Phase 2
N=12
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
Hypophosphatasia
Bottom Line
View on ClinicalTrials.gov: NCT05890794 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Feb 2025
Primary outcome: Primary: Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) — -36.46; -77.31; -56.88 percent change — p=0.0005
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ilofotase Alfa, 0.8 mg/kg (Biological); Ilofotase Alfa, 3.2 mg/kg (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- AM-Pharma
- Primary completion
- Jul 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) |
-36.46; -77.31; -56.88 | 0.0005 sig |
| PRIMARY Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP) |
-34.68; -65.56; -50.12 | <0.0001 sig |
Summary
The goal of this clinical trial is to compare the effectiveness of two doses of ilofotase alfa, an enzyme replacement treatment, in patients with hypophosphatasia (HPP). The main question it aims to answer is if the harmful accumulating levels of extracellular inorganic pyrophosphate (PPi) and pyridoxal 5'-phosphate (PLP) can be reduced with ilofotase alfa.
Researchers will compare the two doses of ilofotase alfa to see if treatment effects differ between the doses.
Eligibility Criteria
Inclusion Criteria
- Genetically confirmed variant in the tissue-nonspecific isozyme alkaline phosphatase (ALPL)-Gene.
- Clinical symptoms of HPP.
- Medical history with 1) at least two independent measures of Alkaline Phosphatase (ALP) below lower level of normal (LLN) and 2) at least one measurement of either PPi or PLP above upper level of normal (ULN).
- Provision of signed and dated informed consent form (ICF) in accordance with local regulations at screening.
- Patients must agree not to get pregnant/not to get their partner pregnant, during the trial. Consequently, patients must agree to use adequate contraception as detailed in study protocol.
Exclusion Criteria
- Participant is unable or unwilling to participate in all scheduled visits and perform all protocol-mandated assessments.
- Has a known or suspected hypersensitivity to ilofotase alfa or any components of the formulation used.
- Body weight 120 kilogram.
- Patient has a history of clinically significant abnormalities or of any illness that, in the opinion of the trial investigator, might confound the results of the trial or pose an additional risk to the patient by their participation in the trial.
- NSAID use in the past 2 weeks.
- Use of corticosteroids in the past 4 weeks.
- Use of compounds intended to interfere with bone metabolism (e.g. Denosumab, Teriparatide, Romosozumab, Raloxifene) in the past 3 months.
- Use of bisphosphonates in the past 2 years.
- Participation in a drug trial within 60 days, or five times the half-life of the drug, whichever is longer, prior to administration of ilofotase alfa.
- Use of asfotase alfa in the previous 3 months. Patients will not be withheld from approved asfotase alfa if medically indicated.
- A patient who is currently pregnant or lactating.
- Use of supplements including Vitamin B6.
Data sourced from ClinicalTrials.gov (NCT05890794). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.