HypophosphatasiaPhase 4NCT02531867

Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan

A Phase 4 study testing Asfotase Alfa in people with Hypophosphatasia, run by Alexion Pharmaceuticals, Inc..

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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. Read more about this study

Who is it for?: Any age
Conditions the study is for: Hypophosphatasia
What is being tested?: Asfotase Alfa
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 13 participants
Where: No sites listed yet
Study dates: Jun 2015to Nov 2015
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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.
Patient has completed the investigator-initiated clinical study (HPPJEAP-01) protocol for asfotase alfa

Who cannot join

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
Patient has serum calcium and/or phosphorus levels below the normal range
Patient is pregnant or lactating
Patient received treatment with bisphosphonates within 2 years prior to the Screening visit
Patient has a documented sensitivity to any of the components of asfotase alfa
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)
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.

About this study

Official title: A Multicenter, Post-Approval Clinical Study for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Hypophosphatasia (HPP) in Japan

Read the 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.

Conditions: Hypophosphatasia
Being tested: Asfotase Alfa
Keywords: HPP, Bone Disease, Soft Bones, Low Alkaline Phosphatase, Genetic Metabolic Disorder, Alkaline Phosphatase, Rickets, Osteomalacia
Medical subject headings: Hypophosphatasia, Bone Diseases, Rickets, Osteomalacia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Asfotase AlfaBiological
Also called Strensiq

Study groups in NCT02531867
Study group	Role	What they receive
Asfotase Alfa	Experimental	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. Receives: Biological: Asfotase Alfa

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2015Nov 2015

First posted25 Aug 2015 (estimated)
Study startJun 2015
Primary completionNov 2015
Study completionNov 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for HypophosphatasiaStudies of the same condition, open and under way.
More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 13 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02531867
Sponsor's protocol ID: AA-HPP-409
Status last verified: Mar 2017
First submitted: 11 Aug 2015
Last updated: 8 May 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

Enrolling by invitationObservational study
Registry of Patients With Hypophosphatasia
Hypophosphatasia (HPP)
Enrolling by invitationObservational study
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
Osteoporosis
RecruitingObservational study
Clarify HPP: Disease Burden of HPP Patients in China
Hypophosphatasia (HPP)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia
Hypophosphatasia

All Hypophosphatasia trials

Study at a glance

From the registry record, last updated by the sponsor on 8 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Post-Approval Clinical Study for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Hypophosphatasia (HPP) in Japan
ClinicalTrials.gov ID: NCT02531867
Acronym / sponsor ID: AA-HPP-409
Conditions: Hypophosphatasia
Keywords: HPP, Bone Disease, Soft Bones, Low Alkaline Phosphatase, Genetic Metabolic Disorder, Alkaline Phosphatase, Rickets, Osteomalacia
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 13 participants (actual)
Lead sponsor: Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
Study start: Jun 2015 (actual)
Primary completion: Nov 2015 (actual)
Study completion: Nov 2015 (actual)
First posted: 25 Aug 2015 (estimated)
Results: Posted 8 May 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Asfotase Alfa	Experimental	Biological: Asfotase AlfaPatients 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.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (2 listed)

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.
Patient has completed the investigator-initiated clinical study (HPPJEAP-01) protocol for asfotase alfa

Key exclusion criteria (7 listed)

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
Patient has serum calcium and/or phosphorus levels below the normal range
Patient is pregnant or lactating
Patient received treatment with bisphosphonates within 2 years prior to the Screening visit
Patient has a documented sensitivity to any of the components of asfotase alfa

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Asfotase AlfaAlso: Strensiq	Biological	Asfotase Alfa

Primary outcome

Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)
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.
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.

Results and safety

Results posted to the registry on 8 May 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Overall Study	13	13

Primary outcome: Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)

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.. Count of participants. Unit: Participants

Measure	Overall Study n = 13
Number of Patients with AEs	9
Number of Patients with ISRs	2
Number of Patients with IARs	0

Population analysed: 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

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.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Overall Study	0 of 13 (0%)	9 of 13 (69.2%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Hypophosphatasia

Enrolling by invitationObservational study
Registry of Patients With Hypophosphatasia
Hypophosphatasia (HPP)
Enrolling by invitationObservational study
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
Osteoporosis
RecruitingObservational study
Clarify HPP: Disease Burden of HPP Patients in China
Hypophosphatasia (HPP)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia
Hypophosphatasia

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Kenji Fujita, MD study director

Medical Monitor

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Asfotase Alfa in people with Hypophosphatasia, run by Alexion Pharmaceuticals, Inc..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 May 2017. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 8 May 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) (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.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02531867.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2017; AzTrials last refreshed its copy on 7 Nov 2026.