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 range18 Years – 85 Years
SexALL
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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…
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)