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Pilot trial of single dose ilofotase alfa in hypophosphatasia
EU CTIS ID: 2023-503186-35-00
What this study is testing
The change in Inorganic Pyrophosphate (PPi), and Pyridoxal 5’-phosphate (PLP) levels after low, and high dose ilofotase alfa in adult hypophosphatasia (HPP) patients.
- Human Pharmacology (Phase I)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 18-85 years old, inclusive.
- 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 to use adequate double-barrier contraception when sexually active. This applies for the period between trial drug administration and 14 days thereafter.
You likely can't join if
- Is unable or unwilling to participate in all scheduled visits and perform all protocol-mandated assessments.
- Use of asfotase alfa in the previous 3 months.
- A patient who is currently pregnant or lactating.
- Use of supplements including Vitamin B6.
- Has a known or suspected hypersensitivity to ilofotase alfa or any components of the formulation used.
- Body weight < 40 kilogram and > 120 kilogram.
See the full eligibility criteria
- 18-85 years old, inclusive.
- 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 to use adequate double-barrier contraception when sexually active. This applies for the period between trial drug administration and 14 days thereafter.
- Is unable or unwilling to participate in all scheduled visits and perform all protocol-mandated assessments.
- Use of asfotase alfa in the previous 3 months.
- A patient who is currently pregnant or lactating.
- Use of supplements including Vitamin B6.
- Has a known or suspected hypersensitivity to ilofotase alfa or any components of the formulation used.
- Body weight < 40 kilogram and > 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 prior to administration of ilofotase alfa.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.