Study of ALXN1850 for Children Aged 2 to 12 with Hypophosphatasia Who Have Not Previously Received Asfotase Alfa Treatment

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What is this study about?

This clinical trial is focused on studying a rare condition called hypophosphatasia (HPP), which affects the development of bones and teeth. The study will evaluate a treatment known as ALXN1850, which is given as a solution for injection under the skin. The trial will compare the effects of ALXN1850 with a placebo in children aged 2 to less than 12 years who have not previously been treated with another medication called asfotase alfa.

The purpose of the study is to assess how well ALXN1850 works in improving bone health in children with HPP. Participants will receive either ALXN1850 or a placebo through subcutaneous injections. The study will last for several months, during which the children will have regular check-ups to monitor their progress and any changes in their condition. The main focus will be on how the treatment affects the bones, as seen in X-ray images, and other health measures.

Throughout the study, the safety and effectiveness of ALXN1850 will be closely monitored. The trial aims to provide valuable information on whether ALXN1850 can be a beneficial treatment option for children with hypophosphatasia, potentially improving their quality of life by addressing the symptoms of this condition.

1 joining the study

Upon joining the study, the participant must be between 2 and 12 years old and have a documented diagnosis of hypophosphatasia (HPP).

The participant’s legal guardian must provide written consent, and the participant must provide assent if applicable.

2 screening period

During this period, the participant will undergo various assessments to confirm eligibility, including skeletal X-rays to check for HPP-related rickets and blood tests to measure serum alkaline phosphatase (ALP) activity.

The participant must meet specific genetic or biochemical criteria related to HPP.

3 randomized evaluation period

Participants will be randomly assigned to receive either the ALXN1850 solution for injection or a placebo. The medication is administered via subcutaneous injection.

The treatment will continue until Day 169, with regular assessments to monitor the participant’s response to the treatment.

4 primary and secondary assessments

The primary assessment will focus on the RGI-C score at the end of the evaluation period.

Secondary assessments will include changes in the Rickets Severity Score (RSS), physical performance tests, and quality of life measures.

5 safety monitoring

Throughout the study, the participant will be monitored for any adverse effects related to the treatment.

Regular blood tests and other evaluations will be conducted to ensure the participant’s safety.

6 end of study

The study is expected to conclude by December 31, 2027.

Final assessments will be conducted to evaluate the overall efficacy and safety of the treatment.

Who Can Join the Study?

The participant must be at least 2 years old but younger than 12 years old on the first day of the study.
The participant must have a diagnosis of hypophosphatasia (HPP), which is a condition affecting bone development, documented in their medical records.
The participant must show signs of rickets, a bone condition, related to HPP on X-rays taken during the screening period, with a minimum Rickets Severity Score (RSS) of 1.0.
The participant must have low levels of serum ALP (alkaline phosphatase), an enzyme in the blood, below the normal range for their age and sex during the screening period, as measured by a certified laboratory.
The participant must meet one of the following criteria:
- Have a documented change in the ALPL gene, which is related to HPP, from a certified laboratory.
- Have levels of PLP (pyridoxal 5′-phosphate), a form of vitamin B6, above the normal limit during the screening period.
The participant must be at Tanner stage 2 or less during the screening period. Tanner stages are used to describe physical development in children.
Female participants who can have children and male participants must follow specific birth control guidelines as defined in the study protocol.
The participant’s legal guardian must be willing and able to provide written informed consent, and the participant must be willing to give written informed assent if applicable. This means agreeing to follow the study’s rules and requirements.

Who Cannot Join the Study?

Participants who have been previously treated with a medication called asfotase alfa cannot join the study. This medication is used to treat a condition called hypophosphatasia.
Participants who are not within the specified age range for the study cannot participate. The study is for children, so adults cannot join.
Participants who have any other medical conditions that might interfere with the study or its results cannot take part.
Participants who are unable to follow the study procedures or instructions cannot be included.
Participants who are part of another clinical trial at the same time cannot join this study.

Where you can join this trial?

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Other Sites

Site Name	City	Country
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Instytut Centrum Zdrowia Matki Polki	Lodz	Poland
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Wojewodzki Specjalistyczny Szpital Dzieciecy Im Sw Ludwika W Krakowie	Cracow	Poland
University Childrens Hospital Queen Fabiola	Brussels	Belgium
National Institute Of Endocrinology C.I. Parhon	Bucharest	Romania
Ospedale San Raffaele S.r.l.	Milan	Italy
Karolinska University Hospital	Solna	Sweden
Hospital Universitario Araba	Vitoria	Spain
Hopital Beaujon	Clichy	France
Hpjtjjts Ufhnecsypd Clwtfct Hlafqvto	Helsinki	Finland
Fgspxiihy Pwgj Lx Iluuzcuzluvmt Brotdbpkf Dua Hlaydist Ucwczcmumsrcp Lt Pks	Madrid	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	15.03.2024
Finland	Not recruiting	15.03.2024
France	Not yet recruiting	15.03.2024
Italy	Not yet recruiting	15.03.2024
Poland	Not recruiting	15.03.2024
Romania	Not recruiting	15.03.2024
Spain	Not recruiting	15.03.2024
Sweden	Not recruiting	15.03.2024

Trial locations

Investigated drugs:

Alxn1850

ALXN1850 is a medication being studied for its effectiveness in treating children with hypophosphatasia, a rare bone disorder. This medication is given by injection under the skin and is being compared to a placebo to see how well it works in improving bone health in children who have not been treated with another medication called asfotase alfa.

Hypophosphatasia – Hypophosphatasia is a rare genetic disorder that affects the development of bones and teeth. It is caused by mutations in the gene responsible for producing an enzyme called alkaline phosphatase, which is crucial for bone mineralization. As a result, individuals with this condition may experience soft and weak bones, leading to skeletal abnormalities and an increased risk of fractures. The severity of symptoms can vary widely, ranging from mild dental issues to severe bone deformities and growth problems. In some cases, hypophosphatasia can also affect the respiratory system and muscle strength. The progression of the disease depends on the specific genetic mutation and the age at which symptoms first appear.

Trial ID:

2023-505675-73-00

Protocol code:

ALXN1850-HPP-305

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of ALXN1850 for Children Aged 2 to 12 with Hypophosphatasia Who Have Not Previously Received Asfotase Alfa Treatment

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?