Glutamine Supplementation in People With Immune Dysregulation
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|ClinicalTrials.gov Identifier: NCT04019769|
Recruitment Status : Suspended (Status updated due to upcoming study staffing change)
First Posted : July 15, 2019
Last Update Posted : September 11, 2019
Glutamine is an amino acid. People get amino acids from food or from the body s cells. The body needs amino acids to stay healthy. Glutamine might help treat some people with immune system problems like atopic dermatitis.
To study the safety and effectiveness of glutamine supplements for people with certain immune system problems.
People ages 5-65 with atopic dermatitis and other immune system problems
Participants will be screened in another protocol.
Participants will have 8 visits.
Visit 1 includes:
Blood and urine tests
For participants with AD, photographs of the skin
Participants will get a diary to record their symptoms every day during the study. They will record any glutamine side effects and bring the diary to every visit.
Visit 2 is about 1 month after visit 1. Participants will repeat visit 1 tests and get glutamine to take home. It is a powder that can be added to drinks or food. They will take it twice a day for 3 months. They will record their doses in a diary each day and bring the diary to all visits.
Participants will have a phone call 5 days after starting glutamine to discuss how they are feeling.
Visit 3 is about 7 days after participants start taking glutamine. They will have blood tests.
Visits 4, 5, and 6 occur each month participants are taking glutamine. Participants will repeat visit 1 tests.
Participants will stop taking glutamine after visit 6.
Visits 7 and 8 occur 1 and 3 months after participants stop taking glutamine. Participants will repeat visit 1 tests.
|Condition or disease||Intervention/treatment||Phase|
|Eczema MTOROC1 Signaling||Drug: L-glutamine||Early Phase 1|
Atopic dermatitis (AD) is a chronic, relapsing, inflammatory skin condition that typically begins in infancy or early childhood and can be highly debilitating with a marked reduction in quality of life. Current treatment modalities for AD include frequent use of skin moisturizers such as creams or emollients, topical corticosteroids and topical calcineurin inhibitors, systemic immunosuppressive drugs, and, in select cases of refractory disease, wet wrap therapy. The available systemic treatments for AD when topical treatment fails can have substantial adverse effects and are not always effective. The search for targeted therapies based on pathway disruptions in patients could present opportunities to provide therapies on a more personalized basis, tailored to the pathogenic pathways.
Based on prior studies, we have demonstrated that supplemental glutamine has potential as an effective AD treatment in patients with caspase activation and recruitment domain (CARD) gene mutations. Prior studies have demonstrated that loss of CARD signaling leads to a decrease in the mammalian target of rapamycin complex 1 (mTORC1) signaling pathway. Glutamine supplementation can normalize this defect in mTORC1. The CARD11 BCL10 MALT1 (CBM) signalosome complex is a critical component of mTORC1 activation. Some patients with gene mutations affecting the CBM complex do not develop AD but do develop other symptoms of immunoregulation and may also benefit from glutamine supplementation. We will conduct a phase 1, open-label study to investigate the use of supplemental glutamine in patients with immune dysregulation. Subjects 5 to 65 years of age will take oral glutamine daily for 3 months. To assess the role of the mTORC1 pathway in response to glutamine supplementation, we will enroll 3 groups of patients: 1) those with AD and a defect in mTORC1 signaling or a mutation in the CBM complex; 2) those with AD who do not have a defect in mTORC1 signaling or a mutation in the CBM complex; and 3) those who have a defect in mTORC1 signaling or a mutation in the CBM complex and do not have AD. Subjects will complete a daily symptom diary for 1 month before starting glutamine to document baseline disease status. Disease severity will be assessed at baseline, at the end of glutamine supplementation, and 1 and 3 months after completing the glutamine regimen. Blood will be collected periodically for safety, tolerability, and research assessments.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||35 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 1 Trial of Glutamine Supplementation in Patients With Immune Dysregulation|
|Estimated Study Start Date :||September 16, 2019|
|Estimated Primary Completion Date :||January 1, 2020|
|Estimated Study Completion Date :||January 1, 2020|
All patients will receive L-glutamine 10-30mg daily based on weight for 3 months
product will be obtained by Endari and administered by NIH pharmacy. Patient will take supplement as prescribed
- To characterize the safety and tolerability of glutamine supplementation in patients with immune disregulation. [ Time Frame: 1 year ]assess safety and tolerability of glutamine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04019769
|United States, Maryland|
|National Institutes of Health Clinical Center|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Joshua D Milner, M.D.||National Institute of Allergy and Infectious Diseases (NIAID)|