Rituximab for Anti-cytokine Autoantibody-Associated Diseases

Conditions

• Pulmonary Alveolar Proteinosis (PAP)
• Severe Mucocutaneous Candidiasis

Eligibility Criteria

Sex

ALL

Age

18 Years - 99 Years

Healthy Volunteers

Not accepted

Interventions

• Rituximab — DRUG
  Subjects will receive intravenous (IV) infusions of rituximab 1 gram on days 1 and 15, and subsequently if indicated up to once a month for 5 months (+/-5 days for each visit) starting approximately on day 42. Subjects whose infections respond positively to the treatment but then relapse may be offered additional treatment.

Study Details

Background: * Healthy people have white blood cells that protect them against bacteria, viruses, and fungi. However, some people have diseases which cause the body to make white blood cells that do not work properly. These white blood cells can attack the body s own proteins. These types of diseases are called anti-cytokine autoantibody-associated diseases. They can cause severe illnesses and even death. They are also difficult to treat with standard drugs. * Rituximab is a drug used to treat rheumatoid arthritis. It attacks white blood cells that do not work properly. Currently, it is not approved for treating anti-cytokine autoantibody-associated diseases. However, researchers think that it may be able to help treat people with these immune diseases. Objectives: \- To see if rituximab is a safe and effective treatment for anti-cytokine autoantibody-associated diseases. Eligibility: * Individuals at least 18 years of age who have anti-cytokine autoantibody-associated diseases. * Participants must also be enrolled in a related immune disorder study at the National Institutes of Health. Design: * The study will last 24 months. Participants will take rituximab for 6 months and have follow-up visits for the remaining 18 months. * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Other samples will be collected as needed if participants currently have an infection. * Participants will enter the hospital for 1 week at the start of treatment. They will have four doses of rituximab given 2 days apart. This first treatment will be monitored with frequent blood tests. * Over the next 6 months, participants will have four more doses of rituximab given about 1 month apart. Treatment will be monitored with frequent blood tests and sample collections as needed. * There will be four follow-up study visits at 3, 6, 12, and 18 months after the last dose of rituximab.

Key Dates

Start date

Apr 29, 2014

Status verified

Apr 2026

Primary completion

May 6, 2021

Completion

May 6, 2021

Study Design

Enrollment

7 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Rituximab
  Adults (=18 years of age) with anticytokine autoantibodyassociated diseases who are refractory to conventional treatment and who test negative for the human immunodeficiency virus (HIV)

Primary Outcome Measure

safe and tolerable administration of rituximab in subjects with anticytokine autoantibody-associated diseases who are refractory to conventional treatment [ Time Frame: at study end ]

Locations (1)

Find similar trials in Bethesda, MD