At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism

ConditionsUrea Cycle Disorders Organic Acidemias Fatty Acid Oxidation Disorder Ammonia; Metabolic Disorder

Trial statusRecruiting

Trial phaseNot applicable

Trial typeInterventional

Biological sexAll

Age12+

SponsorSequitur Health Corp.

About this trial

The goal of this observational study is to learn if people with certain ammonia metabolism disorders will measure their ammonia levels at home.

The main question it aims to answer is:

• Will participants measure their ammonia every day?

Participants will be asked to:

* Attend two in-person study visits at the clinic. * Measure temperature, heart rate, and blood oxygen every day. * Complete a short survey every day. * Measure ammonia every day.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Confirmed ammonia disorder such as

Ornithine transcarbamylase deficiency

Systemic primary carnitine deficiency

Type I citrullinemia

Disqualifiers

Incarcerated

Presence of a non-inborn error of metabolism medical condition associated with abnormal ammonia metabolism (e.g., end stage liver disease).

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Trial design

Design model

Single group

Treatments tested in this trial

Ammonia Study Device
Diagnostic test
Ammonia study device for capillary ammonia measurement

Treatment groups

30 Participants

are divided into 1 treatment group

Group A: Ammonia DeviceExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

Percentage of daily ammonia measurements completed.

Daily ammonia measurements will be completed on the ammonia study device. A single participant will have a single device. The device will store information about the time, date, and results from the measurements completed. The primary hypothesis is that participants will complete 90% of the daily ammonia measurements during their individual study periods.

Time frame

through individual participant study completion - approximately 240 days per participant with an optional 120 day additional extension period available.

Secondary outcomes

Descriptive Statistics and Correlations

After the study is complete, descriptive statistics (mean, median, range) will be calculated for individual participants for the study for the quantitative home measurements (temperature, heart rate, pulse oxygenation, and capillary ammonia). Descriptive statistics (mean, median, range) will be calculated for all aggregated participant data for the study for the quantitative home measurements (temperature, heart rate, pulse oxygenation, and capillary ammonia). The strength of correlations between quantitative values and participant self reported survey information and/or medical record documented clinical events will be investigated.

Time frame

From enrollment through study completion, this is an average of 240 days per participant. If a participant chooses to participate in the optional study extension then the time frame will be approximately 360 days per participant.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sequitur Health Corp.

Lead sponsor

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Oregon Health and Science University