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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT02199496
Date of registration: 23/07/2014
Prospective Registration: Yes
Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Public title: Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency
Scientific title: An Open-Label Phase I/II Pilot Study to Assess the Safety/Tolerability and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency
Date of first enrolment: October 19, 2015
Target sample size: 5
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT02199496
Study type:  Interventional
Study design:  Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1/Phase 2
Countries of recruitment
United States
Contacts
Name:     Ivan J Fuss, M.D.
Address: 
Telephone:
Email:
Affiliation:  National Institute of Allergy and Infectious Diseases (NIAID)
Key inclusion & exclusion criteria

- DIAGNOSIS AND CRITERIA FOR INCLUSION:

A subject is eligible for the study if all of the following criteria are met:

- Has given written informed consent.

- Is male or female aged 18 through 75 years.

- Has CVID or selective IgG subclass deficiency of either one or concomitant IgG
subclasses comprising IgG1, IgG2, IgG3 or IgG4 (functional hypogammaglobulinemia)
diagnosed prior to screening as based on the International Union of Immunological
Societies (IUIS) criteria.

- Has a documented, unintended loss of >5% of their body weight over the last year or
requires nutritional supplements to maintain his/her body weight and/or has chronic
diarrhea defined as a complaint of at >/= 50% of stools are non-formed for at least 4
consecutive weeks per patient history. Alternately, must be dependent on a therapeutic
dose of antidiarrheals (e.g., loperamide or diphenoxylate with atropine) for control
of chronic diarrhea.

- If taking oral antibiotics chronically, must have used a stable dose of the antibiotic
continuously for at least 2 weeks prior to start of screening period.

- Is willing to have samples stored.

- Be willing to consistently take appropriate measures to avoid pregnancy through the
Week 48 study point. All subjects will be informed of the potential risks of
ustekinumab during pregnancy and counseled on pregnancy avoidance appropriate to the
subject s circumstances (e.g. fertility status, medical contraindications to hormonal
birth control, and/or personal or religious beliefs regarding pregnancy avoidance).
Subject to the judgment and discretion of the PI, some subjects may not need to take
pregnancy

avoidance measures. Patient handout on pregnancy avoidance will be provided to patients at
the time of consent and discussion regarding pregnancy avoidance during the study.

-Subjects who have previously been treated with a single 270 mg dose of ustekinumab on this
study must be greater than 6 months from their treatment dose and have had recurrence of
enteropathy symptoms.

CRITERIA FOR EXCLUSION:

A subject is excluded from the study if any of the following criteria are met:

GENERAL CRITERIA:

- Has any clinically significant disease or condition (e.g., renal, hepatic,
neurological, cardiovascular, pulmonary, endocrinologic, psychiatric, hematologic,
urologic, or other acute or chronic illness) that in the opinion of the investigator
would make the subject an unsuitable candidate for this trial, or put the subject at
undue risk by participating in this study.

- Is a woman who has a positive pregnancy test or who is breast-feeding

- Is a woman who does not agree to abide by the contraceptive measures required to
prevent pregnancy during participation in the study, or meets exemption criteria for
contraceptive measures, as outlined in the protocol.

- Has any of the following clinical chemistry values:

- AST >2.5 times upper limit of normal (ULN).

- ALT >2.5 times ULN.

- Serum bilirubin >1.5 times ULN.

- Serum creatinine >1.5 times ULN.

- Alkaline phosphatase >2.5 times ULN.

- Has a hemoglobin level <9 g/dL or hematocrit <30%.

- Has an International Normalized Ratio (INR) >1.3 or a Partial Thromboplastin Time
(PTT) >3 sec of ULN.

- Has the following cell counts (cells/microL):

- Platelet count <75,000 or >800,000.

- White blood cell count <2,000.

- Neutrophil count <1,000.

- Has a current infection requiring intravenous antibiotics, a serious local infection
(e.g., cellulitis, abscess) or systemic infection (e.g., pneumonia, septicemia).

- Has a history of cancer within the past 5 years, with the exception of excised basal
cell carcinoma, squamous cell carcinoma of the skin, or cervical carcinoma in situ.

- Had a dependency for any illicit drug, chemical, or alcohol within the past 5 years.

- Has a history of active tuberculosis (TB) (or a chest x-ray (CXR) with findings
suggestive of old TB infection including calcified nodular lesions, apical fibrosis,
or pleural scarring), acute or chronic hepatitis B, hepatitis C, human
immunodeficiency virus (HIV) or opportunistic infections.

GASTROINTESTINAL CRITERIA

-Has a stool sample determined positive for acute gastrointestinal infection with impact of
occurrence on gastrointestinal inflammation as determined by principal investigator during
screening. In addition, stool samples positive for GI pathogens will be discussed with an
infectious disease physician to determine impact of occurrence on gastrointestinal
inflammation. If organism thought to be pathogenic, the subject will be treated with
appropriate therapy. This will be documented in the subject s medical record.

PRIOR MEDICATION CRITERIA

- Received daily corticosteroids within 1 month prior to receiving study agent. The use
of short-term or single-dose corticosteroids as a pretreatment regimen for IVIG is
acceptable.

- Received any investigational drug within 3 months prior to receiving study agent.

- Received certolizumab or natalizumab within 3 months prior to receiving study agent

- Received vedolizumab, infliximab, etanercept, or adalimumab within 2 months prior to
receiving study agent.

- Received cyclosporine, tacrolimus, sirolimus, pimecrolimus, mycophenolate mofetil or
any other systemic immunosuppressants within 1 month prior to receiving study agent.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All
Health Condition(s) or Problem(s) studied
CVID Enteropathy
Gastrointestinal Inflammation Associated With CVID
Intervention(s)
Biological: Stelara (ustekinumab)
Primary Outcome(s)
Number of Participants With Increase in Infection or Serious Adverse Events (SAEs) Related to Multi-dose Administration of Ustekinumab (Cohort 2) [Time Frame: 48 weeks]
Number of Participants With Increase in Infection or Serious Adverse Events (SAEs) Related to Single Dose Administration of Ustekinumab [Time Frame: 6 months]
Number of Participants With Increase in Infection or Serious Adverse Events (SAEs) Related to Multi-dose Administration of Ustekinumab (Cohort 1) [Time Frame: 48 weeks]
Secondary Outcome(s)
Secondary ID(s)
14-I-0153
140153
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 24/08/2020
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT02199496
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