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Study on ALXN1850 for Adolescents and Adults with Hypophosphatasia Not Previously Treated with Asfotase Alfa

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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, a type of enzyme therapy, which is given as a solution for injection under the skin. The trial will compare the effects of ALXN1850 to a placebo in adolescents and adults 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 physical function in participants with HPP. Participants will receive either ALXN1850 or a placebo and will be monitored over a period of time to see how their condition changes. The study will involve regular visits to the clinic for assessments and injections, and participants will be asked to perform certain physical tests to measure their progress.

This trial is designed to provide valuable information about the safety and effectiveness of ALXN1850 for people with HPP. By participating, individuals will contribute to research that may help improve treatment options for this condition in the future.

1 joining the study

Upon joining the study, the participant will be randomly assigned to receive either the ALXN1850 medication or a placebo. This process is double-blinded, meaning neither the participant nor the researchers will know which treatment is being administered.

2 medication administration

The medication, ALXN1850, is administered as a subcutaneous injection. This means the solution is injected under the skin. The frequency and dosage will be determined by the study protocol and communicated to the participant at the start of the trial.

3 evaluation period

The participant will undergo a series of assessments to evaluate the effectiveness of the treatment. These assessments include the 6-minute walk test (6MWT), which measures the distance walked in six minutes, and other functional tests such as the 30-second sit-to-stand (STS) test and the Timed Up-and-Go (TUG) test.

4 monitoring and follow-up

Throughout the trial, the participant’s health and response to the treatment will be closely monitored. This includes regular blood tests to measure plasma concentrations of certain substances and to check for any adverse effects.

5 end of randomized evaluation period

At the end of the randomized evaluation period, which is on Day 169, the participant will undergo final assessments to determine any changes from the baseline measurements. These results will help evaluate the efficacy and safety of the ALXN1850 treatment.

Who Can Join the Study?

  • The participant must be at least 12 years old on the first day of the study.
  • The participant must have a diagnosis of Hypophosphatasia (HPP), which should be documented in their medical records.
  • The participant must meet one of the following genetic criteria:
    • Have a documented change in the ALPL gene (which can be harmful, likely harmful, or of unknown significance) from a certified laboratory.
    • Have a PLP level above the normal limit during the screening period, based on laboratory results.
  • The participant must meet one of the following criteria related to ALP activity (a type of enzyme in the blood) without any other likely cause than HPP:
    • Have a serum ALP activity below the normal range for their age and sex during the screening period, as measured by the central laboratory.
    • Have two documented serum ALP activity results, at least 15 days apart, below the normal range for their age and sex in the 24 months before the first day of the study. The laboratory must be certified.
  • The participant must have two separate 6-Minute Walk Tests (6MWTs) showing a distance below 85% of what is expected for their age, sex, weight, and height during the screening period, without any other likely cause than HPP. Participants who need walking aids can be included.
  • Female participants who can have children and male participants must follow specific birth control requirements as outlined in the study protocol.
  • The participant or their legal representative must be able to give signed informed consent, which means they understand and agree to the study’s requirements and restrictions. For adolescents, their legal guardian must provide written consent, and the adolescent must give written agreement if required.
  • The participant must not be willing or able to receive asfotase alfa for any reason, including not being able to follow the injection schedule for this medication.

Who Cannot Join the Study?

  • Participants who have been previously treated with asfotase alfa. This is a medication used to treat a condition called hypophosphatasia, which affects the bones.
  • Participants who are not within the specified age range for the study. The study is for adolescents and adults.
  • Participants who are part of a vulnerable population. This means people who might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Careggi Florence Italy
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Casa Sollievo Della Sofferenza San Giovanni Rotondo Italy
Centre Hospitalier Universitaire De Poitiers Poitiers France
Hospital Del Mar Barcelona Spain
University Hospital Bratislava Bratislava Slovakia
Instytut Centrum Zdrowia Matki Polki Lodz Poland
Wojewodzki Specjalistyczny Szpital Dzieciecy Im Sw Ludwika W Krakowie Cracow Poland
Azienda Ospedaliera di Padova Padua Italy
Hospital Universitario Clínico San Cecilio Granada Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Ospedale San Raffaele S.r.l. Milan Italy
Universita’ Degli Studi Di Verona Verona Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Universita’ Di Pisa Pisa Italy
Hanusch Krankenhaus Der Wiener Gebietskrankenkasse Vienna Austria
Hospital Universitario Araba Vitoria Spain
Rheinische Friedrich-Wilhelms-Universitaet Bonn Bonn Germany
Hospital Universitario De Canarias La Laguna Spain
Hopital Beaujon Clichy France
University Childrens Hospital Queen Fabiola Brussels Belgium
Uqfgozqhvw Mugjjuw Clkzvo Hhhebmsmhqfyjvnxr Hamburg Germany
Hgkzrtzr Uvhzpbzurgwed Mhtnaas Da Vnsmgspgqu Santander Spain
Pfmoks fuh Ijzgom Mpwkzzf Sbefxwc Shctart Bad Reichenhall Germany
Ustdrxlbiqafmwqvvpxyg Wplkdnahc Avg Wuerzburg Germany
Ujyisuagnc Ov Axmuryy Edegem Belgium
Furgthocu Ptaz La Inqnhrhuqwkmj Bwsaltdai Doz Hdqckqzd Ulbivrwlajuyl Ls Pgx Madrid Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
08.04.2024
Belgium Belgium
Not recruiting
08.04.2024
France France
Not recruiting
08.04.2024
Germany Germany
Not recruiting
08.04.2024
Italy Italy
Not recruiting
08.04.2024
Poland Poland
Not recruiting
08.04.2024
Slovakia Slovakia
Not recruiting
08.04.2024
Spain Spain
Not recruiting
08.04.2024

Trial locations

Investigated drugs:

ALXN1850 is a medication being studied for its effectiveness and safety in treating adolescents and adults with a condition called hypophosphatasia (HPP). This medication is a form of recombinant alkaline phosphatase, which is a type of enzyme. The trial aims to see how well ALXN1850 works in improving the physical abilities of people with HPP who have not been treated with another medication called asfotase alfa. ALXN1850 is given as an injection under the skin.

Investigated diseases:

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 frequent fractures and skeletal abnormalities. The severity of symptoms can vary widely, ranging from mild dental issues to severe bone deformities and growth problems. In some cases, muscle weakness and pain may also be present. The progression of the disease can differ significantly among individuals, with some experiencing symptoms from infancy and others not until adulthood.

Trial ID:
2023-505673-32-00
Protocol code:
ALXN1850-HPP-301
Trial Phase:
Therapeutic confirmatory (Phase III)

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