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Calcitonin for Treating X-linked Hypophosphatemia

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ClinicalTrials.gov Identifier: NCT01652573
Recruitment Status : Completed
First Posted : July 30, 2012
Results First Posted : May 31, 2017
Last Update Posted : May 31, 2017
Sponsor:
Collaborator:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
Karl Insogna, Yale University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Hypophosphatemic Rickets, X Linked Dominant
Interventions Drug: nasal salmon calcitonin
Drug: Saline Nasal Spray Placebo
Enrollment 21
Recruitment Details 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.
Pre-assignment Details 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.
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Period Title: Overall Study
Started 10 11
Completed 10 10
Not Completed 0 1
Reason Not Completed
Adverse Event             0             1
Arm/Group Title Nasal Calictonin Saline Nasal Spray Total
Hide Arm/Group Description

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Total of all reporting groups
Overall Number of Baseline Participants 10 11 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 10 participants 11 participants 21 participants
47
(26 to 66)
47
(32 to 64)
47
(26 to 66)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 11 participants 21 participants
Female
9
  90.0%
7
  63.6%
16
  76.2%
Male
1
  10.0%
4
  36.4%
5
  23.8%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants 11 participants 21 participants
10 11 21
1.Primary Outcome
Title Area Under the Curve for FGF23
Hide Description FGF23 will be measured 0 to 24 hours post dose during a 24 hour admission and AUC calculated.
Time Frame Time 0
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Least Squares Mean (95% Confidence Interval)
Unit of Measure: pg/ml*hr
3172.34
(3068.7 to 3332.68)
3215.34
(3107.17 to 3374.97)
2.Primary Outcome
Title Area Under the Curve for FGF23
Hide Description 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.
Time Frame 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Least Squares Mean (95% Confidence Interval)
Unit of Measure: pg/ml*hr
2698.38
(2623.07 to 2830.39)
2994.26
(2907.61 to 3134.26)
3.Secondary Outcome
Title Area Under the Curve for TmP/GFR
Hide Description Serum phosphate will be measured 0 to 24 hours postdose during a 24 hr admission, AUC calculated, and fasting Tmp/GFR calculated.
Time Frame Time 0
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Least Squares Mean (95% Confidence Interval)
Unit of Measure: mg/100 ml GF*hr
30.70
(29.5 to 32.27)
29.97
(28.78 to 31.49)
4.Secondary Outcome
Title Area Under the Curve for 1,25(OH)2vitamin D
Hide Description Serum 1,25(OH)2vitamin D will be measured 0 to 24 hours post dose during a 24 hr admission and AUC calculated.
Time Frame Time 0
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Least Squares Mean (95% Confidence Interval)
Unit of Measure: ng/ml*hr
904.06
(862.86 to 961.90)
838.58
(799.18 to 892.99)
5.Secondary Outcome
Title Number of Patients With Nasal Congestion at Baseline
Hide Description This symptom will be assessed at baseline
Time Frame Time 0
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
6.Secondary Outcome
Title Area Under the Curve for TmP/GFR
Hide Description 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.
Time Frame Time 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Least Squares Mean (95% Confidence Interval)
Unit of Measure: mg/100 ml GF*hr
32.17
(31.04 to 33.68)
31.26
(30.13 to 32.74)
7.Secondary Outcome
Title Area Under the Curve for 1,25(OH)2vitamin D
Hide Description 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.
Time Frame Time 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Least Squares Mean (95% Confidence Interval)
Unit of Measure: ng/ml*hr
1277.05
(1235.72 to 1335.02)
1258.84
(1217.74 to 1315.70)
8.Secondary Outcome
Title Number of Participants With Nasal Congestion at 1 Month
Hide Description This symptom will be assessed.
Time Frame Time 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Measure Type: Count of Participants
Unit of Measure: Participants
3
  30.0%
2
  18.2%
9.Secondary Outcome
Title Number of Participants With Nasal Congestion at 2 Months
Hide Description This symptom will be assessed.
Time Frame Time 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
1
  10.0%
0
   0.0%
10.Secondary Outcome
Title Number of Participants With Nasal Congestion at 3 Months
Hide Description This symptom will be assessed.
Time Frame Time 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
11.Secondary Outcome
Title Number of Participants With Nasal Ulcerations at Baseline
Hide Description This symptom will be assessed at baseline
Time Frame Time 0
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
12.Secondary Outcome
Title Number of Participants With Allergic Reactions at Baseline
Hide Description This symptom will be assessed at baseline
Time Frame Time 0
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
13.Secondary Outcome
Title Number of Participants With Nasal Ulceration at 1 Month
Hide Description This symptom will be assessed.
Time Frame Time 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
2
  18.2%
14.Secondary Outcome
Title Number of Participants With Allergic Reactions at 1 Month
Hide Description This symptom will be assessed.
Time Frame Time 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 11
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
15.Secondary Outcome
Title Number of Participants With Nasal Ulceration at 2 Months
Hide Description This symptom will be assessed.
Time Frame Time 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
16.Secondary Outcome
Title Number of Participants With Allergic Reactions at 2 Months
Hide Description This symptom will be assessed.
Time Frame Time 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
17.Secondary Outcome
Title Number of Participants With Nasal Ulcerations at 3 Months
Hide Description This symptom will be assessed.
Time Frame Time 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
1
  10.0%
18.Secondary Outcome
Title Number of Participants With Allergic Reactions at 3 Months
Hide Description This symptom will be assessed.
Time Frame Time 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description:

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
Time Frame Adverse events were collected from the baseline visit until the end of study at 3 months.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Nasal Calictonin Saline Nasal Spray
Hide Arm/Group Description

Subjects will received nasal calcitonin once daily

nasal salmon calcitonin: 400 IU daily in two sprays (one to each nares)

Patients will receive saline nasal spray once daily

Saline Nasal Spray Placebo

All-Cause Mortality
Nasal Calictonin Saline Nasal Spray
Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)      0/11 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Nasal Calictonin Saline Nasal Spray
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/10 (0.00%)      0/11 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Nasal Calictonin Saline Nasal Spray
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/10 (20.00%)      2/11 (18.18%)    
Musculoskeletal and connective tissue disorders     
musculoskeletal events   1/10 (10.00%)  1 2/11 (18.18%)  2
Respiratory, thoracic and mediastinal disorders     
Respiratory events   2/10 (20.00%)  2 2/11 (18.18%)  2
Skin and subcutaneous tissue disorders     
Dermatologic   1/10 (10.00%)  1 2/11 (18.18%)  2
Indicates events were collected by systematic assessment
It is possible that there is a threshold serum concentration of drug required to suppress FGF23 levels and our sample size was small.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Karl Insogna
Organization: Yale School of Medicine
Phone: 203-737-2871
Responsible Party: Karl Insogna, Yale University
ClinicalTrials.gov Identifier: NCT01652573     History of Changes
Other Study ID Numbers: 1010007548
R21AR061818 ( U.S. NIH Grant/Contract )
First Submitted: July 23, 2012
First Posted: July 30, 2012
Results First Submitted: March 6, 2017
Results First Posted: May 31, 2017
Last Update Posted: May 31, 2017