Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 30 May 2022
• Main ID: NCT01670565
• Date of registration: 15/08/2012
• Prospective Registration: No
• Primary sponsor: Hospital for Special Surgery, New York
• Public title: Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis
• Scientific title: Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis: A Phase 2a, Single-centered, Randomized, Placebo-controlled, Double-blind, Proof-of-concept Pilot Study.
• Date of first enrolment: August 2012
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01670565
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Robert Spiera, MD
• Affiliation: Hospital for Special Surgery, New York

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age greater than or equal to eighteen years.

2. Clinical diagnosis of diffuse systemic sclerosis by ACR criteria, with a stable
modified Rodnan skin score in the one month preceding introduction of belimumab
therapy.

3. Disease duration of less than or equal to 3 years as defined by the date of onset of
the first non-Raynaud's symptom.

Exclusion Criteria:

1. Inability to render informed consent in accordance with institutional guidelines.

2. Disease duration of greater than 3 years.

3. Patients with mixed connective tissue disease or "overlap" (i.e. those who satisfy
more than one set of ACR criteria for a rheumatic disease.)

4. Limited scleroderma.

5. Systemic sclerosis-like illness associated with environmental or ingested agents such
as toxic rapeseed oil, vinyl chloride, or bleomycin.

6. Ongoing treatment with immunosuppressive therapies including cyclophosphamide,
azathioprine, methotrexate, or cyclosporine, or use of those medications within 1
month of trial entry.

7. The use of other anti-fibrotic agents including colchicine, D-penicillamine,
minocycline, tyrosine kinase inhibitors (nilotinib, imatinib, dasatinib), or Type 1
oral Collagen in the month prior to enrollment.

8. Use in the prior month of corticosteroids at doses exceeding the equivalent of
prednisone 10 mg daily. Use of corticosteroid at < 10 mg of prednisone can continue
during the course of the study.

9. Treatment with MMF at a dose of = 2 grams daily for > 3 months.

10. Concurrent serious medical condition which in the opinion of the investigator makes
the patient inappropriate for this study such as uncontrollable CHF, arrhythmia,
severe pulmonary or systemic hypertension, severe GI involvement, hepatic impairment,
serum creatinine of greater than 2.0, active infection, severe diabetes, unstable
atherosclerotic cardiovascular disease, malignancy, HIV, or severe peripheral vascular
disease.

11. A positive pregnancy test at entry into this study.

12. Men and women with reproductive potential will be required to use effective means of
contraception through the course of the study, such as a tubal ligation or
hysterectomy, condom or diaphragm used with a spermicide,or an intrauterine device
(IUD). Approved hormonal contraceptives (such as birth control pills, patches,
implants or injections) may interact with and reduce the effectiveness of MMF and
thus, are not acceptable. Contraceptive measures such as Plan B (TM), sold for
emergency use after unprotected sex, are not acceptable methods for routine use.

13. Breastfeeding. Breastfeeding is contraindicated with the use of MMF.

14. Participation in another clinical research study involving the evaluation of another
investigational drug within ninety days of entry into this study.

15. The presence of severe lung disease as defined by a diffusion capacity of less than
30% of predicted or requiring supplemental oxygen.

16. History of HIV infection

17. Known active bacterial, viral, fungal, mycobacterial, or other infection r any major
episode of infection requiring hospitalization or treatment with IV antibiotics within
4 weeks of screening, or oral antibiotics within 2 weeks prior to screening

18. Any other disease, metabolic dysfunction, physical examination finding, or clinical
laboratory finding giving reasonable suspicion of a disease or condition that
contraindicates the use of an investigational drug or that may affect the
interpretation of the results or render the patient at high risk from treatment
complications

19. Prior use of Belimumab, Rituximab, or other B-Cell depleting therapies ever

20. The use of other biologics including TNF inhibitors, abatacept, or tocilizumab within
1 month of enrollment [this is a safety issue]

21. Patients with a history of severe depression, psychosis, or suicidal ideation will be
excluded.



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

Health Condition(s) or Problem(s) studied

Systemic Sclerosis

Intervention(s)

Other: Placebo Infusion

Drug: Belimumab

Drug: Mycophenolate Mofetil

Primary Outcome(s)

Change in Forced Vital Capacity (FVC) [Time Frame: Baseline and Week 52]

Number of Adverse Events and Serious Adverse Events [Time Frame: At 52 weeks]

Change in Modified Rodnan Skin Score (MRSS) [Time Frame: Baseline and at 52 weeks]

Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO) [Time Frame: Baseline and Week 52]

Secondary Outcome(s)

Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ DI) [Time Frame: Baseline and Week 52]

Change in Short Form-36 (SF-36) Questionnaire: Physical Component Summary [Time Frame: Baseline and Week 52]

Change in in Short Form-36 (SF-36) Questionnaire:Mental Component Summary [Time Frame: Baseline and at 52 weeks]

Secondary ID(s)

2014-279

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Human Genome Sciences Inc.

Results

• Results available: Yes
• Date Posted: 24/05/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01670565