Fabry DiseaseNCT04552691
Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
A study testing Pegunigalsidase Alfa in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 11 sites in United States.
Trial 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… 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?
- Pegunigalsidase Alfa
- How many people
- Not given
- Where
- 11 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
University of Alabama-Birmingham
Birmingham, Alabama, 35294
Dallas Nephrology Associates
Dallas, Texas, 75235-2208
Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
Fairfax, Virginia, 22030
Infusion Associates
Grand Rapids, Michigan, 49525
University of Iowa
Iowa City, Iowa, 52242
University of Florida, Division of Pediatric Genetics
Jacksonville, Florida, 32207
University of California Irvine
Orange, California, 92868
Phoenix Children's Hospital, Inc.
Phoenix, Arizona, 85006
Central Coas Nephrology
Salinas, California, 93901
University of Utah
Salt Lake City, Utah, 84108
About this study
Official title: Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
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.
- Conditions
- Fabry Disease
- Being tested
- Pegunigalsidase Alfa
- Keywords
- Fabry Disease, Pegunigalsidase alfa
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pegunigalsidase AlfaDrug
Pegunigalsidase alfa is a recombinant ERT (enzyme replacement therapy) used to treat Fabry disease (dosage: 1 mg/kg body weight every 2 weeks).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Chiesi USA, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04552691
- Sponsor's protocol ID
- PB-102-F90
- Status last verified
- Jul 2024
- First submitted
- 11 Sep 2020
- Last updated
- 31 Jul 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
- ClinicalTrials.gov ID
- NCT04552691
- Acronym / sponsor ID
- PB-102-F90
- Conditions
- Fabry Disease
- Keywords
- Fabry Disease, Pegunigalsidase alfa
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Collaborators
- Chiesi USA, Inc.
- First posted
- 17 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pegunigalsidase AlfaPegunigalsidase alfa is a recombinant ERT (enzyme replacement therapy) used to treat Fabry disease (dosage: 1 mg/kg body weight every 2 weeks). | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Pegunigalsidase Alfa in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 11 sites in United States.
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 5 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 31 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. 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.
Are results available for this study?
Results are not available in the source record.
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.
Where can I read the original study information?
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 31 Jul 2024.