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

• ClinicalTrials.gov Identifier: NCT04552691
• Expanded Access Status: Available
• First Posted: September 17, 2020
• Last Update Posted: January 25, 2021
• Sponsor: Protalix
• Collaborators: Chiesi Farmaceutici S.p.A.; Covance
• Information provided by (Responsible Party): Protalix

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

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)

Contacts

Contact: Kelsey Schaulsohn, PharmD; 919-678-6611 ext 1957; GRD.EAP@Chiesi.com

• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT04552691
• Other Study ID Numbers: PB-102-F90
• Responsible Party: Protalix
• Study Sponsor: Protalix

Collaborators

• Chiesi Farmaceutici S.p.A.
• Covance

Investigators

• Study Director: Marcel van Kuijck, PhD; Chiesi Farmaceutici S.p.A.

• PRS Account: Protalix
• Verification Date: January 2021