Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasia

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01859312
• Date of registration: 17/05/2013
• Prospective Registration: No
• Primary sponsor: National Institutes of Health Clinical Center (CC)
• Public title: Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasia
• Scientific title: A Pilot Study Assessing the Use of Continuous Subcutaneous Hydrocortisone Infusion in the Treatment of Congenital Adrenal Hyperplasia
• Date of first enrolment: May 6, 2013
• Target sample size: 8
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01859312
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Deborah P Merke, M.D.
• Affiliation: National Institutes of Health Clinical Center (CC)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

- Patients with known classic CAH due to 21-hydroxylase deficiency as evidenced by
hormonal and genetic testing

- Male or female patients 18 years or older

- Females must have a negative pregnancy test initially and at all visits. Sexually
active females must be using a medically acceptable method of contraception.

- Patients with elevated adrenal androgens (defined as 17-OHP >1200 ng/dL and
androstenedione >210 ng/dL)

- One or more co-morbidities:

- Obesity [body mass index (BMI) greater than 30.0 kg/m(2)]

- Fatty liver disease; assessed by AST/ALT liver enzyme ratio (AST to ALT ratio <1 (11))
liver ultrasound or MRI imaging (Steatosis score as previously described)

- Low insulin sensitivity; assessed by the Homeostasis Model Assessment Insulin
Resistance (HOMA-IR) method [HOMA-IR = insulin (micro U/ml) times glucose (mmol/L)/
22.5]. Elevated HOMA-IR index is defined as >2.6 in adults17.

- Osteopenia [bone mineral density by DEXA (at the spine, hip, or forearm) with T-score
of -1 to -2.5) or osteoporosis (bone mineral density by DEXA (at the spine, hip, or
forearm) with T-score of <-2.5] defined according to World Health Organization (WHO).

- Glucocorticoid-related gastrointestinal side effects (nausea, vomiting, dyspepsia,
anorexia, gastritis, peptic ulcer disease and gastric bleeding)

EXCLUSION CRITERIA:

- Co-morbid conditions requiring daily administration of medications that induce hepatic
enzymes or interfere with the metabolism of glucocorticoids

- Females who are pregnant or lactating

- Patients on inhaled or oral steroids given for reasons other than treatment of CAH

- Women who have taken estrogen-containing oral contraceptive pills within 6 weeks of
recruitment

- Patients who required stress dose glucocorticoids for an illness within 4 weeks of
recruitment

- Patients who changed their glucocorticoid agent within 3 months of recruitment

- Patients who underwent bilateral adrenalectomy

- Co-morbid conditions that could interfere with the ability to comply to the protocol



Age minimum: 18 Years
Age maximum: 99 Years
Gender: All

Health Condition(s) or Problem(s) studied

Adrenal Insufficiency

Congenital Adrenal Hyperplasia (CAH)

Excess Androgen

Intervention(s)

Drug: Hydrocortisone (Solucortef)

Device: Insulin pump (Medtronic)

Primary Outcome(s)

Number of Patients With 17-OH Progesterone Levels Equal or Below 1,200 ng/dL at 0700 [Time Frame: At 6 months]

Number of Patients With 17-OHP Levels Equal or Below 1,200 ng/dL at 0700 [Time Frame: At baseline]

Secondary Outcome(s)

Participant Lean Body Mass [Time Frame: At 6 months]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - 24 Hours [Time Frame: At baseline]

Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours [Time Frame: At baseline]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At 6 months]

Participant Lean Body Mass [Time Frame: At baseline]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - 24 Hours [Time Frame: At 6 months]

Participants Mean Level of Androstenedione at 0700 [Time Frame: At 6 months]

Participants Mean Level of Androstenedione at 0700 [Time Frame: At baseline]

Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At baseline]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - 24 Hours [Time Frame: At baseline]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At baseline]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - 24 Hours [Time Frame: At 6 months]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At baseline]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At 6 months]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At 6 months]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At baseline]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At 6 months]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At baseline]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At baseline]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At baseline]

Participants Mean Level of 17-OHP at 0700 [Time Frame: At baseline]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - 24 Hours [Time Frame: At baseline]

Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At 6 months]

Participants Mean Level of ACTH at 0700 [Time Frame: At 6 months]

Participants Mean Level of ACTH at 0700 [Time Frame: At baseline]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At 6 months]

Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At 6 months]

Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At baseline]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At baseline]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At 6 months]

Participants Mean Progesterone Level at 0700 [Time Frame: At 6 months]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - 24 Hours [Time Frame: At 6 months]

Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At 6 months]

Participants Mean Progesterone Levels at 0700 [Time Frame: At baseline]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At 6 months]

Participants Mean Level of Progesterone Area Under the Curve (AUC) - Daytime (0700-1500) [Time Frame: At baseline]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At 6 months]

Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At baseline]

Participants Mean Level of 17-OHP at 0700 [Time Frame: At 6 months]

Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours [Time Frame: At 6 months]

Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300) [Time Frame: At baseline]

Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700) [Time Frame: At 6 months]

Secondary ID(s)

13-CH-0121

130121

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

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

Results

• Results available: Yes
• Date Posted: 22/11/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01859312