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A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease
EU CTIS ID: 2022-503128-29-00
What this study is testing
To evaluate the safety, pharmacodynamics, efficacy and pharmacokinetics of PRX-102 in three different age cohorts in paediatric patients with confirmed Fabry disease.
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to <18 years (Cohort C)
- A documented diagnosis of Fabry disease, as determined by the following: • Males: Plasma and/or leukocyte alpha-galactosidase-A (α-GAL-A) activity (by activity assay) that is ≤ 5% of mean normal laboratory levels, or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the α-GAL-A (GLA)gene. • Females: Historical genetic test results consistent with Fabry mutations, or, in the case of novel mutations, a first-degree male relative with Fabry disease. • All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma.
- History of Fabry pain: • Episodic crises (Fabry crises) characterised by agonizing burning pain originating in the extremities and radiating inwards to the limbs and other parts of the body, OR • Chronic pain characterised by burning and tingling paraesthesia
- Clinical condition that, in the opinion of the Investigator, requires treatment with enzyme replacement therapy (ERT).
You likely can't join if
- Estimated glomerular filtration rate (eGFR) at screening < 80 mL/min/1.73 m2, calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation (2012).
- Additional Exclusion Criteria for Subjects Enrolled in Stage I:For subjects enrolled in Stage I (targeting up to 9 subjects total) these specific exclusion criteria, in addition to those above, apply: a) Female b) Non-classic form of Fabry disease c) Receipt of treatment for Fabry disease within 6 months prior to screening d) Positive for anti-PRX-102 antibodies at screening e) Aged 4 years or younger
- Additional Exclusion Criteria for Subjects in Stage II : For subjects enrolled in Stage II, these specific exclusion criteria, in addition to exclusion criteria #1 to #11 apply a) Unwilling to discontinue current ERT treatment for Fabry disease at least 14 days, or chaperone therapy at least 3 days, before baseline.
- Additional Exclusion Criteria for Subjects in Stage II: Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and/or unwilling to undergo pregnancy testing as outlined and to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion. Note: Before the start of treatment, the Investigator will decide whether or not pregnancy testing and contraception counselling are necessary. Since over the course of the study, pre-pubertal girls may reach menarche and adolescents of either gender may become sexually active, the Investigator must periodically check on the status of these issues and implement pregnancy testing and/or contraception counselling if required. A female subject is considered of childbearing potential, i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
- History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or to any component of the study drug.
- Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB), or a change of dose in ongoing treatment, in the 4 weeks prior to screening.
See the full eligibility criteria
Who can join
- Male or female aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to <18 years (Cohort C)
- A documented diagnosis of Fabry disease, as determined by the following: • Males: Plasma and/or leukocyte alpha-galactosidase-A (α-GAL-A) activity (by activity assay) that is ≤ 5% of mean normal laboratory levels, or, if the enzymatic activity is above the 5% limit but still under the normal level, a confirmed disease-causing mutation of the α-GAL-A (GLA)gene. • Females: Historical genetic test results consistent with Fabry mutations, or, in the case of novel mutations, a first-degree male relative with Fabry disease. • All subjects: At least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma.
- History of Fabry pain: • Episodic crises (Fabry crises) characterised by agonizing burning pain originating in the extremities and radiating inwards to the limbs and other parts of the body, OR • Chronic pain characterised by burning and tingling paraesthesia
- Clinical condition that, in the opinion of the Investigator, requires treatment with enzyme replacement therapy (ERT).
What rules you out
- Estimated glomerular filtration rate (eGFR) at screening < 80 mL/min/1.73 m2, calculated using the Creatinine Cystatin C-based Chronic Kidney Disease in Children (CKiD) equation (2012).
- Additional Exclusion Criteria for Subjects Enrolled in Stage I:For subjects enrolled in Stage I (targeting up to 9 subjects total) these specific exclusion criteria, in addition to those above, apply: a) Female b) Non-classic form of Fabry disease c) Receipt of treatment for Fabry disease within 6 months prior to screening d) Positive for anti-PRX-102 antibodies at screening e) Aged 4 years or younger
- Additional Exclusion Criteria for Subjects in Stage II : For subjects enrolled in Stage II, these specific exclusion criteria, in addition to exclusion criteria #1 to #11 apply a) Unwilling to discontinue current ERT treatment for Fabry disease at least 14 days, or chaperone therapy at least 3 days, before baseline.
- Additional Exclusion Criteria for Subjects in Stage II: Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and/or unwilling to undergo pregnancy testing as outlined and to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion. Note: Before the start of treatment, the Investigator will decide whether or not pregnancy testing and contraception counselling are necessary. Since over the course of the study, pre-pubertal girls may reach menarche and adolescents of either gender may become sexually active, the Investigator must periodically check on the status of these issues and implement pregnancy testing and/or contraception counselling if required. A female subject is considered of childbearing potential, i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
- History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or to any component of the study drug.
- Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB), or a change of dose in ongoing treatment, in the 4 weeks prior to screening.
- Subject with 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.
- Carry only known non-pathogenic Fabry mutations.
- History of acute kidney injury in the 12 months prior to 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 6 months prior to screening.
- Presence of any medical, emotional, behavioural, or psychological condition that in the judgement of the Investigator could interfere with the subject’s compliance with the requirements of the study.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- 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, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. 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 EU CTIS; the study team decides eligibility.