Trial Outcomes & Findings for Bone Properties in Hypoparathyroidism: Effects of PTH (NCT NCT00473265)
NCT ID: NCT00473265
Last Updated: 2015-07-16
Results Overview
Serum and urinary calcium levels maintained by change in requirements for calcium supplementation
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
68 participants
Primary outcome timeframe
2 years
Results posted on
2015-07-16
Participant Flow
68 subjects were enrolled from 05/12/2004 to 06/19/2014 at Columbia University Medical Center.
The first 6 participants in the study were randomly assigned to PTH1-84 100mcg daily, 100mcg every other day, and 100mcg every three days as a pilot project to determine the correct dosing regimen.
Participant milestones
| Measure |
PTH1-84
participants received PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Overall Study
STARTED
|
68
|
|
Overall Study
COMPLETED
|
48
|
|
Overall Study
NOT COMPLETED
|
20
|
Reasons for withdrawal
| Measure |
PTH1-84
participants received PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Overall Study
Unrelated adverse event
|
5
|
|
Overall Study
Off treatment early
|
15
|
Baseline Characteristics
Bone Properties in Hypoparathyroidism: Effects of PTH
Baseline characteristics by cohort
| Measure |
PTH1-84
n=68 Participants
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
66 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=99 Participants
|
|
Age, Continuous
|
45 years
STANDARD_DEVIATION 27 • n=99 Participants
|
|
Sex: Female, Male
Female
|
50 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
68 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 2 yearsPopulation: Only the first 30 participants to complete 24 months in the study were analyzed
Serum and urinary calcium levels maintained by change in requirements for calcium supplementation
Outcome measures
| Measure |
PTH1-84
n=30 Participants
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Requirements for Calcium Supplementation
Starting requirements
|
3.03 grams per day of calcium supplementation
Standard Deviation 2.32
|
|
Requirements for Calcium Supplementation
Requirements at month 24
|
1.66 grams per day of calcium supplementation
Standard Deviation 1.26
|
SECONDARY outcome
Timeframe: baseline versus 24 monthsPopulation: Only the first 30 participants who completed 24 months of the study were analyzed
Bone Mineral Density (BMD) as measured by Dual-energy X-ray absorptiometry (DXA).
Outcome measures
| Measure |
PTH1-84
n=30 Participants
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Percent Change in BMD From Baseline to 24 Months by DXA
Lumbar Spine BMD
|
2.9 percentage of change to BMD
Standard Deviation 4
|
|
Percent Change in BMD From Baseline to 24 Months by DXA
Femoral Neck BMD
|
0.95 percentage of change to BMD
Standard Deviation .2
|
|
Percent Change in BMD From Baseline to 24 Months by DXA
Distal 1/3 Radius BMD
|
-2.4 percentage of change to BMD
Standard Deviation 4
|
SECONDARY outcome
Timeframe: baseline versus two yearsPopulation: Only 16 subjects had paired bone biopsies obtained at baseline and after 24 months of PTH(1-84) treatment.
Trabecular width was obtained from histomorphometric assessment of percutaneous iliac crest bone biopsy. Trabecular width is the thickness of individual pieces of the spongy bone section. The structure and microscopic organization of a small piece of biopsied pelvic bone was analyzed.
Outcome measures
| Measure |
PTH1-84
n=16 Participants
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Trabecular Width
Baseline
|
144 micrometers
Standard Deviation 34
|
|
Trabecular Width
Year 2
|
128 micrometers
Standard Deviation 34
|
SECONDARY outcome
Timeframe: baseline versus two yearsPopulation: Only 16 subjects had paired bone biopsies obtained at baseline and after 24 months of PTH(1-84) treatment.
trabecular number done on histomorphometric assessment of percutaneous iliac crest bone biopsy. Trabecular number is the number of individual pieces of the spongy bone section. The structure and microscopic organization of a small piece of biopsied pelvic bone was analyzed.
Outcome measures
| Measure |
PTH1-84
n=16 Participants
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Trabecular Number
Baseline
|
1.74 mm^-1
Standard Deviation 0.34
|
|
Trabecular Number
Year 2
|
2.07 mm^-1
Standard Deviation 0.5
|
SECONDARY outcome
Timeframe: baseline versus two yearsPopulation: Only 16 subjects had paired bone biopsies obtained at baseline and after 24 months of PTH(1-84) treatment.
Cortical porosity done on histomorphometric assessment of percutaneous iliac crest bone biopsy. Cortical Porosity measures how many tiny holes there are in the solid bone section. The structure and microscopic organization of a small piece of biopsied pelvic bone was analyzed.
Outcome measures
| Measure |
PTH1-84
n=16 Participants
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Cortical Porosity
Baseline
|
7.4 percentage of porosity
Standard Deviation 3.2
|
|
Cortical Porosity
Year 2
|
9.2 percentage of porosity
Standard Deviation 2.4
|
SECONDARY outcome
Timeframe: baseline versus one yearPopulation: Only 14 subjects had paird bone biopsies obtained at baseline and after 12 months of PTH(1-84) treatment.
Mineralizing surface done on histomorphometric assessment of percutaneous iliac crest bone biopsy. Mineralizing surface measures how much of bone is getting new mineral put on it. The structure and microscopic organization of a small piece of biopsied pelvic bone was analyzed.
Outcome measures
| Measure |
PTH1-84
n=14 Participants
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Mineralizing Surface
Baseline
|
0.7 percentage of surface
Standard Deviation 0.6
|
|
Mineralizing Surface
Year 1
|
7.1 percentage of surface
Standard Deviation 6.0
|
Adverse Events
PTH1-84
Serious events: 15 serious events
Other events: 68 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
PTH1-84
n=68 participants at risk
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Infections and infestations
Infection at bone biopsy site
|
1.6%
1/63 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Renal and urinary disorders
Right Kidney mass
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Musculoskeletal and connective tissue disorders
Abdominal Pain
|
2.9%
2/68 • Number of events 3 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Gastrointestinal disorders
pancreatitis, accute
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Hepatobiliary disorders
ampullary mass
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Blood and lymphatic system disorders
anemia
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Cardiac disorders
angina
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Nervous system disorders
concussion
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Surgical and medical procedures
damage to surgical site of recent hernia repair
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Blood and lymphatic system disorders
dehydration
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Surgical and medical procedures
cyst removal
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Endocrine disorders
symptomatic hypocalcemia
|
5.9%
4/68 • Number of events 9 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Respiratory, thoracic and mediastinal disorders
pneumonia
|
2.9%
2/68 • Number of events 2 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Musculoskeletal and connective tissue disorders
laryngeal spasm
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Blood and lymphatic system disorders
Fainted
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Reproductive system and breast disorders
L Oophorectomy
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Surgical and medical procedures
Elective Surgery
|
1.5%
1/68 • Number of events 1 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
Other adverse events
| Measure |
PTH1-84
n=68 participants at risk
participants recieved PTH1-84 in either 100mcg daily, every other day, or every three days based on investigators clinical judgement
|
|---|---|
|
Psychiatric disorders
Anxiety/Irritabilty/Stress
|
29.4%
20/68 • Number of events 29 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal Pain
|
69.1%
47/68 • Number of events 134 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.4%
5/68 • Number of events 5 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Skin and subcutaneous tissue disorders
Bruising at injection site
|
13.2%
9/68 • Number of events 9 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Respiratory, thoracic and mediastinal disorders
Chest Pain
|
11.8%
8/68 • Number of events 8 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Infections and infestations
Cold/Flu Virus/Symptoms
|
41.2%
28/68 • Number of events 32 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Gastrointestinal disorders
Constipation
|
10.3%
7/68 • Number of events 8 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Renal and urinary disorders
Decrease in Urination
|
10.3%
7/68 • Number of events 7 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Psychiatric disorders
Depression
|
16.2%
11/68 • Number of events 12 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Gastrointestinal disorders
Diarrhea
|
13.2%
9/68 • Number of events 9 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Nervous system disorders
Dizziness
|
30.9%
21/68 • Number of events 24 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Endocrine disorders
Fatigue
|
33.8%
23/68 • Number of events 29 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Nervous system disorders
Headache/Migraine
|
47.1%
32/68 • Number of events 42 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Endocrine disorders
Temperature Sensitivity
|
14.7%
10/68 • Number of events 11 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Endocrine disorders
Hypercalcemia
|
7.4%
5/68 • Number of events 6 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Blood and lymphatic system disorders
Hypertension
|
11.8%
8/68 • Number of events 8 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Renal and urinary disorders
Increased Urination
|
29.4%
20/68 • Number of events 22 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Nervous system disorders
Insomnia
|
23.5%
16/68 • Number of events 17 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal Irritability
|
60.3%
41/68 • Number of events 74 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Metabolism and nutrition disorders
Weight Fluctuations
|
20.6%
14/68 • Number of events 14 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Gastrointestinal disorders
Vomitting
|
5.9%
4/68 • Number of events 4 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Renal and urinary disorders
Increased Thirst
|
20.6%
14/68 • Number of events 14 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Gastrointestinal disorders
Gastrointestinal Pain
|
26.5%
18/68 • Number of events 23 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Gastrointestinal disorders
Nausea
|
50.0%
34/68 • Number of events 47 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
14.7%
10/68 • Number of events 10 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Psychiatric disorders
Confusion\Disorientation
|
16.2%
11/68 • Number of events 13 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Musculoskeletal and connective tissue disorders
Bone Fracture
|
8.8%
6/68 • Number of events 7 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Blood and lymphatic system disorders
Body Swelling
|
5.9%
4/68 • Number of events 6 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Respiratory, thoracic and mediastinal disorders
Difficulty Breathing
|
14.7%
10/68 • Number of events 10 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
7.4%
5/68 • Number of events 5 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Cardiac disorders
Arrhythmia
|
10.3%
7/68 • Number of events 9 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cysts
|
14.7%
10/68 • Number of events 12 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Gastrointestinal disorders
Heartburn
|
5.9%
4/68 • Number of events 4 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Infections and infestations
Infection
|
42.6%
29/68 • Number of events 42 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Renal and urinary disorders
Kidney Stone/Kidney Pain
|
11.8%
8/68 • Number of events 9 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Eye disorders
Opthalmological Irritability/Visual Impairment
|
16.2%
11/68 • Number of events 16 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
|
Endocrine disorders
Hypocalcemic Symptoms
|
8.8%
6/68 • Number of events 6 • earliest SAE report is 8/2/05 and latest SAE report is 6/19/09
|
Additional Information
John P. Bilezikian MD
Columbia University Medical Center
Phone: 212-305-2663
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place