Last reviewed · How we verify
Study of Safety, Tolerability, and Efficacy of Ustekinumab for Symptomatic Gastrointestinal Inflammation Associated With Common Variable Immunodeficiency
Phase 1, PHASE2 trial testing Stelara (ustekinumab) in Gastrointestinal Inflammation Associated With CVID in 5 participants. Completed in 10 July 2020.
| Lead sponsor | National Institute of Allergy and Infectious Diseases (NIAID) |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 5 |
| Start date | 19 October 2015 |
| Primary completion | 16 August 2019 |
| Estimated completion | 10 July 2020 |
| Sites | 1 location across United States |
National Institute of Allergy and Infectious Diseases (NIAID)
Adults 18 to 75, any sex, with Gastrointestinal Inflammation Associated With CVID or CVID Enteropathy. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Whether treatment with ustekinumab is safe and tolerated in patients with common variable immunodeficiency (CVID) enteropathy as determined by no significant increase in infection or serious adverse events
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: Ustekinumab-Single Dose Phase 1 | 0 |
Whether treatment with ustekinumab is safe and tolerated in patients with common variable immunodeficiency (CVID) enteropathy as determined by no significant increase in infection or serious adverse events
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1: Ustekinumab-Multi-dose Phase 2 | 0 |
Whether treatment with ustekinumab is safe and tolerated in patients with common variable immunodeficiency (CVID) enteropathy as determined by no significant increase in infection or serious adverse events
| Group | Value | 95% CI |
|---|---|---|
| Cohort 2: Ustekinumab-Multi-dose Phase 2 | 0 |
Time frame: 6 months for Phase 1 and 48 Weeks for Phase 2. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Cohort 1: Ustekinumab-Sing… | Cohort 1: Ustekinumab-Mult… | Cohort 2: Ustekinumab-Mult… |
|---|---|---|---|---|
| Gastritis | Gastrointestinal disorders | — | — | — |
| Respiratory syncytial virus infection | Infections and infestations | — | — | — |
| Neuropathy peripheral | Nervous system disorders | — | — | — |
| Reaction | System | Cohort 1: Ustekinumab-Sing… | Cohort 1: Ustekinumab-Mult… | Cohort 2: Ustekinumab-Mult… |
|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | — | — | — |
| Gastroenteritis Escherichia coli | Infections and infestations | — | — | — |
| White blood cell count decreased | Investigations | — | — | — |
| Hypoproteinaemia | Metabolism and nutrition disorders | — | — | — |
| Anaemia | Blood and lymphatic system disorders | — | — | — |
| Abdominal distension | Gastrointestinal disorders | — | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — | — |
| Fatigue | General disorders | — | — | — |
| Tenderness | General disorders | — | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — | — |
| Blood alkaline phosphatase increased | Investigations | — | — | — |
| Blood glucose increased | Investigations | — | — | — |
| Blood lactate dehydrogenase | Investigations | — | — | — |
| Electrocardiogram abnormal | Investigations | — | — | — |
| Haematocrit decreased | Investigations | — | — | — |
| Neutrophil count decreased | Investigations | — | — | — |
| Norovirus test positive | Investigations | — | — | — |
| Prothrombin time prolonged | Investigations | — | — | — |
| Hypoalbuminaemia | Metabolism and nutrition disorders | — | — | — |
| Hypocalcaemia | Metabolism and nutrition disorders | — | — | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — | — | — |
| Headache | Nervous system disorders | — | — | — |
| Iron deficiency anaemia | Blood and lymphatic system disorders | — | — | — |
| Lymph node pain | Blood and lymphatic system disorders | — | — | — |
| Splenomegaly | Blood and lymphatic system disorders | — | — | — |
| Tachycardia | Cardiac disorders | — | — | — |
| Ear pain | Ear and labyrinth disorders | — | — | — |
| Vertigo | Ear and labyrinth disorders | — | — | — |
| Eye irritation | Eye disorders | — | — | — |
| Constipation | Gastrointestinal disorders | — | — | — |
| Dry mouth | Gastrointestinal disorders | — | — | — |
| Dyspepsia | Gastrointestinal disorders | — | — | — |
| Haematochezia | Gastrointestinal disorders | — | — | — |
| Haemorrhoids | Gastrointestinal disorders | — | — | — |
| Nausea | Gastrointestinal disorders | — | — | — |
| Parotid gland enlargement | Gastrointestinal disorders | — | — | — |
| Portal hypertensive gastropathy | Gastrointestinal disorders | — | — | — |
| Rectal tenesmus | Gastrointestinal disorders | — | — | — |
| Toothache | Gastrointestinal disorders | — | — | — |
| Varices oesophageal | Gastrointestinal disorders | — | — | — |
Most-reported serious reactions: Gastritis, Respiratory syncytial virus infection, Neuropathy peripheral.
Data from ClinicalTrials.gov NCT02199496 adverse events section.
Background: \- Some people with Common Variable Immunodeficiency Disease (CVID) have gastrointestinal inflammation. This can cause diarrhea, weight loss, and not being able to absorb nutrition from food. Researchers want to see if the drug ustekinumab can help these problems. This drug blocks some proteins that cause inflammation. Objective: \- To test the safety and efficacy of the drug ustekinumab for people with CVID with gastrointestinal inflammation. Eligibility: \- Adults ages 18-75 with CVID. They must have chronic diarrhea, have unintentionally lost weight in the last year, and/or need to use nutritional supplements to maintain their weight. Design: Participants will undergo the following screening studies to make sure that this study is a good fit for your medical situation, and to make sure it is safe for you to receive the study medications tests, including tests for HIV and hepatitis . This will be done as an inpatient at the NIH Clinical Center and takes about 5-6 days: * Participants will be screened with: * Medical history * Physical exam * Blood tests, including tests for HIV and hepatitis. * Stool tests, including a timed 48 hour collection for fat malabsorption and a 24 hour collection for protein malabsorption * Urine tests, including a pregnancy test for any women with the ability to have a child * Chest CT scan to look for infection * D-xylose testing, which involves drinking a sugary solution and then having a blood sample drawn to test carbohydrate (sugar) malabsorption * Hydrogen breath testing for test for small intestinal bacterial overgrowth (SIBO) this test also involves drinking a sugary solution and then collecting breath samples * Upper endoscopy (EGD) and/or colonoscopy to look at the lining of the GI tract and take biopsies for testing. This will be done under sedation by a qualified gastroenterologist. Participants who complete screening and meet all criteria will then return to the NIH Clinical Center for the following visits: * First Treatment Visit (1 clinic day): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. A pregnancy test for women of childbearing potential. A nurse will give you three shots of 90 mg ustekinumab (270 mg total dose) by very small needles injected under the skin, and then observe you for 1 hour. * Week 8 Treatment Visit (1 clinic day): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. * Week 16 Treatment Visit (1 clinic day): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. * Week 24 Treatment and Mid-point Evaluation Visit (4-6 inpatient days): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected, including repeating the d-xylose carbohydrate malabsorption testing, the 24 hour stool collection for protein malabsorption and the 48 hour stool collection for fat malabsorption. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. * Week 32 Treatment Visit: Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. * Week 40 Treatment Visit: Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. * Week 48 ...
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
Verify or expand the search:
Trials by the same sponsor.
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02199496.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing