Hypophosphatasia
Conditions
Keywords
HPP, Bone Disease, Soft Bones, Low Alkaline Phosphatase, Genetic Metabolic Disorder, Alkaline Phosphatase, Rickets, Osteomalacia
Brief summary
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.
Detailed description
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.
Interventions
BIOLOGICALAsfotase Alfa
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Patient or parent (or legal guardian) must provide written informed consent prior to the performance of any study-related procedures and must be willing to comply with study procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained. 2. Patient has completed the investigator-initiated clinical study (HPPJEAP-01) protocol for asfotase alfa
Exclusion criteria
1. Patient has a documented form of rickets caused by a condition other than HPP, including, but not limited to, rickets caused by 25(OH) vitamin D deficiency 2. Patient has serum calcium and/or phosphorus levels below the normal range 3. Patient is pregnant or lactating 4. Patient received treatment with bisphosphonates within 2 years prior to the Screening visit 5. Patient has a documented sensitivity to any of the components of asfotase alfa 6. Patient is currently enrolled in any other program or clinical study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation) 7. Patient has clinically significant other disease in the opinion of the Investigator, defined as any other non HPP-related condition for which the patient is considered medically unstable.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) | 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. | 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. |
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 13 |
| Total | 13 |
Baseline characteristics
| Characteristic | Overall Study |
|---|---|
| Age, Categorical <=18 years | 12 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Age, Continuous | 1.39 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 13 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 12 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Region of Enrollment Japan | 13 Participants |
| Sex: Female, Male Female | 9 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 9 / 13 |
| serious Total, serious adverse events | 0 / 13 |
Outcome results
Primary
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.
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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Overall Study | Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) | Number of Patients with AEs | 9 Participants |
| Overall Study | Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) | Number of Patients with ISRs | 2 Participants |
| Overall Study | Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) | Number of Patients with IARs | 0 Participants |