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Experimental drug shows promise for rare immune disorders

NCT ID NCT01842386

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 17, 2026 · Updated 12 times

Summary

This early-phase study tested the safety of rituximab, a drug that targets faulty white blood cells, in 7 adults with rare immune diseases like pulmonary alveolar proteinosis and severe candidiasis. Participants received several doses over 6 months and were monitored for 18 months afterward. The goal was to see if rituximab could help control these hard-to-treat conditions.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

7 people

The number who actually took part.

Started

Apr 2014

Finished

May 2021

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 99 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* INCLUSION CRITERIA: Subjects (greater than or equal to 18 years of age) are eligible if they meet the following criteria: 1. Currently enrolled in one of the following protocols: 95-I-0066, 07-I-0033, 01-I-0202, or 93-I-0119. 2. Presence of anticytokine autoantibodies in serum or plasma, along with the anticipated clinical consequences of the identified anticytokine autoantibody including, but not limited to: * Anti-IFN- \>= autoantibodies and disseminated NTM. * Anti-IL-17 autoantibodies and CMC. * Anti-GM-CSF autoantibodies and PAP or cryptococcosis. 3. Progression of anticytokine autoantibody-associated diseases despite conventional therapy, including, but not limited to: * Antimycobacterials for disseminated NTM. * Antifungals for mucocutaneous candidiasis or cryptococcosis. * Subcutaneous or inhaled GM-CSF and/or whole lung lavage for PAP. 4. For ongoing autoantibody-associated infection, stable, optimized antibiotic regimen for at least 1 month prior to initiation of rituximab and ability to continue these antibiotics throughout treatment with rituximab. 5. Willingness to comply with study medication, visits, and procedures, as deemed necessary by the study investigator. 6. Willingness to have samples stored for future research and genetic testing. 7. Willingness to be hospitalized for the inpatient visits (initial doese on day 1 and day 15 will occur in the inpatient unit. 8. Negative serum pregnancy test result for women of childbearing potential. * Women of childbearing potential and men are eligible if they agree to postpone conception for 18 months following rituximab therapy. They must agree to use 2 adequate methods of contraception, such as: * Hormonal contraception. * Male or female condoms with or without a spermicide, diaphragm or cervical cap with a spermicide, or intrauterine device. * Sterilization of either partner. EXCLUSION CRITERIA: Subjects who meet the following criteria are not eligible to enter the study: 1. HIV seropositivity. 2. Active underlying malignancy, except thymoma and basal and squamous cell carcinoma. 3. Immunomodulatory or immunosuppressive therapy, including: * Corticosteroids at a dose equivalent to greater than or equal to 15 mg of prednisone/day at any time during the month immediately prior to enrollment. * History of using biologic agents or any other systemic immune-suppressive or immunomodulatory agents within the past year. 4. Use of another investigational study agent within 8 weeks of enrollment. 5. Known anaphylaxis or IgE-mediated hypersensitivity to murine proteins or any component of the study medication. 6. History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies. 7. Evidence of significant uncontrolled concomitant diseases, such as cardiovascular disease, or nervous system, pulmonary, renal, hepatic, endocrine, or gastrointestinal disorders. 8. Diagnosis of an unrelated underlying immunodeficiency. 9. Hepatitis B (subjects with hepatitis C are eligible to enter the study). 10. Live vaccines within 1 month prior to receiving the study drug. 11. Unsuitable participation as judged by the principal investigator. 12. History of cancer, including solid tumors and hematologic malignancies (except basal cell or squamous cell carcinoma of the skin that have been excised and cured and thymoma). 13. History of alcohol, drug, or chemical abuse within 6 months prior to screening. 14. Poor peripheral venous access. 15. Intolerance or contraindications to oral or IV corticosteroids. 16. Screening laboratory values: * Serum creatinine \>1.4 mg/dL for women and \>1.6 mg/dL for men. * Platelet count \<100,000/ L. * Absolute neutrophil count \<1500 cells/ L. * IgG \<5.65 times 10(-2) mg/dL or IgM \<0.55 times 10(-2) mg/dL. 17. Breastfeeding.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

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