and European League Against Rheumatism
Must have disease duration (defined as interval from first non Raynaud disease manifestation) of 6 years
Must have mRSS of 15 but 40
Must have active disease as determined by the Principal Investigator within the 6 months prior to screening
Adequate organ and bone marrow functions evaluated during the 28 days prior to enrollment as follows
Absolute neutrophil count 1.5 10^9/L
Platelet count 100 10^9/L
Total bilirubin 1.0 upper limit of normal (ULN)
Alanine aminotransferase (ALT) and aspartate aminotransferase (AST), and serum creatinine 1.5 ULN
Female subjects of childbearing potential have a negative pregnancy test at screening. Females of childbearing potential are defined as sexually mature women without prior hysterectomy or who have had any evidence of menses in the past 12 months. However, women who have been amenorrheic for 12 or more months are still considered to be of childbearing potential if the amenorrhea is possibly due to prior chemotherapy, anti-estrogens, or ovarian suppression
Women of childbearing potential (i.e., menstruating women) must have a negative urine pregnancy test (positive urine tests are to be confirmed by serum test) documented within the 24-hour period prior to the first dose of study drug
Sexually active women of childbearing potential enrolled in the study must agree to use two forms of accepted methods of contraception during the course of the study and for 3 months after their last dose of study drug. Effective birth control includes (i) intrauterine device plus 1 barrier method; (ii) on stable doses of hormonal contraception for at least 3 months (e.g., oral, injectable, implant, transdermal) plus 1 barrier method; or (iii) 2 barrier methods. Effective barrier methods are male or female condoms, diaphragms, and spermicides (creams or gels that contain a chemical to kill sperm), or a vasectomized partner
For male patients who are sexually active and who are partners of premenopausal women: agreement to use 2 forms of contraception as in criterion number 6b above during the treatment period and for at least 3 months after the last dose of study drug
Male subjects must not donate sperm for 3 months after last dose of study drug
Able to provide written informed consent prior to the performance of any study-specific procedures

Exclusion Criteria

Subject has corrected QT interval using Fridericia's formula (QTcF) > 450 ms
Ongoing use or current use of concomitant medication known to have the potential for QTc prolongation
Female subject who is pregnant or breastfeeding
Participated in another study with an investigational drug within 28 days of study entry (for studies involving biologics, within 3 half-lives of the biologic)
History or other evidence of severe illness or any other conditions that would make the subject, in the opinion of the Investigator, unsuitable for the study
Chronic heart failure with New York Heart Association Classes II, III, or IV
Positive human immunodeficiency virus (HIV) test
Acute or chronic liver disease (e.g., cirrhosis)
Active hepatitis C virus (HCV), hepatitis B virus (HBV), or positive whole blood tuberculin test
Diagnosed with any malignancy within 3 years of enrollment, with the exception of basal cell or completely resected squamous cell carcinoma of the skin, resected in situ cervical malignancy, resected breast ductal carcinoma in situ, or low risk prostate cancer after curative resection
Has had previous exposure to belumosudil or known allergy/sensitivity to belumosudil, or any other Rho-associated Protein Kinase-2 (ROCK2) inhibitor
Scleroderma renal crisis within 4 months prior to enrollment
FVC 50% Predicted
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