HypophosphatasiaNCT04195763
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
An observational study of Hypophosphatasia, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Hypophosphatasia
- What is being tested?
- asfotase alfa
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Nov 2019to 12 Apr 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Male or female participants ≥18 years of age who have been diagnosed with pediatric-onset HPP, have been newly prescribed treatment with asfotase alfa, and are registered in the patient support program managed by OneSource will be invited to participate.
Who may be able to join
- ≥ 18 years old
- Clinical diagnosis of pediatric-onset HPP
- Naïve to asfotase alfa
- Expected to begin treatment with asfotase alfa for HPP
- Registered in OneSource
- Willing and able to provide voluntary, verbal informed consent to participate in this study
Who cannot join
- Pregnant or breastfeeding
- Unable to speak and understand English
- Unable or unwilling to complete the study surveys via telephone interview at the protocol-required time points
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™.
Read the detailed description
Participants consenting to participate in this study will be asked to complete study questionnaires by phone interviews upon consenting (Baseline), and up to 12 months following treatment initiation with asfotase alfa. Demographic and clinical characteristics of participants in the study will be characterized.
This is an observational study and no intervention will be administered. Participants will be treated in accordance with standard of care.
- Conditions
- Hypophosphatasia
- Being tested
- asfotase alfa
- Keywords
- Pediatric-onset hypophosphatasia, Pediatric-onset, Hypophosphatasia, HPP, Patient reported outcome
- Medical subject headings
- Hypophosphatasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
asfotase alfaDrug
Also called Strensiq®
This is an observational study and no intervention will be administered. All participants will be treated by their physician in accordance with standard of care. All medications are commercially available and will be used as directed by the treating physician.
| Study group | Role | What they receive |
|---|---|---|
| Participants with Pediatric-onset HPP | Adult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource. Receives: Drug: asfotase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores
Measured: Baseline, up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2019
- Study start6 Nov 2019
- Primary completion12 Apr 2024
- Study completion12 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Xcenda, LLC
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04195763
- Sponsor's protocol ID
- ALX-HPP-503
- Status last verified
- Jul 2024
- First submitted
- 10 Dec 2019
- Last updated
- 10 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia
- ClinicalTrials.gov ID
- NCT04195763
- Acronym / sponsor ID
- ALX-HPP-503
- Conditions
- Hypophosphatasia
- Keywords
- Pediatric-onset hypophosphatasia, Pediatric-onset, Hypophosphatasia, HPP, Patient reported outcome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Xcenda, LLC
- Study start
- 6 Nov 2019 (actual)
- Primary completion
- 12 Apr 2024 (actual)
- Study completion
- 12 Apr 2024 (actual)
- First posted
- 12 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™.
Detailed description
Participants consenting to participate in this study will be asked to complete study questionnaires by phone interviews upon consenting (Baseline), and up to 12 months following treatment initiation with asfotase alfa. Demographic and clinical characteristics of participants in the study will be characterized.
This is an observational study and no intervention will be administered. Participants will be treated in accordance with standard of care.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with Pediatric-onset HPP | – | Drug: asfotase alfaAdult participants diagnosed with pediatric-onset HPP, newly prescribed treatment with asfotase alfa, and registered in the patient support program managed by OneSource. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Male or female participants ≥18 years of age who have been diagnosed with pediatric-onset HPP, have been newly prescribed treatment with asfotase alfa, and are registered in the patient support program managed by OneSource will be invited to participate.
Key inclusion criteria (6 listed)
- ≥ 18 years old
- Clinical diagnosis of pediatric-onset HPP
- Naïve to asfotase alfa
- Expected to begin treatment with asfotase alfa for HPP
- Registered in OneSource
Key exclusion criteria (3 listed)
- Pregnant or breastfeeding
- Unable to speak and understand English
- Unable or unwilling to complete the study surveys via telephone interview at the protocol-required time points
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®This is an observational study and no intervention will be administered. All participants will be treated by their physician in accordance with standard of care. All medications are commercially available and will be used as directed by the treating physician. | Drug | Participants with Pediatric-onset HPP |
Primary outcome
Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores
Time frame: Baseline, up to 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2024.
- Individual participant dataSharing planned: No
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypophosphatasia, run by Alexion Pharmaceuticals, Inc. at 1 site in United States.
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 6 inclusion and 3 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 10 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores (time frame: Baseline, up to 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 10 Jul 2024.