NCT06328608 A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
| NCT ID | NCT06328608 |
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Sponsor | Chiesi Farmaceutici S.p.A. |
| Condition | Fabry Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 22 participants |
| Start Date | 2025-07-29 |
| Primary Completion | 2028-10 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 22 participants in total. It began in 2025-07-29 with a primary completion date of 2028-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
Eligibility Criteria
Inclusion 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. Exclusion Criteria: All Subjects: * 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., ischaemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, acute postrenal obstructive nephropathy). * 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 Criteria for Subjects Enrolled in Stage I: * 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 Subjects 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 infusion.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06328608 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 17 Years, studying Fabry Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06328608 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06328608 currently recruiting?
Yes, NCT06328608 is actively recruiting participants. Contact the research team at clinicaltrials_info@chiesi.com for enrollment information.
Where is the NCT06328608 trial being conducted?
This trial is being conducted at Phoenix, United States, Atlanta, United States, Iowa City, United States, Cincinnati, United States and 8 additional locations.
Who is sponsoring the NCT06328608 clinical trial?
NCT06328608 is sponsored by Chiesi Farmaceutici S.p.A.. The trial plans to enroll 22 participants.