A Phase 4 interventional study of Asfotase Alfa in Hypophosphatasia, sponsored by Alexion Pharmaceuticals, Inc.. Completed. Per ClinicalTrials.gov, last updated 2017-05-08.
Sponsored by Alexion Pharmaceuticals, Inc. · Phase 4, Interventional, and Treatment
This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further information on the safety and efficacy of treatment with asfotase alfa.
The primary objective of this study was to collect data on the safety of repeated subcutaneous (SC) injections of asfotase alfa. There were no secondary objectives. Exploratory objectives related to the efficacy of asfotase alfa are not being reported.
45 studies on the registry are indexed under Hypophosphatasia; 10 are open to participants now.
This study's enrollment of 13 is close to the median of 14 across 18 interventional studies indexed under Hypophosphatasia.
Browse Hypophosphatasia studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 249 studies on the registry; 25 are open to participants now.
Of its 98 completed or terminated interventional studies of FDA-regulated products, 70 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Patients will receive asfotase alfa by subcutaneous injection. Asfotase alfa will be administered at either 2 mg/kg 3 times per week or 1 mg/kg 6 times per week depending on investigator's discretion.
Biological: Asfotase Alfa
Also known as: Strensiq
Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)
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.
Time frame: 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.
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.
| Milestone | Overall Study |
|---|---|
| Started | 13 |
| Completed | 13 |
| Not completed | 0 |
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.
| Participants | Overall Study |
|---|---|
| Number of Patients with AEs | 9 |
| Number of Patients with ISRs | 2 |
| Number of Patients with IARs | 0 |
Collected over 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.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Overall Study | — | 0/13 (0%) | 9/13 (69.2%) |
| Event | Overall Study |
|---|---|
| NasopharyngitisInfections and infestations | 2/13 |
| Upper respiratory tract infectionInfections and infestations | 2/13 |
| EnterocolitisGastrointestinal disorders | 1/13 |
| Injection site bruisingGeneral disorders | 1/13 |
| Injection site erythemaGeneral disorders | 1/13 |
| Injection site indurationGeneral disorders | 1/13 |
| BronchitisInfections and infestations | 1/13 |
| Conjunctivitis viralInfections and infestations | 1/13 |
| Viral rashInfections and infestations | 1/13 |
| Feeding tube complicatinoInjury, poisoning and procedural complications | 1/13 |
All patients who met all of the inclusion and none of the exclusion criteria were considered the Baseline Analysis Population.
| Age, Categorical(Participants) | Overall Study |
|---|---|
| <=18 years | 12 |
| Between 18 and 65 years | 1 |
| >=65 years | 0 |
| Age, Continuous(years) | Overall Study |
|---|---|
| Median | 1.39 (0.13 to 35.04) |
| Sex: Female, Male(Participants) | Overall Study |
|---|---|
| Female | 9 |
| Male | 4 |
| Ethnicity (NIH/OMB)(Participants) | Overall Study |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 0 |
| Unknown or Not Reported | 13 |
| Race (NIH/OMB)(Participants) | Overall Study |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 12 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 0 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | Overall Study |
|---|---|
| Japan | 13 |
No study locations are listed for this record.
This study is completed, as verified in Mar 2017. You cannot join it, but the record below documents what was studied.
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Alexion Pharmaceuticals, Inc.