Trial Outcomes & Findings for Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan (NCT NCT02531867)
NCT ID: NCT02531867
Last Updated: 2017-05-08
Results Overview
Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
13 participants
Primary outcome timeframe
Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
Results posted on
2017-05-08
Participant Flow
There were 11 study sites, all in Japan; 13 patients were enrolled into this study. The first patient signed informed consent in June 2015; the study continued through November 2015 at multiple centers in Japan.
Participant milestones
| Measure |
Overall Study
Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week.
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|---|---|
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Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
Baseline characteristics by cohort
| Measure |
Overall Study
n=13 Participants
Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week.
|
|---|---|
|
Age, Categorical
<=18 years
|
12 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
1.39 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
12 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Japan
|
13 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.Population: All patients who met all of the inclusion and none of the exclusion criteria were defined as the enrolled population, which was also the analysis population.The safety set comprised all patients who received at least 1 dose of the investigational drug.
Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study.
Outcome measures
| Measure |
Overall Study
n=13 Participants
Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week.
|
|---|---|
|
Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)
Number of Patients with AEs
|
9 Participants
|
|
Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)
Number of Patients with ISRs
|
2 Participants
|
|
Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)
Number of Patients with IARs
|
0 Participants
|
Adverse Events
Overall Study
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Overall Study
n=13 participants at risk
Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week.
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|---|---|
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Gastrointestinal disorders
Enterocolitis
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
General disorders
Injection site bruising
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
General disorders
Injection site erythema
|
7.7%
1/13 • Number of events 3 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
General disorders
Injection site induration
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Infections and infestations
Bronchitis
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Infections and infestations
Conjunctivitis viral
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Infections and infestations
Nasopharyngitis
|
15.4%
2/13 • Number of events 3 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Infections and infestations
Upper respiratory tract infection
|
15.4%
2/13 • Number of events 5 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Infections and infestations
Viral rash
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Injury, poisoning and procedural complications
Feeding tube complicatino
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Nervous system disorders
Intercostal neuralgia
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
7.7%
1/13 • Number of events 1 • Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Investigators are subject to certain disclosure and publication limitations. In cases of publication of the study results at conferences or on academic journals, the post-approval clinical study coordinating investigator and the study sponsor should discuss about details of publication, publication methods and presenters before decision-making
- Publication restrictions are in place
Restriction type: OTHER