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Tests treatment safety and results for Fabry Disease
Official title A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
ClinicalTrials.gov ID: NCT06328608
What this study is testing
What is PRX-102 1 mg/kg every two weeks?
PRX-102 1 mg/kg every two weeks is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fabry disease.
Also referred to as pegunigalsidase alfa, Recombinant human alpha galactosidase-A.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 17
You may be able to join if
- Participants with the provision of informed consent from their legal guardians
- Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to \<18 years (Cohort C).
- Confirmed diagnosis of Fabry disease
- Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered...
- History of Fabry pain: Fabry crises OR chronic pain.
You likely can't join if
- All people:
- Estimated glomerular filtration rate (eGFR) at screening \< 80 mL/min/1.73 m2.
- History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or...
- Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB) or a dose change in ongoing...
- Urine protein to creatinine ratio (UPCR) \> 0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB.
- Currently taking another investigational drug for any condition.
See the full eligibility criteria
- Participants with the provision of informed consent from their legal guardians
- Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to \<18 years (Cohort C).
- Confirmed diagnosis of Fabry disease
- Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma.
- History of Fabry pain: Fabry crises OR chronic pain.
- Clinical condition that, in the investigator's opinion, requires ERT treatment.
- All people:
- Estimated glomerular filtration rate (eGFR) at screening \< 80 mL/min/1.73 m2.
- History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or any component of the study drug.
- Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB) or a dose change in ongoing treatment in the four weeks before screening.
- Urine protein to creatinine ratio (UPCR) \> 0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB.
- Currently taking another investigational drug for any condition.
- History of acute kidney injury in the 12 months before screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g...
- History of renal dialysis or kidney transplantation.
- History of or current malignancy requiring treatment.
- Severe cardiomyopathy or significant unstable cardiac disease within six months before screening.
- A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within three months before screening.
- Presence of any medical, emotional, behavioural, or psychological condition that, in the Investigator's judgement, could interfere with the subject's compliance with the requirements of the study. Additional Exclusion...
- Female
- Non-classic form of Fabry disease
- Receipt of treatment for Fabry disease within six months before screening
- Positive for anti-PRX-102 antibodies at screening Additional Exclusion Criteria for people in Stage II (i.e., non-treatment naïve males or females):
- Unwilling to discontinue current ERT treatment for Fabry disease before baseline.
- Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last...
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Atlanta, Georgia, United States
- Iowa City, Iowa, United States
- Cincinnati, Ohio, United States
- Salt Lake City, Utah, United States
- Fairfax, Virginia, United States
- Salzburg, Austria
- Bordeaux, France
- Montpellier, France
- Bergen, Norway
- Santiago de Compostela, Spain
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Atlanta, Georgia, United States; Iowa City, Iowa, United States; Cincinnati, Ohio, United States; Salt Lake City, Utah, United States; Fairfax, Virginia, United States and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.