CSL Behring Sclero XIII

• Register: ClinicalTrials.gov
• Last refreshed on: 13 June 2016
• Main ID: NCT02551042
• Date of registration: 17/12/2014
• Prospective Registration: Yes
• Primary sponsor: University College, London
• Public title: CSL Behring Sclero XIII
• Scientific title: A Phase II, Double-blind, Randomized, Placebo-controlled Study to Investigate Pharmacokinetics (PK), Safety and Efficacy of Intravenous Factor XIII Treatment in Patients With Systemic Sclerosis
• Date of first enrolment: September 2015
• Target sample size: 26
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT02551042
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United Kingdom

Contacts

• Name: Christopher Denton, PhD

• Name: Christopher Denton, PhD
• Affiliation: Royal Free London NHS Foundation Trust

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male and female adults.

- Subjects with a diagnosis of systemic sclerosis (scleroderma, SSc) according to the
2013 American College of Rheumatology European League Against Rheumatism (ACR EULAR)
classification criteria. They will be classified according to LeRoy criteria as
limited or diffuse subset.

- Females of childbearing potential must be willing to use a reliable form of medically
acceptable contraception and have a negative pregnancy test.

- Subjects will have serological status for hepatitis A and B assessed at screening.

- Patients who have given their free and informed consent. -= 18 years.

Exclusion Criteria:

Participants must:

- Be willing to use an effective method of contraception (hormonal or barrier method of
birth control; abstinence) from the time consent is signed until 6 weeks after
treatment discontinuation (females of childbearing potential and males)

- Have a negative pregnancy test within 7 days prior to being registered for trial
treatment (females of childbearing potential). NOTE: Subjects are considered not of
child bearing potential if they are surgically sterile (i.e. they have undergone a
hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are
postmenopausal.

- Not be breastfeeding (females).

Participants must not:

- Have allergies to excipients of the investigational medicinal product (IMP) and
placebo

- Have uncontrolled systemic hypertension as evidenced by sitting systolic blood
pressure = 160 mmHg or sitting diastolic blood pressure = 100 mmHg.

- Have portal hypertension or chronic liver disease defined as mild to severe hepatic
impairment (Child-Pugh Class A-C). Subjects positive for Hepatitis C with evidence of
active viral replication on sensitive polymerase chain reaction (PCR) testing are
also excluded.

- Have hepatic dysfunction, defined as aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) = 3 times the upper limit of the normal range (× ULN) at the
Screening Visit.

- Have chronic renal insufficiency as defined by a serum creatinine = 2.5 mg/dL (= 221
micromol/L) or requires dialysis.

- Have a haemoglobin concentration = 10 g/dL (= 100 g/L) at the Screening Visit.

- Have a history of left-sided heart disease and/or clinically significant cardiac
disease, including but not limited to any of the following: aortic or mitral valve
disease (stenosis or regurgitation) defined as more than minimum aortic insufficiency
and more than moderate mitral regurgitation (stenosis or regurgitation= grade 1);
pericardial constriction; restrictive or congestive cardiomyopathy; left ventricular
ejection fraction = 40 % by multiple gated acquisition scan (MUGA), angiography, or
echocardiography; left ventricular shortening fraction = 22 % by echocardiography; or
symptomatic coronary disease with demonstrable ischemia.

- Have a history of malignancies within 5 years of Screening Visit with the exception
of localized skin or cervical carcinomas.

- Have psychiatric, addictive, or other disorder that compromises the ability to give
informed consent for participating in this study. This includes subjects with a
recent history of abusing alcohol or illicit drugs.

- Be receiving ongoing treatment with hyperbaric oxygen

- Have pulmonary artery hypertension (PAH)

- Have received IV Iloprost within the last 2 months

- Have been treated with sympathectomy or toxin botulinum A within the last 3 months

- Have had thrombosis, stroke, pulmonary embolism or myocardial infarction in the last
6 months

- Have a diagnosis of diabetes mellitus requiring dietary restriction of carbohydrate.

- Be on a low sodium diet on medical advice.

- Be participating in another clinical trial involving an investigational medicinal
product.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Systemic Sclerosis

Intervention(s)

Drug: Fibrogammin®P, coagulation factor XIII concentrate (Human)

Drug: 0.9% sodium chloride

Primary Outcome(s)

Primary outcome assessed by skin involvement measured with modified Rodnan skin score [Time Frame: 24 weeks]

Primary outcome assessed by skin involvement measured with Raynaud condition score [Time Frame: 24 weeks]

Secondary Outcome(s)

Complete healing of digital ulcers (DU) [Time Frame: 24 weeks]

Digital ulcer worsening: Need of oral or parenteral antibiotic [Time Frame: 24 weeks]

Digital ulcer worsening: Gangrene [Time Frame: 24 weeks]

Digital ulcer worsening: hospitalisation required [Time Frame: 24 weeks]

Digital ulcer worsening: Amputation [Time Frame: 24 weeks]

Digital ulcer worsening: Digital ulcer infection [Time Frame: 24 weeks]

Digital ulcer worsening: Need of intravenous (IV) Iloprost : this is considered treatment failure [Time Frame: 24 weeks]

Digital ulcer worsening: Need of local sympathectomy [Time Frame: 24 weeks]

Digital ulcer worsening: Need of toxin Botulinum A [Time Frame: 24 weeks]

Number of new digital ulcers (DU) [Time Frame: 24 weeks]

Digital ulcer pain assessment [Time Frame: 24 weeks]

Digital ulcer worsening: surgical intervention required [Time Frame: 24 weeks]

Pulmonary function measured by pulmonary function test [Time Frame: 24 weeks]

Hand function measured with Cochin hand function [Time Frame: 24 weeks]

Quality of life measured with Short Form-36 (SF-36) quality of life questionnaire [Time Frame: 24 weeks]

Secondary ID(s)

2014-001101-40

13/0417

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

CSL Behring