NCT01842386

Rituximab for Anti-cytokine Autoantibody-Associated Diseases

Completed Phase 1 Last updated 15 April 2026

What this trial tests

Phase 1 trial testing Rituximab in Pulmonary Alveolar Proteinosis (PAP) in 7 participants. Completed in 6 May 2021.

Timeline

29 April 2014

Primary endpoint
6 May 2021

6 May 2021

Quick facts

Lead sponsor	National Institute of Allergy and Infectious Diseases (NIAID)
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	7
Start date	29 April 2014
Primary completion	6 May 2021
Estimated completion	6 May 2021
Sites	1 location across United States

Drugs / interventions tested

• Rituximab — full drug profile →

Conditions studied

• Pulmonary Alveolar Proteinosis (PAP) — all drugs for Pulmonary Alveolar Proteinosis (PAP) →
• Severe Mucocutaneous Candidiasis — all drugs for Severe Mucocutaneous Candidiasis →

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Who can join

Adults 18 to 99, any sex, with Pulmonary Alveolar Proteinosis (PAP) or Severe Mucocutaneous Candidiasis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Biologics or tofacitinib for people with rheumatoid arthritis naive to methotrexate: a systematic review and network meta-analysis.
   Singh JA, Hossain A, Mudano AS, Tanjong Ghogomu E, et al · · 2017 · cited 44× · PMID 28481462 · DOI 10.1002/14651858.cd012657
2. Biologics or tofacitinib for people with rheumatoid arthritis unsuccessfully treated with biologics: a systematic review and network meta-analysis.
   Singh JA, Hossain A, Tanjong Ghogomu E, Mudano AS, et al · · 2017 · cited 38× · PMID 28282491 · DOI 10.1002/14651858.cd012591
3. Primary and Acquired Immunodeficiencies Associated With Severe Varicella-Zoster Virus Infections.
   Ansari R, Rosen LB, Lisco A, Gilden D, et al · · 2021 · cited 36× · PMID 32856043 · DOI 10.1093/cid/ciaa1274
4. Bortezomib treatment for refractory nontuberculous mycobacterial infection in the setting of interferon gamma autoantibodies.
   Rocco JM, Rosen LB, Hong GH, Treat J, et al · · 2021 · cited 11× · PMID 34041472 · DOI 10.1016/j.jtauto.2021.100102
5. An Easy Assay to Detect Auto-Antibodies Neutralizing Cytokines in Subjects with Critical Infections
   Donadel N, Tesser A, Valencic E, Martino ED, et al · · 2023 · DOI 10.2139/ssrn.4567919