Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 8 January 2018
• Main ID: NCT02995993
• Date of registration: 14/12/2016
• Prospective Registration: No
• Primary sponsor: Greenovation Biotech GmbH
• Public title: Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease
• Scientific title: An Open-Label, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGal in Patients With Fabry Disease
• Date of first enrolment: November 2016
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02995993
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Basic Science. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Germany

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with Fabry disease evidenced by a deficient a-galactosidase A (a-Gal A)
activity or an a-Gal A gene mutation (the latter is mandatory in women);

- Treatment naïve Fabry patients or Fabry patients who paused any enzyme replacement
therapy for Fabry disease due to personal reasons for 3 months before study entry;

- Female and male patients between 18 and <=65 years;

- At least one of the clinical manifestations of Fabry disease including neuropathic
pain, angiokeratoma, cornea verticillata, cardiomyopathy, hypo- or anhydrosis,
abdominal pain, diarrhea, serum creatinine >1.0 mg/dL, or proteinuria >300 mg/24
hours;

- Lyso-Gb3 concentrations in plasma above upper limit of normal;

- Male patients with a female partner of child-bearing potential agree to use a
medically acceptable method of contraception (e.g. condoms, sexual abstinence,
vasectomy), not including the rhythm method for 30 days after administration of the
study medication;

- Female patients of childbearing potential must apply a highly effective method of
birth control (failure rate less than 1% per year when used consistently and correctly
[e.g. implants, injectables, combined oral contraceptives, some intrauterine
contraceptive devices, sexual abstinence, or a vasectomized partner]). The birth
control method must have been applied for at least one monthly cycle prior to the
first administration of study medication and 30 days after administration of the study
medication.

- Patient is willing and able (in the opinion of the investigator) to understand and
comply with the procedures and evaluations of the study;

- Patient must be willing and legally able to give written informed consent.

Exclusion Criteria:

- Treatment with any enzyme replacement therapy for Fabry disease within 3 months before
study entry;

- Fabry patients who paused any enzyme replacement therapy for Fabry disease due to
intolerability;

- Patient is positive for anti-alpha-Gal A immunoglobulin G (IgG) at Screening;

- Participation in any other clinical study with a medical device or investigational
medicinal product concurrently or within 3 months before study start;

- Patient is currently on dialysis, is expected to begin dialysis during the study, has
received a kidney transplant, or is on the renal transplant waiting list;

- Patient is unable to comply with the protocol (e.g. clinical relevant medical
condition making implementation of the protocol difficult, unstable social situation,
or otherwise unlikely to complete the study) or is, in the opinion of the
investigator, otherwise unsuited for the study;

- Known human immunodeficiency virus, hepatitis B surface antigen and/or hepatitis C
infection;

- Known allergies or intolerabilities to enzyme replacement therapy;

- Hypersensitivity (like anaphylactic reaction) to the active substance or to any
excipients of moss-aGal;

- Co-administration of moss-aGal with chloroquine, amiodarone, benoquin or gentamicin;

- Breast-feeding and pregnant women;

- Patients with liver impairment;

- Women with signs of cardiac fibrosis detectable by echocardiography;

- Other, not Fabry disease-related severe illnesses;

- Malignancies within the past 5 years;

- Liver transaminases >=3 times above the upper Limit of normal;

- Alcohol and/or drug abuse;

- Weight >100 kg;

- Employees of the sponsor or patients who are employees or relatives of the
investigator.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss)

Primary Outcome(s)

AUC0-inf [Time Frame: PK sampling for 24 h after moss-aGal administration]

Number of patients with drug-related adverse events [Time Frame: Adverse event monitoring for 28 days after moss-aGal administration]

Secondary Outcome(s)

Lyso-Gb3 concentration in plasma [Time Frame: Monitoring up to Day 28 after moss-aGal administration]

Gb3 concentration in morning urine [Time Frame: Monitoring up to Day 28 after moss-aGal administration]

Gb3 concentration in plasma [Time Frame: Monitoring up to Day 28 after moss-aGal administration]

Secondary ID(s)

CT-GR-MaGal-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

FGK Clinical Research GmbH