Efficacy and Safety of Belimumab in Subjects With Primary Sjögren's Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01160666
• Date of registration: 09/07/2010
• Prospective Registration: No
• Primary sponsor: Assistance Publique - Hôpitaux de Paris
• Public title: Efficacy and Safety of Belimumab in Subjects With Primary Sjögren's Syndrome BELISS
• Scientific title: A Phase 2, Proof of Concept, 52-Week Open Study to Evaluate the Efficacy and Safety of Belimumab (HGS1006, LymphoStat-B™), a Fully Human Monoclonal Anti-BLyS (BAFF) Antibody, in Subjects With Primary Sjögren's Syndrome
• Date of first enrolment: March 2010
• Target sample size: 20
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01160666
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

France

Contacts

• Name: Xavier Mariette, PhD
• Affiliation: Rheumatology Department of BICETRE Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

- Have a diagnosis of primary SS according to the updated American European Consensus
Group Criteria. In addition, patients must be always positive for anti-SSA or
anti-SSB antibodies

- Have the presence, at screening, of Systemic involvement (polysynovitis, skin, renal,
lung, CNS involvement, peripheral neuropathy, vasculitis, autoimmune cytopenia,
defined in Annex 1) or persistent (up to 2 months) parotid, submandibular or
lachrymal gland swelling of more than 2 cm OR

Objective sicca (positive oral and/or ocular tests reported in the American European
Consensus Group Criteria) with at least one among the following biological features of
serum B lymphocyte activation :

increased IgG levels increased free light chain levels of immunoglobulins (according to
central laboratory ranges) increased serum beta2-microglobulin levels decreased C4 levels
(C4 levels inferior to central laboratory ranges) monoclonal gammapathy cryoglobulinemia
OR

- SS of more recent onset, i.e., less than 5 years of duration of symptoms, associated
with:

- oral or ocular dryness

- fatigue

- musculoskeletal pain (i.e, 3 criteria for response as reported at page (ix-x),
characterized by VAS score more than 50/100 in all the 3 fields.

Exclusion Criteria:

1. Any BLyS-targeted (BLyS-receptor fusion protein [BR3], TACI Fc, or belimumab) at any
time.

2. Any of the following within 364 days of Day 0:

- B-cell targeted therapy (eg, rituximab, other anti-CD20 agents, anti-CD22
[epratuzumab], anti-CD52 [alemtuzumab]

- A biologic investigational agent other than B cell targeted therapy (eg,
abetimus sodium, anti CD40L antibody [BG9588/ IDEC 131]).

4- Intravenous or oral cyclophosphamide within 180 days of Day 0.

5- Any of the following within 90 days of Day 0:

- Anti-TNF therapy

- Interleukin-1 receptor antagonist

- Abatacept

- Interleukin-6 receptor antagonist

- Intravenous immunoglobulin

- Prednisone > 100 mg/day

- Plasmapheresis.

9- Very severe SS disease.

10- Major organ or hematopoietic stem cell/marrow transplant.

11- Unstable or uncontrolled acute or chronic diseases not due to SS

13- History of malignant neoplasm within the last 5 years, except for adequately
treated cancers of the skin (basal or squamous cell) or carcinoma in situ of the
uterine cervix.

14- Required management of acute or chronic infections, as follows:

- Currently on any suppressive therapy for a chronic infection

- Hospitalization for treatment of infection within 60 days of Day 0.

- Use of parenteral (IV or IM) antibiotics

16- Historically or at screening positive test for HIV antibody, hepatitis C virus
antibodies, or, hepatitis B surface antigen (HbsAg) (with or without positive serum
HBV DNA), or antiHBcAg positivity (without anti-HbsAg positivity).

17- Grade 3 or greater laboratory abnormality based on the protocol toxicity scale
except for the following that are allowed:

- Stable Grade 3 prothrombin time (PT) secondary to warfarin treatment.

- Stable Grade 3/4 proteinuria (= 6 g/24 hour equivalent by spot urine protein to
creatinine ratio allowed). (mentioned earlier in Exclusion #8)

- Stable Grade 3 neutropenia or stable Grade 3 white blood cell count.



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

Health Condition(s) or Problem(s) studied

Sjögren's Syndrome

Intervention(s)

Drug: Belimumab

Primary Outcome(s)

response rate [Time Frame: week 28]

Secondary Outcome(s)

safety and tolerability of belimumab [Time Frame: 52 weeks]

Secondary ID(s)

P090208

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Human Genome Sciences Inc.