MIGALASTAT HYDROCHLORIDE for Fabry disease

Inclusion criteria

• {"criterion_text":"- 1. Male or female subjects, diagnosed with Fabry disease who are between ages 2 and < 12 years at randomization (subjects aged 11 years must have birthdays > 30 days after randomization)\n- 2. 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 information, and subject provides assent, if applicable.\n- 3. Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2. • For subjects without a known GLA variant, an amenable GLA genotyping result from the local laboratory must be received prior to Visit 2.\n- 4. Subject has not received ERT (eg, Replagal® [agalsidase alfa] or Fabrazyme® [agalsidase beta]) for at least 14 days prior to Baseline visit.\n- 5. Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease, including but not limited to: a. corneal whorls b. neuropathic pain and/or acroparesthesia and/or acute crises persisting or recurring at least twice over the previous 3 months or longer, or requiring management with analgesia c. Fabry disease-related gastrointestinal signs and symptoms (eg, diarrhea, abdominal pain) persisting or recurring at least twice over the previous 3 months or longer d. hypohidrosis (present for at least 3 months) e. left ventricular mass index (LVMi) above the normal range for age and sex f. rhythm and/or conduction disturbances, for example: − episode of tachycardia or bradycardia, − arrhythmia, or; − abnormal PR, QRS, or QT interval g. reduced estimated glomerular filtration rate (eGFR) (using the Schwartz formula) for age and sex, or hyperfiltration (> 135 mL/min/1.73 m2) h. proteinuria or albuminuria in a spot urine (early morning preferable) or as determined by the investigator i. elevated plasma globotriaosylsphingosine (lyso-Gb3) j. hearing impairment and/or tinnitus k. transient ischemic attack/stroke\n- 6. If of reproductive potential, both male and female subjects 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."}

Exclusion criteria

• {"criterion_text":"- 1. Subject has moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at Visit 1 [screening]).\n- 2. Subject has advanced kidney disease requiring dialysis or kidney transplantation.\n- 3. Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).\n- 4. Subject 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).\n- 5. Subject has received any gene therapy at any time or anticipates starting gene therapy during the study period.\n- 6. Subject requires treatment with Glyset® (miglitol) or Zavesca® (miglustat), within 6 months before Visit 1 (screening) or throughout the study.\n- 7. Subject 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 may have an unacceptable risk by participating in this study.\n- 8. Female subject is pregnant or breastfeeding or is planning to become pregnant during the study period.\n- 9. In the opinion of the investigator, the subject and/or parent or legally-authorized representative is unlikely or unable to comply with the study requirements."}

Primary endpoints

• {"endpoint_text":"- Pharmacokinetics: Derived pharmacokinetic parameters for dosing confirmation will be estimated based on concentrations of migalastat in plasma.","definition_or_measurement_approach":"PK parameters will be estimated based on measured concentrations of migalastat in plasma."}
• {"endpoint_text":"- Safety: incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug; change from baseline over time in clinical laboratory test results; change from baseline over time in vital signs;change from baseline over time in physical examination findings","definition_or_measurement_approach":"Safety measured by incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events leading to discontinuation; serial clinical laboratory tests, vital signs, and physical examinations with change from baseline over time."}
• {"endpoint_text":"- change from baseline over time in body weight and height; change from baseline over time in ECG results; change from baseline to Month 12/ET in echocardiogram parameters; change from baseline to Month 12/ET in Tanner stage (for female subjects aged ≥ 8 years and male subjects aged ≥ 9 years)","definition_or_measurement_approach":"Anthropometric measures (weight, height) tracked over time; ECG results compared to baseline; echocardiogram parameters compared baseline to Month 12 or early termination; Tanner staging assessed baseline to Month 12/ET for specified age thresholds."}

Spain

Earliest CTIS Part Ii Submission Date

18-08-2025

Latest Decision Or Authorization Date

24-04-2026

Processing Time Days

250

Number Of Sites

1

Number Of Participants

1

Belgium

Earliest CTIS Part Ii Submission Date

14-11-2025

Latest Decision Or Authorization Date

10-04-2026

Processing Time Days

148

Number Of Sites

1

Number Of Participants

1

Germany

Earliest CTIS Part Ii Submission Date

06-11-2025

Latest Decision Or Authorization Date

13-04-2026

Processing Time Days

159

Number Of Sites

1

Number Of Participants

1

Primary sponsor

Full Name

Amicus Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Syneos Health Inc.

Responsibilities

sponsorDuties codes: 1, 11, 12, 2, 5, 6; 15: Home nursing service

Name

Everest Clinical Research Corporation

Responsibilities

sponsorDuties: code 3

Name

Pra International

Responsibilities

Exploratory PD biomarkers analysis

Third parties

• {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"sponsorDuties: code 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Clario Medical Imaging Inc.","duties_or_roles":"Cardiac Monitoring and Patient Outcomes","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Celerion Inc.","duties_or_roles":"PK samples analysis","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties: code 7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"sponsorDuties codes: 1, 11, 12, 2, 5, 6; 15: Home nursing service","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Everest Clinical Research Corporation","duties_or_roles":"sponsorDuties: code 3","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"long-term sample storage for future research","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pra International","duties_or_roles":"Exploratory PD biomarkers analysis","organisation_type":"Pharmaceutical company"}