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Enzyme Replacement Therapy

ELAPRASE + Prophylactic Therapy for Hunter Syndrome

Phase 4

Recruiting

Research Sponsored by Takeda

Eligibility Criteria Checklist

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

Must have

Participant must have a documented diagnosis of MPS II

Participant has a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of <= 10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes

Must not have

Participant has current or relevant history of physical or psychiatric illness, or any medical disorder that may require treatment or make the participant unlikely to fully complete the study, or any condition that presents undue risk from the investigational product or procedures

Participant has received or is receiving treatment with idursulfase-IT

Timeline

Screening 3 weeks

Treatment Varies

Follow Up every 6 months up to 24 months

Awards & highlights

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial investigates if a prophylactic therapy can prevent or reduce high titer antibodies in Hunter syndrome patients receiving ELAPRASE for up to 104 weeks.

Who is the study for?

This trial is for treatment-naïve boys under 6 with Hunter syndrome, having specific enzyme deficiencies and gene mutations. They must not have used ELAPRASE or certain other treatments recently and should be able to follow the study protocol without any medical conditions that could interfere.

What is being tested?

The study tests if a preventive immune regimen can stop or lessen high titer anti-idursulfase antibodies in patients taking ELAPRASE. It includes Rituximab, Methotrexate, IVIG alongside ELAPRASE over up to 104 weeks with assessments every six months.

What are the potential side effects?

Possible side effects include reactions related to the immune system such as infusion reactions from Rituximab or IVIG, liver issues due to Methotrexate, and general symptoms like fever, chills, nausea which may vary among individuals.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with MPS II.

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My enzyme (I2S) activity is very low, below 10% of the normal range.

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I have a severe IDS gene mutation linked to a strong immune response to enzyme therapy.

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I am under 6 years old.

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I am male.

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I have never taken ELAPRASE before.

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 health conditions that could interfere with the study or pose a risk.

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I am being treated with idursulfase-IT.

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I have received growth hormones, a cord blood infusion, or a bone marrow transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ every 6 months up to 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~every 6 months up to 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and every 6 months up to 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Rate of Anti-Idursulfase Antibodies Formation, Including Anti-Idursulfase Antibodies That Have Enzyme Neutralizing Activity

Secondary study objectives

Change From Baseline in Liver Volume

Change From Baseline in Normalized uGAG per Upper Limit of Normal for age (uGAG)/ULN)

Glycosaminoglycans

+1 more

Awards & Highlights

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: ITR + ELAPRASEExperimental Treatment4 Interventions

Participants will receive prophylactic ITR which consist of rituximab, methotrexate and IVIG in a 5-week cycle. Following the completion of 1 cycle and at the Month 6, 12, and 18 study visits, an assessment will be made regarding the need for administering another 5-week cycle of the ITR depending on the trend of the participants anti-idursulfase antibody titers and lymphocyte quantitation and CD19 percent (%) recovery. Elaprase treatment (IV, weekly) will start 1 day after the initiation of the first cycle of ITR and continue for 104 weeks. The dose of ELAPRASE will be calculated based on the participant's weight at each visit.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rituximab

1999

Completed Phase 4

~2990

Methotrexate

2019

Completed Phase 4

~4400