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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. Identifier: NCT04552691
Expanded Access Status : Available
First Posted : September 17, 2020
Last Update Posted : January 25, 2021
Chiesi Farmaceutici S.p.A.
Information provided by (Responsible Party):

Tracking Information
First Submitted Date September 11, 2020
First Posted Date September 17, 2020
Last Update Posted Date January 25, 2021
Descriptive Information
Brief Title Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Official Title Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
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.
Detailed Description Not Provided
Study Type Expanded Access
Expanded Access Type Individual Patients
Condition Fabry Disease
Intervention 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).
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Eligibility 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.
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Contact: Kelsey Schaulsohn, PharmD 919-678-6611 ext 1957
Listed Location Countries Not Provided
Removed Location Countries  
Administrative Information
NCT Number NCT04552691
Other Study ID Numbers PB-102-F90
Responsible Party Protalix
Study Sponsor Protalix
  • Chiesi Farmaceutici S.p.A.
  • Covance
Study Director: Marcel van Kuijck, PhD Chiesi Farmaceutici S.p.A.
PRS Account Protalix
Verification Date January 2021