Trial Outcomes & Findings for Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy) (NCT NCT02463409)

NCT ID: NCT02463409

Last Updated: 2017-06-26

Results Overview

Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 6 participants
• Primary outcome timeframe: 1 day
• Results posted on: 2017-06-26

Participant Flow

The screening visit included labs, physical exam and medical history, prior to assignment to the theophylline arm.

Participant milestones

Measure	Theophylline Patients will receive a 24 hour continuous infusion of intravenous theophylline. Theophylline: 24 hour infusion of IV theophylline
Overall Study STARTED	6
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Theophylline Patients will receive a 24 hour continuous infusion of intravenous theophylline. Theophylline: 24 hour infusion of IV theophylline
Overall Study Withdrawal by Subject	1
Overall Study Screen failure	1

Baseline Characteristics

Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)

Baseline characteristics by cohort

Measure	Theophylline n=5 Participants Patients will receive a 24 hour continuous infusion of intravenous theophylline. Theophylline: 24 hour infusion of IV theophylline
Age, Continuous	14.4 years STANDARD_DEVIATION 3.1 • n=93 Participants
Sex: Female, Male Female	5 Participants n=93 Participants
Sex: Female, Male Male	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	5 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	5 participants n=93 Participants

PRIMARY outcome

Timeframe: 1 day

Population: Patients treated with theophylline who maintained appropriate IV access. Only 3 patients had complete data available.

Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline

Outcome measures

Measure	Theophylline n=3 Participants Patients will receive a 24 hour continuous infusion of intravenous theophylline. Theophylline: 24 hour infusion of IV theophylline
Change in Urine cAMP	3048 fm/uL Standard Deviation 8369

SECONDARY outcome

Timeframe: 1 day

Change in REE before and during treatment with theophylline

Outcome measures

Measure	Theophylline n=4 Participants Patients will receive a 24 hour continuous infusion of intravenous theophylline. Theophylline: 24 hour infusion of IV theophylline
Change in Resting Energy Expenditure (REE)	-14 kcals per day Standard Error 68

SECONDARY outcome

Timeframe: 1 day

Change in AHI before and during treatment with theophylline

Outcome measures

Measure	Theophylline n=4 Participants Patients will receive a 24 hour continuous infusion of intravenous theophylline. Theophylline: 24 hour infusion of IV theophylline
Change in Apnea Hypopnea Index (AHI)	-0.60 units on a scale Standard Error 0.67

Adverse Events

Theophylline

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Theophylline n=5 participants at risk Patients will receive a 24 hour continuous infusion of intravenous theophylline. Theophylline: 24 hour infusion of IV theophylline. All patients who enrolled in the study are included in this group (excluding screen fails).
Gastrointestinal disorders Nausea	60.0% 3/5 • Number of events 3 • 3 days We specifically asked patients about headache, nausea and vomiting at least twice daily. They were monitored continuously via telemetry for arrhythmia. Other adverse events were reported as described by the partiicipant.
Nervous system disorders Headache	60.0% 3/5 • Number of events 3 • 3 days We specifically asked patients about headache, nausea and vomiting at least twice daily. They were monitored continuously via telemetry for arrhythmia. Other adverse events were reported as described by the partiicipant.
Gastrointestinal disorders Abdominal pain	20.0% 1/5 • Number of events 1 • 3 days We specifically asked patients about headache, nausea and vomiting at least twice daily. They were monitored continuously via telemetry for arrhythmia. Other adverse events were reported as described by the partiicipant.
Cardiac disorders Tachycardia	20.0% 1/5 • Number of events 1 • 3 days We specifically asked patients about headache, nausea and vomiting at least twice daily. They were monitored continuously via telemetry for arrhythmia. Other adverse events were reported as described by the partiicipant.
Nervous system disorders dizziness	20.0% 1/5 • Number of events 1 • 3 days We specifically asked patients about headache, nausea and vomiting at least twice daily. They were monitored continuously via telemetry for arrhythmia. Other adverse events were reported as described by the partiicipant.
Gastrointestinal disorders Vomiting	20.0% 1/5 • Number of events 1 • 3 days We specifically asked patients about headache, nausea and vomiting at least twice daily. They were monitored continuously via telemetry for arrhythmia. Other adverse events were reported as described by the partiicipant.

Additional Information

Dr. Ashley Shoemaker

Vanderbilt University Medical Center

Phone: 615-343-8816

Email: ashley.h.shoemaker@vanderbilt.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place