A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Key facts
- Where
- Atlanta, Cincinnati, Fairfax, Iowa City, Phoenix és Salt Lake City, Egyesült ÁllamokSee the 6 sites
- Phase
- 2/3. fázis
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 17 years old?
Where in Egyesült Államok
- Emory Genetics Clinical Trials CenterAtlanta, Egyesült ÁllamokTaking people
- Cincinnati Children's Hospital Medical CenterCincinnati, Egyesült ÁllamokTaking people
- Lysosomal and Rare Disorders Research and Treatment Center, IncFairfax, Egyesült ÁllamokTaking people
- University of IowaIowa City, Egyesült ÁllamokTaking people
- Phoenix Children'sPhoenix, Egyesült ÁllamokTaking people
- University of UtahSalt Lake City, Egyesült ÁllamokTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Chiesi Farmaceutici S.p.A.
- Trial code
- NCT06328608EU 2022-503128-29-00
- Record updated
- 2026. augusztus 10.
- Original description
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.Read full original descriptionShow less
Multi-centre, Open-label Trial to Assess the saFety, Pharmacodynamics, Efficacy and Pharmacokinetics of pegunigaLsidase Alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed FabrY Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
Inclusion: 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.
Exclusion: 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; 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., ischaemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, acute postrenal obstructive nephropathy); 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; Receipt of treatment for Fabry disease within six months before screening; Positive for anti-PRX-102 antibodies at screening; Additional Exclusion Criteria for Subjects in Stage II (i.e., non-treatment naïve males or females):; 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; History of renal dialysis or kidney transplantation; History of or current malignancy requiring treatment; 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 Criteria for Subjects Enrolled in Stage I:; Non-classic form of Fabry disease; Unwilling to discontinue current ERT treatment for Fabry disease before baseline; Estimated glomerular filtration rate (eGFR) at screening < 80 mL/min/1.73 m2; 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; All Subjects:; Female; 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 infusion.