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Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology

NCT03366142 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2024-10-22

No results posted yet for this study

Summary

Background:

The disease leukocyte adhesion deficiency type 1 (LAD1) affects white blood cells. Those are immune system cells. In people with LAD1, white blood cells do not properly communicate with the rest of the body. This causes uncontrolled inflammation, particularly in the gums. People with LAD1 can get frequent infections and tend to lose their teeth before adulthood. Researchers want to see if a drug called ustekinumab helps people with LAD1.

Objective:

To study the safety and tolerability of ustekinumab in treating gum inflammation in people with LAD1.

Eligibility:

People ages 12 65 with LAD1

Design:

Participants will be screened with:

* Medical history
* Physical exam
* Oral exam
* A scan of the chest, abdomen, and pelvis for possible infection
* Blood and urine tests

The baseline visit will take 2 days. Participants will:

* Repeat most screening tests
* Have a skin exam
* Have small pieces of their gums removed (biopsy)
* Have mouth fluids collected
* Get the study drug injected under the skin of the abdomen, thigh, or elsewhere on the body. They will be watched for at least 2 hours.

Participants will be instructed on tracking how they are feeling and any side effects.

Participants will have 4 more visits over 40 weeks. They will get the study drug and repeat the baseline tests.

Participants may have up to 5 more tissue biopsies.

Participants will be called between visits to discuss how they are feeling and side effects.

Participants will have a final visit 52 weeks after the baseline. They will repeat most of the baseline tests.

Participants will answer questions about their oral ulcers.

Conditions

  • LAD1

Interventions

DRUG

Ustekinumab

0.75 mg/kg or 45 or 90 mg via subcutaneous injection for 5 doses (second dose 4 weeks after first, subsequent doses every 12 weeks thereafter).

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Beatriz E Marciano, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-07-02
Primary Completion
2024-10-16
Completion
2024-10-17
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03366142 on ClinicalTrials.gov