Trial Outcomes & Findings for Calcitonin for Treating X-linked Hypophosphatemia (NCT NCT01652573)

NCT ID: NCT01652573

Last Updated: 2017-05-31

Results Overview

FGF23 will be measured 0 to 24 hours post dose during a 24 hour admission and AUC calculated.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 21 participants
• Primary outcome timeframe: Time 0
• Results posted on: 2017-05-31

Participant Flow

Subjects were recruited from the individual practices of two of the physicians on this study and from a panel of patients with XLH who had previously participated or inquired about participation in ongoing clinical trials at this institution.

Patients who were receiving conventional therapy with calcitriol and phosphorus at the time of screening were asked to stop both agents two weeks prior to enrolling in the study and no subjects took calcitriol or phosphorus during the entire study.

Participant milestones

Measure	Nasal Calictonin Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Overall Study STARTED	10	11
Overall Study COMPLETED	10	10
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Measure	Nasal Calictonin Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Overall Study Adverse Event	0	1

Baseline Characteristics

Calcitonin for Treating X-linked Hypophosphatemia

Baseline characteristics by cohort

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo	Total n=21 Participants Total of all reporting groups
Age, Continuous	47 years n=5 Participants	47 years n=7 Participants	47 years n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants	7 Participants n=7 Participants	16 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	4 Participants n=7 Participants	5 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants	11 participants n=7 Participants	21 participants n=5 Participants

PRIMARY outcome

Timeframe: Time 0

FGF23 will be measured 0 to 24 hours post dose during a 24 hour admission and AUC calculated.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Area Under the Curve for FGF23	3172.34 pg/ml*hr Interval 3068.7 to 3332.68	3215.34 pg/ml*hr Interval 3107.17 to 3374.97

PRIMARY outcome

Timeframe: 3 months

FGF23 will be measured 0 to 24 hours post dose during a 24 hour admission at 3 months and AUC calculated and compared to baseline.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Area Under the Curve for FGF23	2698.38 pg/ml*hr Interval 2623.07 to 2830.39	2994.26 pg/ml*hr Interval 2907.61 to 3134.26

SECONDARY outcome

Timeframe: Time 0

Serum phosphate will be measured 0 to 24 hours postdose during a 24 hr admission, AUC calculated, and fasting Tmp/GFR calculated.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Area Under the Curve for TmP/GFR	30.70 mg/100 ml GF*hr Interval 29.5 to 32.27	29.97 mg/100 ml GF*hr Interval 28.78 to 31.49

SECONDARY outcome

Timeframe: Time 0

Serum 1,25(OH)2vitamin D will be measured 0 to 24 hours post dose during a 24 hr admission and AUC calculated.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Area Under the Curve for 1,25(OH)2vitamin D	904.06 ng/ml*hr Interval 862.86 to 961.9	838.58 ng/ml*hr Interval 799.18 to 892.99

SECONDARY outcome

Timeframe: Time 0

This symptom will be assessed at baseline

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Patients With Nasal Congestion at Baseline	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 3 months

TmP/GFR will be measured 0 to 24 hours postdose during a 24 hr admission at 3 months and AUC calculated and compared to baseline.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Area Under the Curve for TmP/GFR	32.17 mg/100 ml GF*hr Interval 31.04 to 33.68	31.26 mg/100 ml GF*hr Interval 30.13 to 32.74

SECONDARY outcome

Timeframe: Time 3 months

Serum 1,25(OH)2vitamin D will be measured 0 to 24 hours post dose during a 24 hr admission and AUC calculated and results will be compared to baseline values.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Area Under the Curve for 1,25(OH)2vitamin D	1277.05 ng/ml*hr Interval 1235.72 to 1335.02	1258.84 ng/ml*hr Interval 1217.74 to 1315.7

SECONDARY outcome

Timeframe: Time 1 month

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Nasal Congestion at 1 Month	3 Participants	2 Participants

SECONDARY outcome

Timeframe: Time 2 months

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=10 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Nasal Congestion at 2 Months	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 3 months

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=10 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Nasal Congestion at 3 Months	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 0

This symptom will be assessed at baseline

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Nasal Ulcerations at Baseline	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 0

This symptom will be assessed at baseline

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Allergic Reactions at Baseline	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 1 month

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Nasal Ulceration at 1 Month	0 Participants	2 Participants

SECONDARY outcome

Timeframe: Time 1 month

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Allergic Reactions at 1 Month	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 2 months

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=10 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Nasal Ulceration at 2 Months	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 2 months

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=10 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Allergic Reactions at 2 Months	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Time 3 months

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=10 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Nasal Ulcerations at 3 Months	0 Participants	1 Participants

SECONDARY outcome

Timeframe: Time 3 months

This symptom will be assessed.

Outcome measures

Measure	Nasal Calictonin n=10 Participants Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=10 Participants Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Number of Participants With Allergic Reactions at 3 Months	0 Participants	0 Participants

Adverse Events

Nasal Calictonin

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Saline Nasal Spray

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Nasal Calictonin n=10 participants at risk Subjects will received nasal calcitonin once daily nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)	Saline Nasal Spray n=11 participants at risk Patients will receive saline nasal spray once daily Saline Nasal Spray Placebo
Skin and subcutaneous tissue disorders Dermatologic	10.0% 1/10 • Number of events 1 • Adverse events were collected from the baseline visit until the end of study at 3 months.	18.2% 2/11 • Number of events 2 • Adverse events were collected from the baseline visit until the end of study at 3 months.
Musculoskeletal and connective tissue disorders musculoskeletal events	10.0% 1/10 • Number of events 1 • Adverse events were collected from the baseline visit until the end of study at 3 months.	18.2% 2/11 • Number of events 2 • Adverse events were collected from the baseline visit until the end of study at 3 months.
Respiratory, thoracic and mediastinal disorders Respiratory events	20.0% 2/10 • Number of events 2 • Adverse events were collected from the baseline visit until the end of study at 3 months.	18.2% 2/11 • Number of events 2 • Adverse events were collected from the baseline visit until the end of study at 3 months.

Additional Information

Dr. Karl Insogna

Yale School of Medicine

Phone: 203-737-2871

Email: karl.insogna@yale.edu

Results disclosure agreements

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