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

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ClinicalTrials.gov Identifier: NCT02995993
Recruitment Status : Completed
First Posted : December 19, 2016
Last Update Posted : December 13, 2017
Sponsor:
Collaborator:
FGK Clinical Research GmbH
Information provided by (Responsible Party):
Greenovation Biotech GmbH

Brief Summary:
Six patients with Fabry disease will be recruited. Patients will receive a single dose of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion. Patients will be hospitalized during the infusion and for at least 24 hours after the end of the infusion. Treatment will be administered sequentially: if a patient shows no safety concerns on the treatment day, treatment of the next patient will commence on the following day.

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss) Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: An Open-Label, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGal in Patients With Fabry Disease
Study Start Date : November 2016
Actual Primary Completion Date : October 9, 2017
Actual Study Completion Date : October 9, 2017

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Moss-aGal
Single administration of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion
Drug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
Single i.v. Infusion of 0.2 mg/kg moss-aGal over 60 minutes




Primary Outcome Measures :
  1. AUC0-inf [ Time Frame: PK sampling for 24 h after moss-aGal administration ]
    Area under the serum concentration curve extrapolated to infinity

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

Secondary Outcome Measures :
  1. Gb3 concentration in plasma [ Time Frame: Monitoring up to Day 28 after moss-aGal administration ]
    Globotriaosylceramide concentration in plasma

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

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



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with Fabry disease evidenced by a deficient α-galactosidase A (α-Gal A) activity or an α-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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02995993


Locations
Germany
Ruhruniversität Bochum, Klinik für Kinder- und Jugendmedizin im St. Josef-Hospital im Katholischen Klinikum Bochum
Bochum, Germany, 44791
Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin
Mainz, Germany, 55131
Sponsors and Collaborators
Greenovation Biotech GmbH
FGK Clinical Research GmbH

Publications:
Responsible Party: Greenovation Biotech GmbH
ClinicalTrials.gov Identifier: NCT02995993     History of Changes
Other Study ID Numbers: CT‐GR‐MaGal‐01
First Posted: December 19, 2016    Key Record Dates
Last Update Posted: December 13, 2017
Last Verified: December 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders