HypophosphatasiaNCT02496689
Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
A study testing asfotase alfa in people with Hypophosphatasia, run by Alexion Pharmaceuticals, Inc. at 5 sites in 2 countries.
Trial summary
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to provide access to treatment in a disease where no approved treatment exists. 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
- How many people
- Not given
- Where
- 5 sitesin 2 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Patients must meet all of the following inclusion criteria for participation in this program:
- Patient or parent (or legal guardian) must provide written informed consent prior to the performance of any program-related procedures and must be willing to comply with program procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained.
- Patient must have a documented diagnosis of HPP as indicated by a documented history of HPP-related skeletal abnormalities and one or more of the following:
- Documented tissue-nonspecific alkaline phosphatase (TNSALP) gene mutation(s)
- Serum alkaline phosphatase (ALP) level below the age-adjusted normal range AND plasma pyridoxal-5'-phosphate (PLP) above the upper limit of normal at Screening. NOTE: Historical results for PLP may be used to determine patient eligibility. The criterion for plasma PLP is not applicable if the patient is receiving pyridoxine treatment.
- Patient must have infantile- or juvenile-onset HPP, defined as documented onset of signs/symptoms of HPP prior to 18 years of age.
Show all 24Show fewer
- Male patient is:
- Prepubertal; OR
- Surgically sterile (defined as vasectomized for ≥6 months at Baseline); OR
- Non-surgically sterile (defined as non-vasectomized or vasectomized for <6 months at Baseline) and his female spouse/partner who is of childbearing potential must be using highly effective contraception consisting of two forms of birth control (at least one of which must be a barrier method) as described below starting at Baseline and continuing for 3 months after program completion.
- Simultaneous use of condom, and for the female partner established use of hormonal contraceptives (eg, oral, injected, implanted) or intra-uterine contraceptive device
- Simultaneous use of condom, and for the female partner occlusive cap (diaphragm or cervical/vault caps) with intravaginally applied spermicide
- Female patient is:
- Of non-childbearing potential, defined as:
- Prepubertal; OR
- Post-menopausal (defined as absence of menses for 12 months prior to Baseline or bilateral oophorectomy or hysterectomy with bilateral oophorectomy at least 6 months prior to Baseline); OR
- Surgically sterile (defined as having hysterectomy or tubal ligation at least 6 months prior to Baseline) OR
- Of childbearing potential, and:
- Not sexually active; OR
- Sexually active with non-sterile male spouse/partner (sterile male spouse/partner defined as a man vasectomized for ≥6 months) and must be using highly effective contraception consisting of two forms of birth control (at least one of which must be a barrier method) as described below and continuing for 3 months after program completion.
- Simultaneous use of hormonal contraceptives (eg, oral, injected, implanted) or intra-uterine contraceptive device since at least 4 weeks prior to Baseline, and condom for the male partner
- Simultaneous use of occlusive cap (diaphragm or cervical/vault caps) with intravaginally applied spermicide, and a condom for the male partner, since at least 14 days prior to Baseline
- Male patients who are pubertal or post-pubertal and not surgically sterile (surgically sterile defined as vasectomized for ≥6 months) must agree to not donate sperm during program participation and for 3 months after program completion.
- Female patients of childbearing potential (ie, those who are not prepubertal or postmenopausal or surgically sterile as defined in inclusion criteria 5 above) must have a negative urine or serum pregnancy test at Screening.
Who cannot join
- Patients will be excluded from participation in this program if they meet one or more of the following exclusion criteria:
- Patient has geographic access to and qualifies for enrollment (as determined by the Sponsor and Investigator) in any other Alexion-sponsored clinical trials of asfotase alfa that are open to enrollment (eg, ENB-010-10).
- Patient is pregnant, trying to become pregnant, or is lactating.
- Patient has a documented sensitivity to any of the components of asfotase alfa.
- Patient has serum calcium and/or phosphorus levels below the normal range at Screening.
- Patient is currently enrolled in any other program or study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation).
- Patient has any other medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with program compliance, including all evaluations and follow-up activities. Patients who previously received treatment with asfotase alfa may be considered for enrollment.
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
France3 sites
Hôpital Bicêtre
Le Kremlin-Bicêtre, 94275
Hôpital Necker - Enfants Malades
Paris, 75015
Hôpital des Enfants
Toulouse, 31259
United States2 sites
Colorado Center for Bone Research
Lakewood, Colorado, 80227
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
About this study
Official title: An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to provide access to treatment in a disease where no approved treatment exists. This is an experimental treatment provided under specific treatment guidelines in which safety endpoints will be collected.
Read the detailed description
U.S. sites participating in the expanded access program are closed to enrollment.
- 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
Patients participating in this program will receive 6 mg/kg/week asfotase alfa (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week at the discretion of the Investigator) by SC injection. During follow-up visits, dose adjustments to account for changes in body weight will be made. Additional incremental dose adjustments for lack of efficacy or safety reasons may also be decided upon by the Investigator in consultation with the Alexion Medical Monitor.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2015 (estimated)
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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Andrew Denker, MD, PhD, Study director, Medical Monitor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02496689
- Sponsor's protocol ID
- AA-HPP-405
- Status last verified
- Mar 2019
- First submitted
- 8 Jul 2015
- Last updated
- 29 Mar 2019
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicenter, Expanded Access Program for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
- ClinicalTrials.gov ID
- NCT02496689
- Acronym / sponsor ID
- AA-HPP-405
- Conditions
- Hypophosphatasia
- Keywords
- HPP, Bone Disease, Soft Bones, Low Alkaline Phosphatase, genetic metabolic disorder, alkaline phosphatase, rickets, osteomalacia
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 14 Jul 2015 (estimated)
- 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 clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to provide access to treatment in a disease where no approved treatment exists. This is an experimental treatment provided under specific treatment guidelines in which safety endpoints will be collected.
Detailed description
U.S. sites participating in the expanded access program are closed to enrollment.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (24 listed)
- Patients must meet all of the following inclusion criteria for participation in this program:
- Patient or parent (or legal guardian) must provide written informed consent prior to the performance of any program-related procedures and must be willing to comply with program procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained.
- Patient must have a documented diagnosis of HPP as indicated by a documented history of HPP-related skeletal abnormalities and one or more of the following:
- Patient must have infantile- or juvenile-onset HPP, defined as documented onset of signs/symptoms of HPP prior to 18 years of age.
- Male patient is:
Key exclusion criteria (7 listed)
- Patients will be excluded from participation in this program if they meet one or more of the following exclusion criteria:
- Patient has geographic access to and qualifies for enrollment (as determined by the Sponsor and Investigator) in any other Alexion-sponsored clinical trials of asfotase alfa that are open to enrollment (eg, ENB-010-10).
- Patient is pregnant, trying to become pregnant, or is lactating.
- Patient has a documented sensitivity to any of the components of asfotase alfa.
- Patient has serum calcium and/or phosphorus levels below the normal range at 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 alfaPatients participating in this program will receive 6 mg/kg/week asfotase alfa (administered at a dosage regimen of 1 mg/kg 6 times per week or 2 mg/kg 3 times per week at the discretion of the Investigator) by SC injection. During follow-up visits, dose adjustments to account for changes in body weight will be made. Additional incremental dose adjustments for lack of efficacy or safety reasons may also be decided upon by the Investigator in consultation with the Alexion Medical Monitor. | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2019.
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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.
Andrew Denker, MD, PhD study director
Medical Monitor
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing asfotase alfa in people with Hypophosphatasia, run by Alexion Pharmaceuticals, Inc. at 5 sites in 2 countries.
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 24 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: status not confirmed, as of the sponsor's last update on 29 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in France, 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?
Results are not available in the source record.
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.
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 29 Mar 2019; AzTrials last refreshed its copy on 30 Oct 2026.