AzTrials
AzTrials

Fabry DiseasePhase 1NCT02995993

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

A Phase 1 study testing Moss-aGal (recombinant human alpha-galactosidase A produced in moss) in people with Fabry Disease, run by Greenovation Biotech GmbH at 2 sites in Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Fabry Disease
What is being tested?
Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
2 sitesGermany
Study dates
Nov 2016to 9 Oct 2017
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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;
Show all 9Show fewer
  • 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.

Who cannot join

  • 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;
Show all 19Show fewer
  • 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.

Trial locations and contacts

2 sites in 1 country.

Germany2 sites

  • Ruhruniversität Bochum, Klinik für Kinder- und Jugendmedizin im St. Josef-Hospital im Katholischen Klinikum Bochum

    Bochum, 44791

  • Universitätsmedizin Mainz, Zentrum für Kinder- und Jugendmedizin

    Mainz, 55131

Other trials in Germany

About this study

Official title: An Open-Label, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGal in Patients With Fabry Disease

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.

Conditions
Fabry Disease
Being tested
Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
Medical subject headings
Fabry Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

    Single i.v. Infusion of 0.2 mg/kg moss-aGal over 60 minutes

Study groups in NCT02995993
Study groupRoleWhat they receive
Moss-aGalExperimentalSingle administration of 0.2 mg/kg recombinant human alpha-galactosidase A produced in moss (moss-aGal) as intravenous infusion
Receives: Drug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. AUC0-inf

    Area under the serum concentration curve extrapolated to infinity

    Measured: PK sampling for 24 h after moss-aGal administration

  2. Number of patients with drug-related adverse events

    Measured: Adverse event monitoring for 28 days after moss-aGal administration

Secondary outcomes (3)

  1. Gb3 concentration in plasma

    Globotriaosylceramide concentration in plasma

    Measured: Monitoring up to Day 28 after moss-aGal administration

  2. Gb3 concentration in morning urine

    Globotriaosylceramide concentration in morning urine

    Measured: Monitoring up to Day 28 after moss-aGal administration

  3. Lyso-Gb3 concentration in plasma

    Globotriaosylsphingosine concentration in plasma

    Measured: Monitoring up to Day 28 after moss-aGal administration

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Dec 2016 (estimated)
  2. Study startNov 2016
  3. Primary completion9 Oct 2017
  4. Study completion9 Oct 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Basic science
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02995993
Sponsor's protocol ID
CT-GR-MaGal-01
Status last verified
Dec 2017
First submitted
14 Dec 2016
Last updated
13 Dec 2017
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • Shen JS, Busch A, Day TS, Meng XL, Yu CI, Dabrowska-Schlepp P, Fode B, Niederkruger H, Forni S, Chen S, Schiffmann R, Frischmuth T, Schaaf A. Mannose receptor-mediated delivery of moss-made alpha-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.

    PubMed 26310963

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Moss-aGal (recombinant human alpha-galactosidase A produced in moss) in people with Fabry Disease, run by Greenovation Biotech GmbH at 2 sites in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

AUC0-inf (time frame: PK sampling for 24 h after moss-aGal administration). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02995993, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Dec 2017.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in