Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Chiesi Farmaceutici S.p.A.

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

31 Jan 2019 → 13 Apr 2026

Decision date (initial)

2024-09-13

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Chiesi Farmaceutici S.p.A

External identifiers

EU CT number

2024-516735-27-00

EudraCT number

2018-001947-30

ClinicalTrials.gov

NCT03614234

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy

Evaluate the ongoing safety and efficacy parameters of 2 mg/kg pegunigalsidase alfa every four weeks in adult Fabry patients.

Conditions and MedDRA coding

Fabry disease (α-galactosidase A deficiency)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Completion of study PB-102-F50.
2. The patient signs informed consent
3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormonereleasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination

Exclusion criteria 1

1. Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Safety. Changes from baseline in: • Clinical laboratory tests. • Physical examination. • Assessment of the injection site. • Electrocardiography • Brain MRI. • Treatment-emergent adverse events • The ability to taper off infusion’s pre-medication at the start of the study. • Requirement for the use of premedication overall to manage infusion reactions. • Treatment-induced anti-pegunigalsidase alfa antibodies.
2. Efficacy. • Estimated glomerular filtration rate (eGFRCKD-EPI). • Left Ventricular Mass Index (g/m2) by echocardiogram. • Plasma Lyso-Gb3 concentration. • Plasma Gb3 concentration • Protein/Creatinine ratio, spot urine test • Frequency of pain medication use. • Exercise tolerance (Stress Test). • Short Form Brief Pain Inventory (BPI). • Mainz Severity Score Index (MSSI). • Quality of life (EQ-5D-5L).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Chiesi Farmaceutici S.p.A.

Sponsor organisation

Chiesi Farmaceutici S.p.A.

Address

Via Palermo 26 A

City

Parma

Postcode

43122

Country

Italy

Scientific contact point

Organisation

Chiesi Farmaceutici S.p.A.

Contact name

Clinical Development, Global Rare Diseases

Public contact point

Organisation

Chiesi Farmaceutici S.p.A.

Contact name

Clinical Development, Global Rare Diseases

Third parties 9

Locations

5 EU/EEA countries · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 33 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications