HypophosphatasiaPhase 4REBUILDNCT04189315
Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease
A Phase 4 study testing asfotase alfa in people with Hypophosphatasia, run by Alexion Pharmaceuticals, Inc..
Trial summary
The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP). Read more about this study
- Who is it for?
- Ages 18 years and older
- 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
- 0 participants
- Where
- No sites listed yet
- Study dates
- Apr 2020to Aug 2022
- Phase
- Phase 4
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female aged ≥ 18 years at the time of signing the informed consent form
- Clinical diagnosis of pediatric-onset HPP based on signs and symptoms consistent with HPP
- Past medical history that includes at least one nonvertebral fracture (or pseudofracture) incurred without evidence of significant trauma.
- The presence of a current fracture is not necessary, but for participants with current unhealed fracture(s) or pseudofracture(s) of the lower extremity(ies) (that is, femoral, tibial, fibular, metatarsal) documentation must be provided of the presence of these fractures for at least 3 months prior to screening (with or without surgical intervention)
Who cannot join
- Medical condition, serious concurrent or recurrent illness and/or injury, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance or the assessment of study endpoints, including all protocol required evaluations and follow up activities, or may put the patient at risk
- Primary or secondary hyperparathyroidism or hypoparathyroidism
- History of hypersensitivity to any ingredient contained in asfotase alfa
- Oral bisphosphonate use within 6 to 12 months (depending on the half-life of the drug as assessed by the investigator) and intravenous (IV) bisphosphonate use within 12 months prior to screening
- Denosumab use within 18 months prior to screening
- Asfotase alfa use within 6 months prior to screening
Show all 11Show fewer
- Teriparatide/parathyroid hormone analog use within 2 months prior to screening
- Treatment with strontium or sclerostin inhibitors within 6 months prior to the first dose of study drug
- Vitamin B6 use for at least 2 weeks prior to screening
- Serum 25-hydroxy (25-OH) vitamin D below 20 nanogram (ng)/milliliter (mL), with repletion and recheck allowed at screening (results from local laboratory may be used if within 4 weeks of screening)
- Female patients who are pregnant, planning to become pregnant, or breastfeeding
About this study
Official title: A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia
The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
- Conditions
- Hypophosphatasia
- Being tested
- asfotase alfa
- Keywords
- Hypophosphatasia, HPP, Pediatric-Onset, Asfotase Alfa
- 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
Asfotase alfa is administered subcutaneously.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 Asfotase Alfa | Experimental | Participants will be administered asfotase alfa per approved dose for 36 weeks. Receives: Drug: asfotase alfa |
| Group 2 Asfotase Alfa | Experimental | Participants will be administered asfotase alfa per approved dose for 12 weeks and then asfotase alfa at a lower dose for 24 weeks. Receives: Drug: asfotase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1
Measured: Baseline, Week 36
Secondary outcomes (3)
Change From Baseline To Week 36 In Plasma Concentrations Of PPi In Group 2
Measured: Baseline, Week 36
Change From Baseline To Week 36 In The 36-item Short-Form Survey (SF-36) Physical Component Summary (PCS) Score In Groups 1 And 2
Measured: Baseline, Week 36
Change From Baseline To Week 36 In The Repeated Chair Stand Test (A Component Of The Short Physical Performance Battery [SPPB]) In Groups 1 And 2
Measured: Baseline, Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2019
- Study startApr 2020 (estimated)
- Primary completionAug 2022 (estimated)
- Study completionAug 2022 (estimated)
Why it stopped: R\&D portfolio review and in light of the global impact of the COVID-19 pandemic
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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04189315
- Sponsor's protocol ID
- AA-HPP-406
- Acronym
- REBUILD
- Status last verified
- Jun 2020
- First submitted
- 4 Dec 2019
- Last updated
- 4 Jun 2020
- 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 4 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia
- ClinicalTrials.gov ID
- NCT04189315
- Acronym / sponsor ID
- REBUILD / AA-HPP-406
- Conditions
- Hypophosphatasia
- Keywords
- Hypophosphatasia, HPP, Pediatric-Onset, Asfotase Alfa
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: R\&D portfolio review and in light of the global impact of the COVID-19 pandemic
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2020 (estimated)
- Primary completion
- Aug 2022 (estimated)
- Study completion
- Aug 2022 (estimated)
- First posted
- 6 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.
The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 Asfotase Alfa | Experimental | Drug: asfotase alfaParticipants will be administered asfotase alfa per approved dose for 36 weeks. |
| Group 2 Asfotase Alfa | Experimental | Drug: asfotase alfaParticipants will be administered asfotase alfa per approved dose for 12 weeks and then asfotase alfa at a lower dose for 24 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female aged ≥ 18 years at the time of signing the informed consent form
- Clinical diagnosis of pediatric-onset HPP based on signs and symptoms consistent with HPP
- Past medical history that includes at least one nonvertebral fracture (or pseudofracture) incurred without evidence of significant trauma.
- The presence of a current fracture is not necessary, but for participants with current unhealed fracture(s) or pseudofracture(s) of the lower extremity(ies) (that is, femoral, tibial, fibular, metatarsal) documentation must be provided of the presence of these fractures for at least 3 months prior to screening (with or without surgical intervention)
Key exclusion criteria (11 listed)
- Medical condition, serious concurrent or recurrent illness and/or injury, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance or the assessment of study endpoints, including all protocol required evaluations and follow up activities, or may put the patient at risk
- Primary or secondary hyperparathyroidism or hypoparathyroidism
- History of hypersensitivity to any ingredient contained in asfotase alfa
- Oral bisphosphonate use within 6 to 12 months (depending on the half-life of the drug as assessed by the investigator) and intravenous (IV) bisphosphonate use within 12 months prior to screening
- Denosumab use within 18 months prior to screening
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 alfaAsfotase alfa is administered subcutaneously. | Drug | Group 1 Asfotase Alfa, Group 2 Asfotase Alfa |
Primary outcome
Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1
Time frame: Baseline, Week 36
3 secondary outcomes
Change From Baseline To Week 36 In Plasma Concentrations Of PPi In Group 2
Time frame: Baseline, Week 36
Change From Baseline To Week 36 In The 36-item Short-Form Survey (SF-36) Physical Component Summary (PCS) Score In Groups 1 And 2
Time frame: Baseline, Week 36
Change From Baseline To Week 36 In The Repeated Chair Stand Test (A Component Of The Short Physical Performance Battery [SPPB]) In Groups 1 And 2
Time frame: Baseline, Week 36
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2020.
- Individual participant dataSharing planned: No
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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.
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 4 inclusion and 11 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: withdrawn, as of the sponsor's last update on 4 Jun 2020. 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?
The study was withdrawn before enrolling participants, so there are no results.
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 To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1 (time frame: Baseline, Week 36). 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 4 Jun 2020.