A Pilot Study of XOMA 052 in Familial Cold Autoinflammatory Syndrome / Muckle-Wells Syndrome and Behcet's Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01211977
• Date of registration: 29/09/2010
• Prospective Registration: No
• Primary sponsor: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Public title: A Pilot Study of XOMA 052 in Familial Cold Autoinflammatory Syndrome / Muckle-Wells Syndrome and Behcet's Disease
• Scientific title: A Pilot Study of XOMA 052 in Familial Cold Autoinflammatory Syndrome (FCAS) / Muckle-Wells Syndrome (MWS) and Behcet's Disease (BD)
• Date of first enrolment: August 27, 2010
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT01211977
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: Double.
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

FOR FCAS / MWS:

1. Male or female subjects with inflammatory disease greater than or equal to 18 years of
age.

2. Participation in NIH study #03-AR-0173 ( Studies of the Natural History, Pathogenesis,
and Outcome of Autoinflammatory Diseases )

3. CIAS1 mutation positivity or FCAS / MWS based on clinical grounds with a history of a
complete response to IL-1 blocking medications.

4. Subjects presenting with active FCAS / MWS based on clinical signs/symptoms in
addition to elevated acute phase reactants (CRP, SAA or ESR). To meet established
clinical criteria for active disease, patients must have recurrent intermittent
episodes of fever and rash, an age of onset < 6 months of age, duration of most
attacks < 24 hours, and the presence of conjunctivitis with attacks. Active disease
will be defined as either the presence of classical features or a history of such
features that became quiescent in the setting of therapy with anakinra or rilonacept.
However, before a patient who has quiescent disease and is currently taking anakinra
or rilonacept can receive study drug, he/she must fulfill criteria for active disease
after anakinra or rilonacept has been discontinued. Subjects must be greater than 48
hours from their last dose of anakinra (half-life 4-6 hours) and 14 days from their
last dose of rilonacept (half-life 7.5 days) before beginning XOMA 052 therapy, and
will not take anakinra or rilonacept for the remainder of their enrollment in the
study.

5. Stable dose of steroids, NSAIDs, DMARDs, or colchicine for four weeks prior to
enrollment visit.

6. Females of childbearing potential (young women who have had at least one menstrual
period regardless of age) must have a negative urine pregnancy test at screening and a
negative serum pregnancy test at baseline prior to performance of any radiologic
procedure or administration of study medication.

7. Women of childbearing age and men able to father a child, who are sexually active, who
agree to use a form of effective birth control, including abstinence.

8. Either (1) a negative PPD test using 5 T.U. intradermal testing per CDC guidelines and
no evidence of active TB by history on chest X-ray at the time of enrollment or (2) a
positive PPD with no evidence of active TB on chest X-ray at the time of enrollment
and either past or present treatment with adequate therapy for at least one month
prior to first dose of study medication. Full prophylaxis regimens will be completed.
Subjects who have been BCG-vaccinated will also be skin-tested.

9. Able to understand, and complete study-related questionnaires.

10. Able and willing to give informed consent and abide with the study procedures.

FOR BD:

1. Male or female subjects with BD associated inflammatory disease greater than or equal
to 18 years of age.

2. Participation in NIH study #03-AR-0173 ( Studies of the Natural History, Pathogenesis,
and Outcome of Autoinflammatory Diseases )

3. Diagnosis of Behcet's disease as determined by the International Study Group Criteria
or by complete Japanese Criteria

4. Active mucocutaneous disease as defined by at least one oral or genital ulcer within
the past month.

5. Stable dose of steroids, NSAIDs, DMARDs, or colchicine for four weeks prior to
enrollment visit.

6. For patients with ocular disease, no active intermediate or posterior disease at
enrolment but history of an ocular flare (greater than or equal to 3 in the last 6
months) in the presence of any systemic anti-inflammatory therapy such as prednisone,
azathioprine, Mycophenolate, methotrexate, cyclosporine, a TNF inhibitor, or a
combination of these medications. Patients must have developed active disease in the
presence of at treatment with at least one of the following medications for at least
six months: azathioprine, cyclosporine, or a TNF inhibitor.

7. Females of childbearing potential (young women who have had at least one menstrual
period regardless of age) must have a negative urine pregnancy test at screening and a
negative serum pregnancy test at baseline prior to performance of any radiologic
procedure or administration of study medication. Female patients will be screened for
pregnancy at all NIH visits.

8. Women of childbearing age and men able to father a child, who are sexually active, who
agree to use a form of effective birth control, including abstinence.

9. Either (1) a negative PPD test using 5 T.U. intradermal testing per CDC guidelines and
no evidence of active TB on chest X-ray at the time of enrollment or (2) a positive
PPD with no evidence of active TB by history or on chest X-ray at the time of
enrollment and either past or present treatment with adequate therapy for at least one
month prior to first dose of study medication. Full prophylaxis regimens will be
completed. Subjects who have been BCG-vaccinated will also be skin-tested.

10. Able to understand, and complete study-related questionnaires.

11. Able and willing to give informed consent and abide with the study procedures.

EXCLUSION CRITERIA:

For both study populations:

1. Treatment with a live virus vaccine during 3 months prior to baseline visit. No live
vaccines will be allowed throughout the course of this study.

2. Patients with ocular disease who received local treatments other than eye drops (i.e.
periocular or intraocular steroids, implants or other antinflammatory agents within 4
weeks prior to enrollment)

3. Current treatment with TNF inhibitors or discontinuation of TNF inhibitors within 8
weeks.

4. Presence of active infections or a history of pulmonary TB infection with or without
documented adequate therapy.

5. Chest x-ray read by a radiologist with pleural scarring and/or calcified granuloma
consistent with prior TB.

6. Positive test for or prior history of HIV, Hepatitis B or C.

7. History or concomitant diagnosis of congestive heart failure.

8. History of malignancy. Subjects deemed cured of superficial malignancies such as
cutaneous basal or squamous cell carcinomas, or in situ cervical cancer may be
enrolled.

9. Known hypersensitivity to CHO cell derived biologicals or any components of XOMA 052.

10. Presence of any additional rheumatic disease or significant systemic disease. For
example, major chronic infectious/ inflammatory/ immunologic disease (such as
inflammatory bowel disease, psoriatic arthritis, spondyloarthropathy, SLE in a


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

Health Condition(s) or Problem(s) studied

Muckle Wells Syndrome

Autoinflammatory

Behcet's Disease

Intervention(s)

Drug: XOMA 052

Primary Outcome(s)

Safety

Changes in clinical and biochemical indicators of inflammation

Secondary Outcome(s)

Long term clinical and biochemical responses

Number of flares during randomized withdrawal

If applicable, assessment of the lack of complete response in relation to pharmacologic parameters.

Duration of response to single dose

Secondary ID(s)

100182

10-AR-0182

Source(s) of Monetary Support

Please refer to primary and secondary sponsors