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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 August 2023
• Main ID: NCT03366142
• Date of registration: 07/12/2017
• Prospective Registration: Yes
• Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Public title: Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology
• Scientific title: Use of Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology
• Date of first enrolment: July 2, 2018
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03366142
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Dawn Shaw, R.N.

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

• Name: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

- Age 12-65 years.

- Molecularly and cellularly confirmed LAD1 with inflammatory lesions.

- Willing to allow storage of biological samples for future research.

- Willing to allow genetic testing of blood samples.

- Able to provide informed consent.

- Participants who can get pregnant or impregnate a partner must agree to use adequate
contraception when engaging in sexual activities that can result in pregnancy.
Adequate contraception must be used consistently starting at screening and lasting
through the final study visit. Appropriate forms of contraception include the
following:

1. Intrauterine device or equivalent.

2. Hormonal contraceptive (eg, consistent, timely, and continuous use of
contraceptive pill, patch, ring, implant, or injection) that has reached full
efficacy before dosing.

3. A double barrier method (eg, male/female condom, cap, or diaphragm plus
spermicide).

4. Be in a stable, long-term monogamous relationship, per assessment of the
principal investigator (PI), with a partner who does not pose any potential
pregnancy risk, eg, has undergone a vasectomy at least 6 months before the first
dose of study agent or is of the same sex as the participant.

5. Have had a hysterectomy and/or a bilateral tubal ligation or both ovaries
removed.

EXCLUSION CRITERIA:

- History of malignancy (except for basal cell carcinoma) within the previous 5 years.

- Infected with HIV.

- Active uncontrolled bacterial, viral, or fungal infection.

- Active or chronic viral hepatitis.

- Active or latent untreated tuberculosis.

- Received Bacillus Calmette-Guerin vaccine within the last year.

- Received live attenuated vaccines within 15 weeks before the first dose.

- Allergy to any component of the ustekinumab formulation.

- Pregnant or breastfeeding.

- Any condition that, in the opinion of the investigator, contraindicates participation
in this study.



Age minimum: 12 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

LAD1

Intervention(s)

Drug: Ustekinumab

Primary Outcome(s)

Evaluate the safety and tolerability of ustekinumab in treating LAD1 affected patients who have inflammation, such as gingival, skin or gut. [Time Frame: 13 months]

Secondary ID(s)

18-I-0022

180022

Source(s) of Monetary Support

Please refer to primary and secondary sponsors