Manipulating the Gut Microbiome Study

• Status: Terminated
• Trial end date: 2018-06-05
• Target enrollment: 0
• Participant gender: All

Summary

The objective is to determine if acetohydroxamic acid (AHA) can prevent hydrolysis of urea by inhibiting the bacterial urease of gut flora of both healthy control adults as well as adults with urea cycle disorders

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Nicholas Ah Mew
• Collaborators: Children's Hospital of Philadelphia; Data Management and Coordinating Center (DMCC)
• Treatments: Acetohydroxamic acid

Criteria

Inclusion Criteria:

• For Group 1 (healthy adults):

• Ages 18-60 years

• Compliant with receiving medications orally and intravenously

• Compliant with providing blood and urine samples

For Group 2 (adult UCD patients):

• Ages 18-60 years

• Compliant with receiving medications orally and intravenously

• Compliant with providing blood and urine samples

• Established diagnosis of CPSD, OTCD, ASSD or ASLD as follows:

• Diagnosis of CPS I deficiency, defined as decreased (less than 20 % of control) CPS I enzyme activity in liver or an identified pathogenic mutation

• Diagnosis of OTC deficiency, defined as the identification of a pathogenic mutation, linkage analysis in an affected family, less than 20% of control of OTC activity in the liver, or elevated urinary orotate (greater than 20 uM/mM) in a random sample or following allopurinol loading with absence of argininosuccinic acid

• Diagnosis of AS deficiency (Citrullinemia), defined as a greater than or equal to 10-fold elevation of citrulline in plasma, decreased AS enzyme activity in cultured skin fibroblasts or other appropriate tissue, or identification of a pathogenic mutation in the AS gene

• Diagnosis of AL deficiency (Argininosuccinic Aciduria, ASA), defined as the presence of argininosuccinic acid in the blood or urine, decreased AL enzyme activity in cultured skin fibroblasts or other appropriate tissue, or identification of a pathogenic mutation in the AL gene

Exclusion Criteria:

• For both Group 1 and Group 2:

• Current or prior Helicobacter pylori infection

• Chronic gastrointestinal illness (e.g., inflammatory bowel disease)

• Chronic renal failure

• Taking probiotic medications within a week of study start date

• Currently pregnant or lactating. Documentation of a negative pregnancy test within a week prior to testing is required, unless pre-menarchal or menopausal, experiencing menses that week, or other circumstances which preclude pregnancy (e.g. hysterectomy).

• Presence of acute infection at the time of inclusion

• Participation in any other clinical interventional trial or received experimental medication within the last 30 days

• Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may put the subject at an additional risk by participating in this study