97 Participants Needed

Pegunigalsidase Alfa for Fabry Disease

Recruiting at 32 trial locations
Age: 18 - 65
Sex: Any
Trial Phase: Phase 3
Sponsor: Chiesi Farmaceutici S.p.A.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

What data supports the effectiveness of the drug pegunigalsidase alfa for Fabry disease?

Research shows that pegunigalsidase alfa, a new enzyme replacement therapy, helps maintain kidney function and reduces harmful substances in the body for people with Fabry disease. It also has a longer-lasting effect in the bloodstream and causes fewer immune reactions compared to similar treatments.12345

Is pegunigalsidase alfa safe for humans?

Pegunigalsidase alfa has been studied for safety in people with Fabry disease, and most side effects were mild or moderate. In a long-term study, one person had a serious side effect, but overall, the treatment was generally well-tolerated.12345

How is the drug pegunigalsidase alfa different from other treatments for Fabry disease?

Pegunigalsidase alfa is unique because it is a PEGylated form of enzyme replacement therapy, which means it has a longer half-life in the body and is less likely to cause immune reactions compared to other treatments. This can lead to more stable and effective management of Fabry disease symptoms.12345

What is the purpose of this trial?

The objective of CLI-06657AA1-04 (formerly PB-102-F60) is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.

Eligibility Criteria

This trial is for adult patients with Fabry Disease who completed previous studies (PB-102-F20, PB-102-F03, or PB-102-F30) and consent to participate. They must use effective contraception if they can have children. Those with conditions affecting study compliance are excluded.

Inclusion Criteria

I agree to use effective birth control during and for 2 weeks after the treatment.
You have already taken part in studies PB-102-F20, PB-102-F03, or PB-102-F30.
The patient signs informed consent

Exclusion Criteria

Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 1 mg/kg pegunigalsidase alfa intravenously every 2 weeks

Until pegunigalsidase alfa is commercially available or at the discretion of the Sponsor
Bi-weekly visits for infusions

Follow-up

Participants are monitored for safety and effectiveness after treatment

7 years
Every 6 months for assessments

Open-label extension

Participants continue receiving treatment to evaluate long-term safety and efficacy

7 years

Treatment Details

Interventions

  • pegunigalsidase alfa
Trial Overview The trial tests the long-term safety and effectiveness of pegunigalsidase alfa at a dose of 1 mg/kg given intravenously every two weeks to adults with Fabry Disease who participated in earlier related trials.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Experimental open labelExperimental Treatment1 Intervention
pegunigalsidase alfa

Find a Clinic Near You

Who Is Running the Clinical Trial?

Chiesi Farmaceutici S.p.A.

Lead Sponsor

Trials
206
Recruited
315,000+
Founded
1935
Headquarters
Parma, Italy
Known For
Respiratory diseases
Top Products
NEXThaler, Trimbow, Curosurf, Holoclar

Protalix

Lead Sponsor

Trials
19
Recruited
490+

Findings from Research

In a phase 3 study involving 20 adults with Fabry disease, pegunigalsidase alfa was well-tolerated, with 97% of treatment-emergent adverse events being mild or moderate, indicating a favorable safety profile.
After switching to pegunigalsidase alfa, the decline in kidney function (measured by eGFR) significantly slowed, with a mean eGFR slope of -1.19 mL/min/1.73 m2/year compared to -5.90 mL/min/1.73 m2/year before the switch, suggesting improved efficacy in preserving kidney function.
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study.Linhart, A., Dostálová, G., Nicholls, K., et al.[2023]
In a study involving 77 adults with Fabry disease, pegunigalsidase alfa was found to be non-inferior to agalsidase beta in terms of kidney function, as measured by the annualized eGFR slope over 2 years.
Pegunigalsidase alfa demonstrated a better safety profile, with significantly lower rates of treatment-related adverse events and infusion-related reactions compared to agalsidase beta.
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.Wallace, EL., Goker-Alpan, O., Wilcox, WR., et al.[2023]
In a long-term study of 15 adults with Fabry disease, pegunigalsidase alfa, a new enzyme replacement therapy, demonstrated favorable safety with most side effects being mild to moderate, and no severe infusion reactions reported.
Patients showed significant reductions in plasma lyso-Gb3 levels over 60 months, indicating effective treatment, and maintained stable kidney and cardiac function, suggesting long-term benefits of this therapy.
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease.Hughes, D., Gonzalez, D., Maegawa, G., et al.[2023]

References

Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study. [2023]
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study. [2023]
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease. [2023]
Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. [2022]
Pre-existing anti-drug antibodies in Fabry disease show less affinity for pegunigalsidase alfa. [2022]
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