Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasia
Status:
Completed
Trial end date:
2016-12-02
Target enrollment:
Participant gender:
Summary
Background:
- Congenital adrenal hyperplasia (CAH) is a genetic disorder of the adrenal gland. The
adrenal gland is located in the abdomen and produces small amounts of hormones such as
cortisol, aldosterone, and androgen. These hormones help control blood pressure, protect
the body, and maintain good health, especially during development. People with CAH do
not make enough cortisol and aldosterone, and make too much androgen. This can lead to
serious medical problems. The standard treatment is to take pills that mimic the effects
of cortisol and aldosterone. However, treatment with pills can have long-term side
effects because of the higher doses needed, and may not work well for some people.
- A possible new treatment for CAH is to use a pump to deliver cortisol under the skin.
Similar pumps are often used to give insulin to people with diabetes. Researchers think
that a cortisol pump might be able to help the body use the cortisol more effectively
than taking pills. They want to compare the results of a cortisol pump and standard pill
treatments for CAH.
Objectives:
- To compare the effectiveness of a cortisol pump with standard cortisol pill therapy for
CAH.
Eligibility:
- Men and women at least 18 years of age who have CAH (see more details in Eligibility
section below).
Design:
- This study will involve four inpatient hospital stays at the National Institutes of
Health in Bethesda, MD over 6 months (spaced 2 months apart). The first and last stays
will last about 5 days. The second and third stays will last about 3 days.
- Participants will be screened with a physical exam and medical history. Blood and urine
samples will be collected.
- At the first study visit, participants will provide regular blood and urine samples.
They will also have imaging studies. These studies will look at the bones, fat, and
muscles in the abdomen and pelvis.
- Participants will receive a cortisol pump during the first visit. They will be shown how
to use the pump. They will also learn what to do, if they need to take extra "stress
dose" cortisol pills.
- At the second and third visits, the cortisol dose given with the pump will be adjusted
as needed. Blood and urine samples will also be collected. No imaging studies are
scheduled for these visits.
- The last study visit will have the same tests as the first visit. Participants will be
offered the chance to continue with the pump treatment for 1 more year, or go back to
their standard pill treatment.
Study type: Interventional non-randomized trial
Official title: A Pilot Study Assessing the use of Continuous Subcutaneous Hydrocortisone
Infusion In the Treatment of Congenital Adrenal Hyperplasia
Estimated enrollment: 8
Study Start Date: May 2013
Estimated Study Completion Date: December 2016
Sponsoring Institute: National Institute of Child Health and Human Development
ELIGIBILITY
Inclusion criteria
1. Men and women 18 years of age or older with classic congenital adrenal hyperplasia
(21-Hydroxylase deficiency)
2. High adrenal androgens in the blood, and
3. One or more of the following conditions: obesity, fatty liver, risk for diabetes, low
bone mass, inability to tolerate cortisol pills
Exclusion criteria
1. Pregnancy
2. Breast feeding
3. Use of inhaled or oral steroids for diseases other than CAH
4. Use of estrogen-containing birth control pills
5. Use of medicines that cross-react with hydrocortisone
6. Use of stress dose steroids for illness during the last 30 days prior to joining the
study
Phase:
Phase 2
Details
Lead Sponsor:
National Institutes of Health Clinical Center (CC)
Collaborator:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)