Hypophosphatasia Clinical Trial
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)
NCT number | NCT02496689 |
Other study ID # | AA-HPP-405 |
Secondary ID | |
Status | Approved for marketing |
Phase | |
First received | |
Last updated |
Verified date | March 2019 |
Source | Alexion Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Expanded Access |
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.
Status | Approved for marketing |
Enrollment | 0 |
Est. completion date | |
Est. primary completion date | |
Accepts healthy volunteers | |
Gender | All |
Age group | N/A and older |
Eligibility |
Inclusion Criteria Patients must meet all of the following inclusion criteria for participation in this program: 1. 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. 2. 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. 3. Patient must have infantile- or juvenile-onset HPP, defined as documented onset of signs/symptoms of HPP prior to 18 years of age. 4. 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 5. 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 6. 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. 7. 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. Exclusion Criteria Patients will be excluded from participation in this program if they meet one or more of the following exclusion criteria: 1. 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). 2. Patient is pregnant, trying to become pregnant, or is lactating. 3. Patient has a documented sensitivity to any of the components of asfotase alfa. 4. Patient has serum calcium and/or phosphorus levels below the normal range at Screening. 5. Patient is currently enrolled in any other program or study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation). 6. 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. |
Country | Name | City | State |
---|---|---|---|
France | Hôpital Bicêtre | Le Kremlin-Bicêtre | |
France | Hôpital Necker - Enfants Malades | Paris | |
France | Hôpital des Enfants | Toulouse Cedex 9 | |
United States | Colorado Center for Bone Research | Lakewood | Colorado |
United States | Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals |
United States, France,
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT03418389 -
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
|
||
Recruiting |
NCT02237625 -
Natural History Study of Patients With Hypophosphatasia (HPP)
|
||
Completed |
NCT02291497 -
Burden of Disease in Hypophosphatasia (HPP)
|
N/A | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Active, not recruiting |
NCT04195763 -
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
|
||
Not yet recruiting |
NCT05596539 -
Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
|
||
Completed |
NCT02796885 -
Characterisation of Adult-Onset Hypophosphatasia
|
||
Completed |
NCT02751801 -
Health Burden of Hypophosphatasia
|
||
Completed |
NCT05890794 -
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
|
Phase 1/Phase 2 | |
Recruiting |
NCT06079359 -
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
|
Phase 3 | |
Recruiting |
NCT05234567 -
A Prospective Sub-Study of the Global Hypophosphatasia Registry
|
||
Completed |
NCT02797821 -
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
|
Phase 2 | |
Completed |
NCT01163149 -
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
|
Phase 2 | |
Completed |
NCT04925804 -
Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)
|
||
Completed |
NCT02531867 -
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
|
Phase 4 | |
Completed |
NCT01406977 -
Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)
|
Phase 2 | |
Recruiting |
NCT01793168 -
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
|
||
Completed |
NCT01176266 -
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
|
Phase 2/Phase 3 | |
Withdrawn |
NCT00894075 -
Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
|
Phase 2 | |
Active, not recruiting |
NCT04222452 -
The PORTRAIT Study
|