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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

Tracking Information
First Submitted Date  ICMJE December 14, 2016
First Posted Date  ICMJE December 19, 2016
Last Update Posted Date December 13, 2017
Study Start Date  ICMJE November 2016
Actual Primary Completion Date October 9, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 14, 2016)
  • AUC0-inf [ Time Frame: PK sampling for 24 h after moss-aGal administration ]
    Area under the serum concentration curve extrapolated to infinity
  • Number of patients with drug-related adverse events [ Time Frame: Adverse event monitoring for 28 days after moss-aGal administration ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02995993 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 14, 2016)
  • Gb3 concentration in plasma [ Time Frame: Monitoring up to Day 28 after moss-aGal administration ]
    Globotriaosylceramide concentration in plasma
  • Gb3 concentration in morning urine [ Time Frame: Monitoring up to Day 28 after moss-aGal administration ]
    Globotriaosylceramide concentration in morning urine
  • Lyso-Gb3 concentration in plasma [ Time Frame: Monitoring up to Day 28 after moss-aGal administration ]
    Globotriaosylsphingosine concentration in plasma
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease
Official Title  ICMJE An Open-Label, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGal in Patients With Fabry Disease
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Condition  ICMJE Fabry Disease
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Moss-aGal
Single administration of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion
Intervention: Drug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
Publications * Shen JS, Busch A, Day TS, Meng XL, Yu CI, Dabrowska-Schlepp P, Fode B, Niederkrüger H, Forni S, Chen S, Schiffmann R, Frischmuth T, Schaaf A. Mannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry mice. J Inherit Metab Dis. 2016 Mar;39(2):293-303. doi: 10.1007/s10545-015-9886-9. Epub 2015 Aug 27.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 14, 2016)
6
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 9, 2017
Actual Primary Completion Date October 9, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02995993
Other Study ID Numbers  ICMJE CT‐GR‐MaGal‐01
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Greenovation Biotech GmbH
Study Sponsor  ICMJE Greenovation Biotech GmbH
Collaborators  ICMJE FGK Clinical Research GmbH
Investigators  ICMJE Not Provided
PRS Account Greenovation Biotech GmbH
Verification Date December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP