Open Label Extension (OLE) for the Patients Treated in the ISD002-P144-07 Study

NCT00781053 | Completed Phase 2

• 2 other identifiers: ISD003-P144-082008-001265-28
• Study Type: interventional
• Target: 38
• Locations: 6 countries
• Sites: 17

Brief Summary

Transforming growth factor-beta 1 is consistently over expressed in most fibrotic diseases and displays a variety of profibrotic effects in fibroblasts(25, 26). Activation of TGF-beta receptors induces the activation of several kinase signalling cascades leading to the phosphorylation of SMAD proteins as well as to the activation of SMAD-independent kinases that collectively activate ECM synthesis and fibroblast growth and differentiation into myofibroblasts. TGF-beta 1 i one of the main mediators in the fibrotic process, associated to both scarring and a long list of pathologies related to chronic inflammation and which affect all type of organs and tissues. An increase in TGF-beta1 mRNA and protein levels has been described in these processes. Peptide 144 (P144)is a acetic salt of a 14mer peptide from human TGF-beta1 type III receptor (betaglycan). P144 TGF-beta1-inhibitor has been specifically designed to block the interaction between TGF-beta1 and TGF-beta1 type III receptor, thus blocking its biological effects. P144 has shown significant antifibrotic activity in mice receiving repeated sucutaneous injections of bleomycin, a widely accepted animal model of human scleroderma, and could contribute to the development. The aim of the study is to asses the long-term safety of topical application of P144 cream in the treatment of skin fibrosis in patients with systemic sclerosis in an extension open-label treatment period of 6 additional months.

Conditions

Skin Fibrosis

Keywords

skin fibrosis; systemic scleroderma; systemic sclerosis; p144; orphan drug

Outcome Measures

Primary Outcomes (1)

• Assess the long-term safety of digna P144 cream topically administered in skin manifestations of systemic sclerosis patients.

  6 months

Secondary Outcomes (1)

• Quality o life assessment, skin induration and hardness. In a subgroup of patients pharmacokinetic and elasticity will be measured.

  6 months

Study Arms (1)

P144 cream

EXPERIMENTAL

P144 cream 0.03% will be used once a day during the whole extension period of 6 months.

Drug: P144 cream

Interventions

P144 cream DRUG

P144 cream 0.03% will be used once a day during the whole extension period of 6 months. The patient will apply the cream by him/herself or with a help of a person uniformly in a 10% maximum affected surface until absorption

P144 cream

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Previous participation and finalization of treatment period of the ISD002-P144-07 study without clinical relevant safety issues medically evaluated by the investigator.
• For female subjects with childbearing potential: use of a known highly effective method of birth control, defined as those which results in a low failure rate: i.e. less 1% per year, (contraceptive pills, intrauterine contraceptive device, implants, vasectomized partner or sexual abstinence), for at least the extension study period and one month after the end of the extension study.
• For male subjects with partners of childbearing potential:
• use of appropriate contraceptive methods (vasectomy, condoms or sexual abstinence), for at least the extension study period and one month after the end of the extension study.
• Stable therapy for at least one month, except in the case of patients under treatment with putative disease modifying agents (immunosupressants like cyclophosphamide, or azathioprine) that will need at least three months of stable therapy, without the expectation of treatment modifications during the trial period..
• Capable of understanding and willing to provide signed and dated written voluntary informed consent before any protocol specific procedures are performed

You may not qualify if:

• Other skin diseases affecting the treatment area which could have been diagnosed during the ISD002-P144-07 study.
• Woman became pregnant during the ISD002-P144-07 study.
• Any new diagnosis since the ISD002-P144-07 study which includes: systemic sclerosis sine scleroderma, localized escleroderma, eosinophilic fascitis, or eosinophilia myalgia syndrome; any other definable connective tissue disease, such as rheumatoid arthritis, systemic lupus erythematosus, polymyositis, or dermatomyositis; clinically significant overlap condition; significant existing internal organ damage as defined in the guidelines for clinical trials in systemic sclerosis; history of skin cancer; other skin diseases affecting the treatment area.
• Substantial history of environmental exposure to tainted rapeseed oil, vinyl chloride, L- tryptophan, bleomycin, trichoroethylene, or silica; PUVA therapy within 1 month of study drug initiation; concurrent interventional therapy that might independently influence outcome of trial, such as D-penicillamine, cyclosporine, methotrexate, interferon-γ or photopheresis; topical corticosteroids treatment affecting the selected area; cosmetics over the treatment area.

Sponsors & Collaborators

• ISDIN: lead
• Digna Biotech S.L.: collaborator

Study Sites (17)

Herz- und Rheumazentrum Kerckhoff-Klinik

Bad Hauheim, Bad Hauheim, 61231, Germany

Location

Klinikum der Johan Wolfgang Goethe-Universitat

Frankfurt, Frankfurt, 60590, Germany

Location

Klinik und Poliklinik für Dermatologie und Vererologie

Cologne, Köln, 50937, Germany

Location

Allergie-Centrum-Charité, Abteilung für

Berlin, State of Berlin, 10117, Germany

Location

Immunologiai es Reumatologiai Klinika

Pécs, Pécs, H-7621, Hungary

Location

Azienda Ospedaliera Universitaria Careggi

Florence, 50139, Italy

Location

Centrum Mirada

Bialystok, Bialystok, 15-297, Poland

Location

Samodzielny Publiczny Szpital Kliniczny

Katowice, Katowice, Poland

Location

Katedra i Klinika Raumatologizno

Poznan, Poznan, 61-545, Poland

Location

Gabinet Lekarski Internistyczno- Reumatologiezny

Wroclaw, Wroclaw, 53-137, Poland

Location

Klinika Ftizjopneumonologii SAM

Zabrze, Zabrze, 41-803, Poland

Location

Hospital Clinic i Provincial de Barcelona

Barcelona, Barcelona, 08036, Spain

Location

Hospital 12 de Octubre

Madrid, Madrid, 28041, Spain

Location

Clinica de Navarra

Pamplona, Pamplona, 31008, Spain

Location

Chapel Allerton Hospital

Leeds, Leeds, NW3 2QG, United Kingdom

Location

Royal Free Hospital

London, London, Nw3 2QG, United Kingdom

Location

University Hospital Aintree

Liverpool, L9 7AL, United Kingdom

Location

Related Links

• ISDIN exists to take care of the skin with reliable and scientifically tested products.

MeSH Terms

Conditions

Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Marco Matucci, MD, PhD

  University of Florence

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: October 24, 2008
• First Posted: October 28, 2008
• Study Start: July 1, 2008
• Primary Completion: December 1, 2010
• Study Completion: December 1, 2010
• Last Updated: February 11, 2013

Record last verified: 2013-02

Locations

DE (4); HU (1); ES (3); IT (1); PL (5); GB (3)