What side effects are associated with this type of intervention?

Enzyme replacement therapies (ERT) for Hypophosphatasia, such as those targeting alkaline phosphatase deficiency, commonly include side effects like injection site reactions (redness, pain, swelling), hypersensitivity reactions (rash, fever, chills), and potential antibody formation against the enzyme. Other reported side effects may include headache, fatigue, and gastrointestinal symptoms such as nausea and diarrhea. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

The FDA has approved asfotase alfa (Strensiq) for the treatment of Hypophosphatasia (HPP), a rare metabolic disorder characterized by defective bone mineralization due to alkaline phosphatase deficiency. Asfotase alfa is an enzyme replacement therapy that targets the underlying enzyme deficiency in HPP, improving bone mineralization and overall patient outcomes. This treatment is similar to the investigational drug ALXN1850, which is also an enzyme replacement therapy aimed at addressing alkaline phosphatase deficiency in HPP patients.

What other types of research exist?

Currently, there are no specific alternatives to ALXN1850 mentioned in the provided context for Hypophosphatasia. However, patients may consider discussing with their healthcare provider the potential of other enzyme replacement therapies or investigational drugs targeting similar metabolic pathways. Additionally, therapies used in related bone disorders, such as those targeting bone mineral density or metabolic bone diseases, might offer some insights into alternative treatment strategies.

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Monoclonal Antibodies

ALXN1850 for Hypophosphatasia (HICKORY 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

Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (Section 8.7 )

Must not have

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

Hypoparathyroidism, unless secondary to HPP

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new treatment called ALXN1850 to see if it helps adolescents and adults with HPP improve their ability to perform daily activities. The participants have not been treated with another common medication for HPP.

Who is the study for?

This trial is for adolescents and adults with Hypophosphatasia (HPP) who have a documented ALPL gene variant, low serum alkaline phosphatase activity without other causes than HPP, high plasma PLP levels, and reduced walking capacity. They must not have been treated with asfotase alfa before. People with hypoparathyroidism unrelated to HPP, recent fractures or surgeries that could affect the study, allergies to ingredients in the treatment or placebo, or significant health issues affecting drug absorption or posing risks are excluded.

What is being tested?

The trial is testing the effectiveness of a new medication called ALXN1850 compared to a placebo on improving physical function in participants with HPP. It's designed to see if those taking ALXN1850 perform better on functional tests like walking distance than those taking an inactive substance.

What are the potential side effects?

While specific side effects for ALXN1850 aren't listed here, common side effects from similar treatments may include allergic reactions to ingredients, potential impact on organ functions due to genetic differences in metabolism of drugs, and general discomforts such as headaches or nausea.

Eligibility Criteria

Inclusion Criteria

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

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My medical records show a diagnosis of HPP.

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My test shows an ALPL gene variant from a certified lab.

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I walked less than 85% of the expected distance for someone my age, sex, weight, and height due to HPP.

Exclusion Criteria

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

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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 been diagnosed with hyperparathyroidism.

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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: ALXN1850 GroupExperimental 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 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: Placebo GroupPlacebo Group1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ALXN1850

2021

Completed Phase 1

~20

Placebo

1995

Completed Phase 3

~2670

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Enzyme replacement therapy (ERT) for Hypophosphatasia (HPP) involves the administration of a synthetic form of the enzyme alkaline phosphatase, which patients with HPP lack or have in insufficient amounts. This enzyme is crucial for the proper mineralization of bones and teeth. By supplementing the deficient enzyme, ERT helps to reduce the accumulation of substrates that interfere with bone mineralization, thereby improving bone strength and reducing the risk of fractures and other skeletal abnormalities. This treatment is significant for HPP patients as it directly addresses the underlying cause of their condition, offering a targeted approach to manage symptoms and improve quality of life.

Therapeutic thoroughfares for adults living with Pompe disease.Substrate Deprivation Therapy to Reduce Glycosaminoglycan Synthesis Improves Aspects of Neurological and Skeletal Pathology in MPS I Mice.Acid sphingomyelinase-ceramide system in steatohepatitis: a novel target regulating multiple pathways.