View clinical trials related to Hyperargininemia.
Filter by:This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D. The study consists of a screening period of up to 4 weeks, a subsequent 12-month treatment period, and a Safety Follow-Up Visit 2 weeks after the last treatment.
Objectives: - To study nutrition and immune system problems in people with urea cycle disorders. - To study how people with urea cycle disorders and healthy volunteers respond to standard flu and/or hepatitis A vaccines. - To compare differences in nutrition and immune systems of people with urea cycle disorders with that of healthy volunteers. Eligibility: - Healthy males and females at least 2 years of age who are able to travel to the National Institutes of Health hospital in Bethesda, MD - Males and females at least 2 years of age who have a urea cycle disorder and are able to travel to the National Institutes of Health hospital in Bethesda, MD. Design: For Patients with urea cycle disorder: - Participants will spend 2 to 3 days in the National Institutes of Health hospital for the following tests: - A physical exam and review of medical history - Food log for 3 days before the start of the study - Blood tests - 24-hour urine collection - Resting metabolism test - DEXA scan imaging study of bones and body fat - Participants who are old enough to do certain tasks by themselves (like dressing and eating) can choose to have the following extra tests: - 24-hour metabolic room measurements - BodPod(Registered Trademark) study to measure bones and body fat - Participants may choose to have a flu shot and/ or Hepatitis A shot at the end of the study and will be monitored to check for possible side effects. - Participants will return within 1 to 3 months for follow-up tests/immunizations. For Healthy Volunteers: - Participants will be seen at the outpatient clinics at the National Institutes of Health hospital for up to 2 visits for the following: - Review food log completed 3 days before the start of the study - Blood tests - Participants may choose to have a flu shot and/ or Hepatitis A shot at the end of the study and will be monitored to check for possible side effects. - Participants will return within 1 to 3 months for follow-up tests/immunizations. - Review of second food log completed 3 days before second outpatient visit