BridgeMD
New treatment option for Fabry Disease
Official title A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
ClinicalTrials.gov ID: NCT06904261
What this study is testing
What is Migalastat HCl 20 mg?
Migalastat HCl 20 mg is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for fabry disease.
Also referred to as AT1001, Galafold.
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
- An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
- Phase 3: a large, late-stage study
- Time commitment: about 9 months
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 11
You may be able to join if
- Male or female people, diagnosed with Fabry disease who are between ages 2 and \ 30 days after randomization)
- Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and...
- Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
- Subject has not received ERT (eg, Replagal® [agalsidase alfa] or Fabrazyme® [agalsidase beta]) for at least 14 days prior to Baseline visit.
- Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease
You likely can't join if
- Has moderate or severe renal impairment (eGFR \< 60 mL/min/1.73 m2 at Visit 1 [screening]).
- Has advanced kidney disease requiring dialysis or kidney transplantation.
- History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
- Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is...
- Has received any gene therapy at any time or anticipates starting gene therapy during the study period.
- Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.
See the full eligibility criteria
- Male or female people, diagnosed with Fabry disease who are between ages 2 and \ 30 days after randomization)
- Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health...
- Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
- Subject has not received ERT (eg, Replagal® [agalsidase alfa] or Fabrazyme® [agalsidase beta]) for at least 14 days prior to Baseline visit.
- Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease
- If of reproductive potential, both male and female people agree to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat.
- Has moderate or severe renal impairment (eGFR \< 60 mL/min/1.73 m2 at Visit 1 [screening]).
- Has advanced kidney disease requiring dialysis or kidney transplantation.
- History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
- Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).
- Has received any gene therapy at any time or anticipates starting gene therapy during the study period.
- Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.
- Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject...
- Pregnant or breastfeeding
- Otherwise unsuitable for the study in the opinion of the investigator
The study team makes the final eligibility decision.
Where it's taking place
- Atlanta, Georgia, United States
- Minneapolis, Minnesota, United States
- Charlotte, North Carolina, United States
- Cincinnati, Ohio, United States
- Pittsburgh, Pennsylvania, United States
- Fairfax, Virginia, United States
- Leuven, Vlaams-Brabant, Belgium
- Münster, North Rhine-Westphalia, Germany
- Madrid, Madrid, Spain
- London, United Kingdom
- Manchester, 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 study runs about 9 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Atlanta, Georgia, United States; Minneapolis, Minnesota, United States; Charlotte, North Carolina, United States; Cincinnati, Ohio, United States; Pittsburgh, Pennsylvania, United States; Fairfax, Virginia, United States and 5 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.