Trial Outcomes & Findings for A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP) (NCT NCT01419028)
NCT ID: NCT01419028
Last Updated: 2019-04-01
Results Overview
Overall survival is defined as the time from birth to time of death.
COMPLETED
48 participants
Retrospective data collected on or before the data of abstraction.
2019-04-01
Participant Flow
Patients were enrolled between Sept 2012 and Apr 2013. 65 patients were screened and 48 were enrolled at 12 sites in 7 countries. Patients were recruited from investigational sites established at academic research and medical centers known to diagnose and/or treat patients with HPP. Both living and deceased patients were considered to partricipate.
Participant milestones
| Measure |
Retrospective Observational
Patients with severe perinatal and/or infantile onset HPP (ie, where signs of the disease are present before 6 months of age).
|
|---|---|
|
Overall Study
STARTED
|
48
|
|
Overall Study
COMPLETED
|
48
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
Baseline characteristics by cohort
| Measure |
Retrospective Observational
n=48 Participants
Patients with severe perinatal and/or infantile onset HPP (ie, where signs of the disease are present before 6 months of age).
|
|---|---|
|
Age, Continuous
|
506.9 weeks
STANDARD_DEVIATION 319.24 • n=5 Participants
|
|
Age, Customized
<=18 years
|
10 weeks
n=5 Participants
|
|
Age, Customized
Between 18 and 65 years
|
3 weeks
n=5 Participants
|
|
Age, Customized
>=65 years
|
0 weeks
n=5 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
40 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
41 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
11 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Switzerland
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Taiwan
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
26 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Retrospective data collected on or before the data of abstraction.Overall survival is defined as the time from birth to time of death.
Outcome measures
| Measure |
Retrospective Observational
n=48 Participants
Patients with severe perinatal and/or infantile onset HPP (ie, where signs of the disease are present before 6 months of age).
|
|---|---|
|
Survival
|
270.5 days
Interval 155.0 to 428.0
|
SECONDARY outcome
Timeframe: Retrospective data collected on or before the date of abstraction.Invasive ventilator-free survival is defined as the time during which the patient is alive and not invasively ventilated. For the purpose of this study, invasive ventilation is defined as mechanical ventilation via intubation of trachaeostomy.
Outcome measures
| Measure |
Retrospective Observational
n=48 Participants
Patients with severe perinatal and/or infantile onset HPP (ie, where signs of the disease are present before 6 months of age).
|
|---|---|
|
Invasive Ventilator-free Survival Time
|
236 days
Interval 78.0 to 300.0
|
Adverse Events
Retrospective Observational
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Participation in a multicenter study involves a commitment to publish the data from the study in a cooperative publication prior to release of study results on an individual basis. Therefore, PIs are subject to certain disclosure and publication limitations.
- Publication restrictions are in place
Restriction type: OTHER