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Monoclonal Antibodies

ALXN1850 for Hypophosphatasia (MULBERRY Trial)

Phase 3

Recruiting

Research Sponsored by Alexion Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of HPP documented in the medical records, and the following criteria fulfilled during the Screening Period without other probable cause than HPP: Presence of HPP-related rickets on skeletal X-rays, with a minimum Rickets Severity Score (RSS) of 1.0 AND Serum ALP activity below the age- and sex-adjusted normal range

Be younger than 18 years old

Must not have

Diagnosis of primary or secondary hyperparathyroidism

History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, neurological disorders, or any other disorders that are capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data as determined by the Investigator

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial tests a new drug to see if it helps children with a rare bone disorder called HPP.

Who is the study for?

This trial is for children with Hypophosphatasia (HPP) who haven't been treated before. They must have a documented ALPL gene variant, high plasma PLP levels, be in early puberty or less, and show signs of HPP-related rickets on X-rays. Kids can't join if they've had recent fractures, allergies to the study drug ingredients, other bone diseases like hyperparathyroidism or hypoparathyroidism (unless due to HPP), or any major health issues that could affect the study.

What is being tested?

The trial tests ALXN1850 against a placebo to see how well it works on improving bone problems seen on X-rays in kids with HPP. It's designed to compare changes between those who get the real medicine and those who get a dummy pill without active ingredients.

What are the potential side effects?

Possible side effects of ALXN1850 are not detailed here but may include reactions related to its ingredients. Since it's being compared with a placebo, some participants might experience no actual medication side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have HPP with rickets shown in X-rays and low ALP levels for my age and sex.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with hyperparathyroidism.

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I do not have any major health issues that could affect how my body handles medication.

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I have hypoparathyroidism not caused by HPP.

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I have not had any new fractures in the last 12 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ALXN1850Experimental Treatment2 Interventions

Starting at Day 1 of the Randomized Evaluation Period, the ALXN1850 group will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once every 2 weeks (q2w) via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Starting at Day 1 during the Randomized Evaluation Period, participants will receive placebo q2w for a total of 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

ALXN1850

2021

Completed Phase 1

~20