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Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04552691
Expanded Access Status : Available
First Posted : September 17, 2020
Last Update Posted : November 17, 2020
Sponsor:
Collaborators:
Chiesi Farmaceutici S.p.A.
Covance
Information provided by (Responsible Party):
Protalix

Brief Summary:
The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.

Condition or disease Intervention/treatment
Fabry Disease Drug: Pegunigalsidase Alfa

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
Official Title: Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients



Intervention Details:
  • Drug: Pegunigalsidase Alfa
    Pengunigalsidase alfa is a recombinant ERT (enzyme replacement therapy) used to treat Fabry disease (dosage: 1 mg/kg body weight every 2 weeks).

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  • In the opinion of the Treating Physician, the patient cannot be adequately treated with any FDA approved drugs for Fabry and is not able to enroll in any current clinical trial for Fabry disease.
  • Patient (or legal guardian) is able to sign an informed consent prior to treatment.
  • A documented diagnosis of Fabry disease.
  • Preferably two, but at minimum 1, historical serum creatinine evaluations in the last 2 years with the latest value within the last 6 months.
  • 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. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 90 days after treatment discontinuation.

Exclusion Criteria:

  • Patients enrolled and currently treated in Study PB-102-F20, and patients enrolled and currently treated in Extension Study PB-102-F60
  • Patients who currently are on treatment under any other ongoing clinical trials of PRX-102
  • History of Type 1 (anaphylaxis or anaphylactoid like) life-threatening hypersensitivity during previous exposure to other ERTs which could not be handled with medication
  • Women who are breastfeeding may not participate unless they agree to stop breastfeeding.
  • Women who are currently pregnant.

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): NCT04552691


Contacts
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Contact: Kelsey Schaulsohn, PharmD 919-678-6611 ext 1957 US.EAP@Chiesi.com
Contact: Lokesh Jha, PhD US.EAP@Chiesi.com

Sponsors and Collaborators
Protalix
Chiesi Farmaceutici S.p.A.
Covance
Investigators
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Study Director: Marcel van Kuijck, PhD Chiesi Farmaceutici S.p.A.
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Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT04552691    
Other Study ID Numbers: PB-102-F90
First Posted: September 17, 2020    Key Record Dates
Last Update Posted: November 17, 2020
Last Verified: November 2020
Keywords provided by Protalix:
Fabry Disease
Pegunigalsidase alfa
Additional relevant MeSH terms:
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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