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HypophosphatasiaPhase 1/2NCT05890794

Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia

A Phase 1/2 study testing Ilofotase Alfa, 0.8 mg/kg and Ilofotase Alfa, 3.2 mg/kg in people with Hypophosphatasia, run by AM-Pharma at 1 site in Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 to 85 years
Conditions the study is for
Hypophosphatasia
What is being tested?
Ilofotase Alfa, 0.8 mg/kg and Ilofotase Alfa, 3.2 mg/kg
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteGermany
Study dates
15 May 2023to 12 Jul 2023
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 12Show fewer
  • 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.

Trial locations and contacts

1 site in 1 country.

Germany1 site

  • Osteologie / Klinische Studieneinheit, Orthopädische Klinik - KLH

    Würzburg, 97074

Other trials in Germany

About this study

Official title: Open-Label Pilot Trial to Evaluate the Effects of Ilofotase Alfa on Biomarkers in Adult Patients With Hypophosphatasia

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.

Read the detailed description

Recombinant human alkaline phosphatase (ilofotase alfa) is a full-length human chimeric alkaline phosphatase (ALP) that could benefit patients with hypophosphatasia (HPP), which is characterized by low activity of tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP).

This is a pilot trial for a potential future trial aimed at identifying whether treatment with ilofotase alfa can normalize circulating levels of PPi, PLP and other biochemical markers of TNSALP deficiency along with the safety/tolerability of different doses of ilofotase alfa. The trial is designed as a single-center, open-label, randomized, parallel group clinical trial in adult patients with HPP. Two different dose levels (0.8 mg/kg and 3.2 mg/kg) of ilofotase alfa will be assessed.

Participants will receive a single dose of ilofotase alfa, administered as a 1-hour intravenous infusion on Study Day 1. Participants will stay at the research center for a total of 12 days; from 2 days before study drug administration (run-in) to 10 days after treatment. An additional follow-up assessment is scheduled 14 days after administration of ilofotase alfa.

Blood and urine samples will be taken daily for drug concentration and laboratory measurements assessing safety and effectiveness of treatment. In addition physical examinations will be performed on Day 1 and as needed afterwards.

Being tested
Ilofotase Alfa, 0.8 mg/kg, Ilofotase Alfa, 3.2 mg/kg
Keywords
Enzyme replacement therapy
Medical subject headings
Hypophosphatasia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ilofotase Alfa, 0.8 mg/kgBiological

    Also called recAP

    Biological: single 1-hour intravenous infusion of 0.8 mg/kg ilofotase alfa

  • Ilofotase Alfa, 3.2 mg/kgBiological

    Also called recAP

    Biological: single 1-hour intravenous infusion of 3.2 mg/kg ilofotase alfa

Study groups in NCT05890794
Study groupRoleWhat they receive
0.8 mg/kg ilofotase alfaExperimentalSingle dose administered intravenously over 1 hour
Receives: Biological: Ilofotase Alfa, 0.8 mg/kg
3.2 mg/kg ilofotase alfaExperimentalSingle dose administered intravenously over 1 hour
Receives: Biological: Ilofotase Alfa, 3.2 mg/kg

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi)

    Percent Change from BaseLine (PCBL) in PPi serum concentration is calculated for all post-dose PPi measures recorded from Day 1 to Day 10. Per patient, 12 measurements were done: 3 at Day 1 and 1 each day from Day 2 to Day 10. The Percent Change from BaseLine for measurement x (x=1,…12) is calculated: PCBL(x)= [(PPi( x)-PPi(baseline))/PPi(baseline)]*100. The subject's maximum percent change from baseline is defined as the Max (PCBL(1), …, PCBL(12)).

    Measured: Day 1 to Day 10

  2. Maximum Percent Change From Baseline in Pyridoxal 5'-Phosphate (PLP)

    Percent Change from BaseLine (PCBL) in PLP serum concentration is calculated for all post-dose PLP measures recorded from Day 1 to Day 10. Per patient, 12 measurements were done: 3 at Day 1 and 1 each day from Day 2 to Day 10. The Percent Change from BaseLine for measurement x (x=1,…12) is calculated: PCBL(x)= [(PLP( x)-PLP(baseline))/PLP(baseline)]*100. The subject's maximum percent change from baseline is defined as the Max (PCBL(1), …, PCBL(12)).

    Measured: Day 1 to Day 10

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Jun 2023
  2. Study start15 May 2023
  3. Primary completion7 Jul 2023
  4. Study completion12 Jul 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
2 doses will be compared
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05890794
Sponsor's protocol ID
AP-recAP-HPP-01-01
Status last verified
Feb 2025
First submitted
10 May 2023
Last updated
27 Feb 2025
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Seefried L, Bernholz J, Kraan M, Nitschke Y, Rutsch F, Mallek M, Kleinert S, Genest F. A randomized Phase 1b trial evaluating the pharmacodynamics of ilofotase alfa in adults with hypophosphatasia. J Bone Miner Res. 2026 Mar 2;41(3):231-241. doi: 10.1093/jbmr/zjaf185.

    PubMed 41378916

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Ilofotase Alfa, 0.8 mg/kg and Ilofotase Alfa, 3.2 mg/kg in people with Hypophosphatasia, run by AM-Pharma at 1 site in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 27 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Percent Change From Baseline in Extracellular Inorganic Pyrophosphate (PPi) (time frame: Day 1 to Day 10). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05890794, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2025.

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