Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT01659749 |
| Other study ID # |
IRB00002447a |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
N/A
|
| First received |
|
| Last updated |
|
| Start date |
June 1995 |
| Est. completion date |
July 2037 |
Study information
| Verified date |
March 2024 |
| Source |
Emory University |
| Contact |
Rani H Singh, PhD RD |
| Phone |
4047278519 |
| Email |
rsingh[@]emory.edu |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
The purpose of this project is to study the effectiveness of teaching teens and young women
with Phenylketonuria (PKU) or Maple Syrup Urine Disease (MSUD) about their disease and
nutrition related issues in a camp environment. It will also look at pregnancy outcome
results in women with PKU who attended Metabolic Camp and compare their results to other
women with PKU who have not attended the Metabolic Camp.
Description:
Metabolic Camp is an educational and social support program with research opportunities that
was developed in 1995 by Rani H. Singh, PhD, RD for females age 11 through adult with
phenylketonuria (PKU) and maple syrup urine disease (MSUD), two inherited metabolic disorders
(IMD). Camp provides a supportive environment for adolescent girls and women to learn about
the importance of nutrition and diet self-management, with the intention of arresting the
disease process and minimizing the instances of miscarriages and severe birth defects, which
are high in this population. After 20+ years, Metabolic Camp is established as a unique,
national program allowing up to 35 campers to live and learn during a week of nutritional
support and productive activities, while simultaneously providing researchers an opportunity
to gather important data.
The aims of Metabolic Camp are 1) to implement education curriculum and support activities as
an intervention program for females with PKU and MSUD, 2) to evaluate knowledge of disease
and dietary management, dietary treatment compliance, quality of life, and reproductive
choice issues and assess change in outcomes post-Camp, 3) to gather data through the
implementation of a research protocol during baseline and post-Camp each year, and 4) to
provide clinical and research training in IMD for professionals, from dietitians to medical
students. Campers must complete an informed consent process, should they decide to
participate in research activities. All campers submit a three-day food record and blood
samples to measure plasma amino acids on the first and last day of camp, both of which are
not classified as research activities, but rather standard of care for their disorder.
Additional studies such as indirect calorimetry, bone health markers, anthropometric markers,
nutritional status, mental and emotional wellbeing, neurocognitive functioning, access to
disease specific care, metabolomic profiling, gut microbiota, and blood and urine analysis,
have been and may continue to be assessed in those who participate in research pre and
post-Camp. These are novel investigations for the study of IMD and essential to understand
the physiological effects of Camp.
In over 20 years of Metabolic Camp, the following data have been collected: health history
and demographics, anthropometrics, dietary intake, compliance with medically prescribed diet,
quality of life, physical activity, plasma amino acids, select blood and urine biomarkers,
and bone density. Data are stored in REDCap, allowing evaluation of short- and long-term
outcomes on the life course of females with PKU and MSUD. Each year, phenylalanine
(Phe)/leucine (Leu) blood concentrations of campers decrease over the one week camp period,
demonstrating better metabolic control and compliance with diet. Campers' knowledge of
disease etiology, management, and effects, including Maternal PKU Syndrome (MPKU) are
improved. Social support networks are strengthened by meeting peers with the same or similar
disorder. The study protocol includes a comprehensive assessment of blood and urine
biomarkers to examine PKU from a much broader approach compared to the traditional
measurement of a single biomarker to assess patient status. Beneficial to campers and
clinicians alike, Metabolic Camp is an invaluable experience to gain insight, knowledge, and
stimulate new ideas related to research and optimal patient care.
