Fabry DiseasePhase 1/2NCT01678898
Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
A Phase 1/2 study testing PRX-102 in people with Fabry Disease, run by Protalix at 14 sites in 6 countries.
Trial summary
This is the first human treatment with PRX-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha galactosidase deficiency). The safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- PRX-102
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 14 sitesin 6 countries
- Study dates
- Oct 2012to 6 Mar 2016
- Phase
- Phase 1/2
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Symptomatic adult Fabry patients (≥18 yrs)
- Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32 nmol/hr/mg/protein)
- Females: historical genetic test results consistent with Fabry mutations
- Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit
- Patients who have never received enzyme replacement therapy (ERT) in the past, or patients who have not received ERT in the past 6 months and have a negative anti alpha galactosidase antibody test
- eGFR ≥ 60 mL/min/1.73m2
- The patient signs informed consent
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method
Who cannot join
- Participation in any trial of an investigational drug within 30 days prior to study screening
- Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)
- History of dialysis or renal transplantation
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
- Severe myocardial fibrosis by MRI (≥2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009)
- History of clinical stroke
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- Pregnant or nursing
- Presence of HIV and/or HBsAg and/or Hepatitis C infections
- Known allergies to ERT
- Known allergy to Gadolinium based contrast agents
- Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study
Trial locations and contacts
14 sites in 6 countries.
14 sites in 6 countries
United States9 sites
Department of Human Genetics, Emory University School of Medicine
Atlanta, Georgia, 30322
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205
Research Baylor Institute of Metabolic Disease
Dallas, Texas, 75226
Duke University Medical Center
Durham, North Carolina, 27710
O & O Alpan LLC
Fairfax, Virginia, 22030
University of Iowa Health Clinics
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
UC Davis Medical Center, MIND Institute Department of Pediatrics, Section of Genetics
Sacramento, California, 95817
About this study
Official title: A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Months to Adult Fabry Patients
This is the first human treatment with PRX-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha galactosidase deficiency). The safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. Patients will be treated with infusions every two weeks for 12 months.
Read the detailed description
Under the PB-102-F01 study protocol, patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) and receive PRX-102 as an intravenous infusion every 2 weeks for 12 weeks (3 months). Patients who finish the PB-102-F01 study will be enrolled in the PB-102-F02 extension study and receive the same dose they had received in the PB-102-F01 study for an additional 38 weeks (9 months).
- Conditions
- Fabry Disease
- Being tested
- PRX-102
- Keywords
- Fabry disease, Alpha galactosidase deficiency, Metabolic storage disease
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRX-102Drug
Also called plant cell expressed recombinant human alpha-galactosidase-A
| Study group | Role | What they receive |
|---|---|---|
| 0.2 mg/kg | Experimental | PRX-102 0.2 mg/kg every 2 weeks Receives: Drug: PRX-102 |
| 1 mg/kg | Experimental | PRX-102 1 mg/kg every 2 weeks Receives: Drug: PRX-102 |
| 2 mg/kg | Experimental | PRX-102 2 mg/kg every 2 weeks Receives: Drug: PRX-102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Reportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG. Results represent the number of AEs that were considered possibly, probably, or definitely related to treatment.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2012 (estimated)
- Study startOct 2012
- Primary completion6 Mar 2016
- Study completion6 Mar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Chiesi Farmaceutici S.p.A.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01678898
- Sponsor's protocol ID
- PB-102-F01 & PB-102-F02
- Status last verified
- Sep 2023
- First submitted
- 31 Aug 2012
- Last updated
- 13 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, Jennette CJ, Maegawa G, Boyadjiev SA, Gonzalez D, Nicholls K, Tuffaha A, Atta MG, Rup B, Charney MR, Paz A, Szlaifer M, Alon S, Brill-Almon E, Chertkoff R, Hughes D. Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. J Inherit Metab Dis. 2019 May;42(3):534-544. doi: 10.1002/jimd.12080. Epub 2019 Apr 8.
PubMed 30834538