Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia (NCT05890794) | Clinical Trial Compass
CompletedPhase 1/2
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
Germany12 participantsStarted 2023-05-15
Plain-language 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.
Who can participate
Age range
18 Years – 85 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 \< 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 dru…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi)
Timeframe: Day 1 to Day 10
2
Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP)