Diffuse cutaneous systemic sclerosis (dcSSc) MedDRA version: 19.0 Level: PT Classification code 10042953 Term: Systemic sclerosis System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
Conditions
Interventions
Product Name: Terguride
Pharmaceutical Form: Tablet
INN or Proposed INN: TERGURIDE HYDROGENMALEATE
CAS Number: 37686-85-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration n
Sponsors
medac GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Each subject must meet all of the following criteria to be enrolled in this study (criterion #7 must also be met for inclusion in the open-label extension phase of the study): 1. Is a male or female =18 years of age. 2. Fulfills the ACR/EULAR 2013 criteria for classification of SSc 3. Has diffuse cutaneous involvement (defined by skin fibrosis in at least 1 of the following sites: upper arms, thorax, abdomen, or thighs) according to scleroderma classification criteria (LeRoy et al 1988), confirmed independently by a second investigator who is unaware of the assessment of the first investigator. 4. Has a baseline mRSS between 10 and 25 units, inclusive. 5. Has had onset of the first non-Raynaud’s manifestation of SSc within 36 months of screening. 6. Is willing and able to comply with all aspects of the study; in particular, has the ability to attend study visits and assessments at least until the end of the double-blind treatment phase. 7. If a male subject is capable of reproduction or a female subject is of childbearing potential, he/she consents to practice sexual abstinence (Sexual abstinence refers to refraining from heterosexual intercourse during the entire period of risk associated with the study treatment. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject.) or use another highly effective method of contraception (Pearl index =65 years) yes F.1.3.1 Number of subjects for this age range 14
Exclusion criteria
Exclusion criteria: Subjects meeting any of the following criteria will be excluded from the study: 1. Has abnormal hematological values prior to baseline (Visit 2). 2. Has abnormal liver function tests prior to baseline (Visit 2). 3. Has abnormal renal function tests prior to baseline (Visit 2). 4. Has any known renal crisis history. 5. Has cardiac abnormalities at screening (Visit 1). 6. Has any of the following lung criteria at screening (Visit 1): • Forced vital capacity (FVC) <40% predicted • Diffusing capacity of the lung for carbon monoxide (DLCO) <30% predicted. 7. Has a previous diagnosis of severe pulmonary arterial hypertension (PAH). 8. Has malabsorption requiring parenteral nutrition at screening (Visit 1). 9. Has had treatment with putative disease-modifying agents within 8 weeks of screening. 10. Has had treatment with cyclophosphamide within 26 weeks of screening (Visit 1). 11. Has had treatment with rituximab within 52 weeks of screening (Visit 1). 12. Has a known acute or uncontrolled chronic infection (eg, hepatitis B or C, tuberculosis, or human immunodeficiency virus [HIV]). 13. History of hematopoietic stem cell transplantation or planned hematopoietic stem cell transplantation within the next 12 months, or total lymphoid irradiation. 14. Has any concurrent medical condition(s) that, in the opinion of the investigator, may confound the results of the study. 15. Is pregnant or lactating.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to assess the efficacy of terguride compared to placebo in terms of absolute change in the mRSS during the double-blind treatment phase in subjects with dcSSc.;Secondary Objective: The secondary and other objectives include the following: • To assess the efficacy of terguride compared to placebo in terms of impact on skin thickening, visceral involvement, functional status, quality of life, and pharmacodynamic parameters during the double-blind treatment phase. • To assess the safety of terguride compared to placebo in a 52-week double-blind treatment phase. • To assess the efficacy and safety of terguride in the open-label, 52-week open-label extension phase. • To assess the prognostic value of CYP2D6 polymorphism with regard to the HIwT terguride dosage.;Primary end point(s): The primary efficacy endpoint is the absolute change in mRSS between baseline and Week 52 of the double-blind treatment phase.;Timepoint(s) of evaluation of this end point: Week 52 of double blind phase | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • The absolute change in total score of the HAQ-DI between baseline and Week 52 of the double-blind treatment phase. • The binary mRSS response defined as improvement of mRSS by at least 30% (relative) or 5 points (absolute) between baseline and Week 52 of the double-blind treatment phase.;Timepoint(s) of evaluation of this end point: Week 52 of double blind phase | — |
Countries
Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Romania, Switzerland, United Kingdom, United States
Contacts
Public ContactProject Manager Clinical Research
medac GmbH
Outcome results
None listed