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Completed Scleroderma Clinical Trials: Positive Results

Author: Shelley Ensz. Scleroderma is highly variable. See Types of Scleroderma. Read Disclaimer
AIMSPRO
Bosentan/Tracleer
Combination Therapies
Electrophoresis with Ximedon
Halofuginone
Infliximab
IVIg
Methotrexate
Photopheresis
Pirfenidone/ESBRIET
PUVA/UVA-1 and Phototherapy
PVAC
Rituximab (Rituxan)
Stem Cell Transplant
Urokinase Therapy

AIMSPRO by Daval

Treatment of diffuse systemic sclerosis (dSSc) with hyperimmune caprine serum (AIMSPRO): a phase II double-blind placebo-controlled trial. These results confirm tolerability and safety of this novel biological agent in established dSSc and the value of a placebo treated control group in small clinical trials evaluating skin disease in SSc is confirmed. PubMed, Ann Rheum Dis, 2014 Jan 1;73(1):56-61. (Also see Diffuse Scleroderma)

Daval AIMSPRO as monotherapy in scleroderma yields positive results. The double-blind placebo-controlled phase II clinical study to assess the safety and tolerability of AIMSPRO as a monotherapy in Established Diffuse Cutaneous Systemic Sclerosis (Scleroderma) yielded positive results. PBR Clinical Trials. 10/11/2011.

Bosentan, Tracleer

Overview
Tracleer for Digital Ulcers
Tracleer for Heart Involvement in SSc
Tracleer for Pulmonary Fibrosis
Tracleer for Pulmonary Hypertension
Tracleer for Raynaud's
Tracleer for Scleroderma Renal Crisis
Tracleer for Skin Fibrosis
Also See

Combination Therapies

Therapeutic strategy combining intravenous cyclophosphamide followed by oral azathioprine to treat worsening interstitial lung disease associated with systemic sclerosis: a retrospective multicenter open-label study. Intravenous CYC followed by oral maintenance immunosuppressive therapy for worsening ILD was well tolerated and was associated with stable or improved PFT in 70% and 51.8% of SSc patients at 6 months and 2 years, respectively. Bérezné A. J Rheumatol. 2008 Jun;35(6):1064-72.

Electrophoresis with Ximedon

Closed Study, Positive Results

Discovery May Lead to Novel Treatments for Autoimmune and Chronic Inflammatory Diseases. For years, doctors have used IVIG to treat patients with autoimmune and chronic inflammatory diseases, but just how the therapy works has remained a mystery. Some researchers have shown that IVIG works, in part, by activating a receptor known as FcγRIIb, which then suppresses auto-antibody-mediated inflammation. Newswise. 01/26/07. (Also see Medications, Dermatomyositis, and Myasthenia Gravis)

A double blind placebo-controlled trial performed in 56 patients with scleroderma systematica showed effectiveness of a domestic drug ximedon--a pirimidine compound--applied during electrophoresis on the affected skin, limbs. The addition of ximedon-electrophoresis to rehabilitation program for scleroderma systematica improved the condition in 77.8%, microhemo-circulation in 72.2%, reduced the area of the affected skin by 9.8% (p < 0.05), skin induration in 55.6% of patients. PubMed, Vopr Kurortol Fizioter Lech Fiz Kult 2002 Sep-Oct;(5):33-6.

Halofuginone

Closed Study, Positive Results

Halofuginone to treat fibrosis in chronic graft-versus-host disease and scleroderma. The results of the human studies provide basis for using halofuginone treatment for dermal fibrosis. As a first step toward future treatment of internal organ involvement, an oral administration study was performed in which halofuginone was well tolerated and plasma levels surpassed the predicted therapeutic exposure. PubMed, Biol Blood Marrow Transplant. 2003 Jul;9(7):417-25. (Also see Skin Fibrosis and Diseases Similar to Scleroderma)

Infliximab

Open-label pilor study completed. Positive results.

In diffuse cutaneous systemic sclerosis (dcSSc), infliximab did not show clear benefit at 26 weeks but was associated with clinical stabilisation and a fall in two laboratory markers of collagen synthesis. The frequency of suspected infusion reactions may warrant additional immunosuppression in any future studies in systemic sclerosis. Annals of the rheumatic diseases, 2009, vol. 68, no9.

Intravenous Immunoglobulins (IVIg)

Intravenous Immunoglobulin (IVIG) May Be an Effective Therapy for Refractory, Active Diffuse Cutaneous Systemic Sclerosis (dcSSc). IVIG may be an effective adjunctive therapy for active dcSSc in patients failing other therapies. PubMed, J Rheumatol, 11/29/2014. (Also see IVIg)

Mainstream Medications for Scleroderma (IVIg). ISN.

Methotrexate

Tumor necrosis factor blockers may not cause cancer after all. Study finds no increased risk of lymphoma or tumors associated with anti-TNF therapy over methotrexate use among rheumatoid arthritis patients. EurekAlert! 08/31/06. (Also see Rheumatoid Arthritis)

Interstitial Pneumonitis Associated with Infliximab Therapy. Interstitial pneumonitis is a well documented, rare complication of methotrexate (MTX). Infliximab may potentiate pulmonary toxicity of MTX. J Rheumatol 2006;33:1189-93.

Small study completed. Positive results, but not statistically significant.

Evaluation of oral methotrexate in the treatment of systemic sclerosis. It was concluded that methotrexate for 6 months only provides subjective improvement, and further studies after 1 year of treatment with methotrexate are recommended. PubMed, Int J Dermatol. 2007 Feb;46(2):218-23. (Also see Medications)

Placebo controlled trial of methotrexate in systemic sclerosis. Clinical improvement following treatment was observed in 33.33% of the patient in MTX group but none in placebo group, but this difference was not statistically significant. Anorexia, nausea and occasional vomiting were common side effects in MTX group and subsided in most cases with the passage of time despite the continuation of therapy. PubMed, Mymensingh Med J. 2005 Jan;14(1):71-4. (Also see Medications)

Methotrexate pneumonitis: review of the literature and histopathological findings in nine patients. Pneumonitis is a serious and unpredictable side-effect of treatment with methotrexate (MTX) that may become life-threatening. The clinical and histological features of nine cases of MTX pneumonitis are reported and the literature reviewed. Eur Respir J. 2000 Feb;15(2):373-81.

Methotrexate (Rheumatrex ®) Open enrollment in clinical trial SCTC

About Methotrexate (Rheumatrex, Trexall) MedicineNet

Photopheresis

No current studies.

A randomized, double-blind, placebo-controlled trial of photopheresis in systemic sclerosis. Photopheresis induced significant improvement of skin and joint involvement in patients with scleroderma of recent onset; however, any effect when compared with sham treatment and a possible placebo effect may be modest. PubMed, J Am Acad Dermatol. 2006 May;54(5):793-799.

Pirfenidone (Esbriet®) by Genentech

Positive Results. Esbriet was FDA approved in 2014 for Idiopathic Pulmonary Fibrosis.

An Open–label, Phase II Study of the Safety and Tolerability of Pirfenidone in Patients with Scleroderma–associated Interstitial Lung Disease (SSc-ILD): the LOTUSS Trial. Pirfenidone showed an acceptable tolerability profile in SSc–ILD, although a longer titration may be associated with better tolerability. PubMed, J Rheumatol, 07/01/2016.

Learn about Esbriet® (pirfenidone). Esbriet has been approved outside of the United States since 2011. More than 27,000 patients have taken Esbriet worldwide. The effectiveness and safety of Esbriet were studied in three clinical trials of patients with IPF. Genentech.

ESBRIET Found To Be Safe in Patients with Interstitial Lung Disease Associated With Systemic Sclerosis. Dr. Dinesh Khanna from the University of Michigan Health System recently presented at the European League Against Rheumatism (EULAR) 2015 Annual European Congress of Rheumatology in Rome new positive safety data regarding ESBRIET for the treatment of interstitial lung disease (ILD). Pulmonary Fibrosis News, 07/07/2015.

Genentech’s ESBRIET Recommended in ATS Guidelines for IPF Treatment. FDA approval of Esbriet in the Fall of 2014 brought much-needed therapeutic options to those who suffer from IPF. Esbriet has been shown in different Phase III clinical trials to slow the decline of lung function in IPF patients and consequently disease progression. Pulmonary Hypertension News, 08/12/2015.

Pirfenidone: A Novel Agent for the Treatment of Idiopathic Pulmonary Fibrosis. Pirfenidone (Esbriet) is the first agent specifically developed for the treatment of IPF. It has been approved for use in Europe and Japan, but not in the US. PubMed, The Annals of Pharmacotherapy, 02/13/2013.

Positive Data From Lung Study May Lead to First FDA Approved Treatment for Pulmonary Fibrosis PF). A pivotal Phase III clinical trial is completed for Pirfenidone and data released. The positive data could pave the way for the first FDA-approved therapy to treat PF. Breakthrough Digest. 02/04/09. (Also see Pulmonary Fibrosis)

PUVA/UVA-1 and Phototherapy

Positive Results.

Video: UVA1 Phototherapy for Scleroderma. Presented by Dr. Sewong Kang. Johns Hopkins Medicine. Daavlin. (Also see UVA1 Phototherapy)

For UVA1 treatment, first contact your scleroderma expert to see if it they will recommend it for you. If they recommend and prescribe it for you, try to find UVA1 facilities near you. Some dermatology offices (usually large offices or with major health centers) have UVA1 equipment (not UVB). It may also be possible to buy UVA1 phototherapy equipment for home use. It is manufactured by Daavlin and distributed worldwide. Home users must be monitored by a physician, who will prescribe the necessary dosage and monitor for possible side-effects, such as skin cancer.

UVA1 Phototherapy: A Concise and Practical Review. Phototherapy is an effective therapeutic option in scleroderma and should be considered among the first approaches in the management of localized scleroderma or morphea. UVA1 phototherapy has also been used for patients with limited and diffuse systemic sclerosis. SkinTherapyLetter, 06/21/12. (Also see Morphea Treatments)

PVAC

Positive Results.

PVAC: A Randomized, Blinded, Parallel Group, Placebo-Controlled, Pilot Study Evaluating The Effect Of PVAC Treatment In Patients With Diffuse Systemic Sclerosis. Use of PVAC in this pilot study of PSS appeared safe and was associated with a trend toward improved skin scores and other efficacy outcomes in the 15 ěg treatment group. However, no dose response relationship was seen. Additional evaluation of this therapeutic approach is warranted. Mark C. Genovese. 1695/520. ACR 2004.

Rituximab (Rituxan)

Rituximab in diffuse cutaneous systemic sclerosis (dc-SSc): an open-label clinical and histopathological study. Rituximab appears to be well-tolerated and may have potential efficacy for skin disease in dc-SSc. V P Smith. Annals of Rheumatic Diseases. 22 December 2008. (Also see Mainstream Medications)

Urokinase Therapy

Postive results for skin involvement in systemic scleroderma.

Urokinase Improves Sclerodermic Skin Pathology in Patients with Systemic Sclerosis. Gradual clinical improvement was observed with urokinase therapy, as evidenced by a noticeable restoration of skin elasticity and softness. Moreover, patients reported less intense symptoms of Raynaud's phenomenon and improvements in articular range, including previously limited movements. The authors conclude that "therapy with urokinase seems to be a rational treatment in this disease" as it appears to "modify the course and evolution of systemic sclerosis." Doctor's Guide. 01/21/04. Scand J Rheumatol 2003;32:5:261-7.

Clinical improvement in systemic sclerosis resulting from urokinase therapy explained by light and electron microscopy skin examination. These observations show that urokinase treatment seems to be an interesting therapeutic strategy to consider for the treatment of SSc. PubMed, Scand J Rheumatol. 2003;32(5):261-7.

Also See

Sjögren's Treatments & Clinical Trials
Scleroderma Medical News

Scleroderma Symptoms (treatments are listed for each symptom)

Go to Scleroderma Clinical Trials: Positive Results, Bosentan
 
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