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History of Changes for Study: NCT03425539
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (MODIFY)
Latest version (submitted August 5, 2022) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 February 1, 2018 None (earliest Version on record)
2 February 13, 2018 Contacts/Locations, Outcome Measures and Study Status
3 April 13, 2018 Study Status
4 June 6, 2018 Recruitment Status, Contacts/Locations, Study Status and Oversight
5 June 20, 2018 Contacts/Locations and Study Status
6 July 5, 2018 Contacts/Locations and Study Status
7 July 25, 2018 Contacts/Locations and Study Status
8 August 28, 2018 Contacts/Locations and Study Status
9 August 29, 2018 Contacts/Locations and Study Status
10 September 13, 2018 Study Status and Contacts/Locations
11 October 24, 2018 Study Status and Contacts/Locations
12 November 20, 2018 Study Status and Contacts/Locations
13 December 18, 2018 Study Status
14 January 22, 2019 Contacts/Locations and Study Status
15 February 13, 2019 Study Status and Contacts/Locations
16 March 8, 2019 Study Status
17 March 12, 2019 Contacts/Locations and Study Status
18 April 10, 2019 Study Status and Contacts/Locations
19 May 9, 2019 Contacts/Locations and Study Status
20 May 14, 2019 Contacts/Locations and Study Status
21 June 21, 2019 Contacts/Locations and Study Status
22 July 17, 2019 Study Status and Contacts/Locations
23 August 16, 2019 Contacts/Locations and Study Status
24 August 20, 2019 Contacts/Locations and Study Status
25 September 25, 2019 Contacts/Locations and Study Status
26 October 15, 2019 Study Status
27 October 18, 2019 Study Status
28 November 21, 2019 Contacts/Locations and Study Status
29 December 12, 2019 Contacts/Locations and Study Status
30 December 20, 2019 Study Status
31 January 28, 2020 Study Status and Contacts/Locations
32 February 26, 2020 Contacts/Locations and Study Status
33 April 28, 2020 Study Status and Contacts/Locations
34 June 23, 2020 Study Status and Contacts/Locations
35 July 22, 2020 Study Status and Contacts/Locations
36 August 19, 2020 Study Status and Contacts/Locations
37 September 23, 2020 Study Status and Contacts/Locations
38 November 17, 2020 Study Status, Outcome Measures, Contacts/Locations and Study Design
39 January 12, 2021 Recruitment Status, Contacts/Locations, Study Status and Study Design
40 January 28, 2021 Contacts/Locations and Study Status
41 March 2, 2021 Study Status
42 September 14, 2021 Study Status
43 September 16, 2021 Recruitment Status and Study Status
44 October 28, 2021 Study Status
45 August 2, 2022 Study Status
46 August 5, 2022 Arms and Interventions and Study Status
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Study NCT03425539
Submitted Date:  February 1, 2018 (v1)

Open or close this module Study Identification
Unique Protocol ID: ID-069A301
Brief Title: Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (MODIFY)
Official Title: A Multicenter, dOuble-blind, ranDomized, Placebo-controlled, Parallel-group Study to Determine the effIcacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With FabrY Disease
Secondary IDs:
Open or close this module Study Status
Record Verification: February 2018
Overall Status: Not yet recruiting
Study Start: March 30, 2018
Primary Completion: August 19, 2019 [Anticipated]
Study Completion: September 21, 2019 [Anticipated]
First Submitted: January 16, 2018
First Submitted that
Met QC Criteria:
February 1, 2018
First Posted: February 7, 2018 [Actual]
Last Update Submitted that
Met QC Criteria:
February 1, 2018
Last Update Posted: February 7, 2018 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Idorsia Pharmaceuticals Ltd.
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
Detailed Description: The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.
Open or close this module Conditions
Conditions: Fabry Disease
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 3
Interventional Study Model: Parallel Assignment
Number of Arms: 2
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation: Randomized
Enrollment: 108 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: Lucerastat Drug: Lucerastat
Hard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) b.i.d.; dose adjusted for renal function.
Placebo Comparator: Placebo Drug: Placebo
Placebo capsules are identical in appearance to the lucerastat capsules, and contain inactive excipients; 4 capsules b.i.d.; dose adjusted for renal function.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Response to study treatment on neuropathic pain defined as a reduction from baseline to Month 6 of at least 30% in the "modified" BPI-SF3 score of "neuropathic pain at its worst in the last 24 hours"
[ Time Frame: From baseline to Month 6 (duration: 6 months) ]

Secondary Outcome Measures:
1. Change from baseline to Month 6 in the average daily 11-point Numerical Rating Scale (NRS-11) score of "abdominal pain at its worst in the last 24 hours" in subjects with GI symptoms at baseline.
[ Time Frame: From baseline to Month 6 (duration: 6 months) ]

2. Change from baseline to Month 6 in the number of days with at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 in subjects with GI symptoms at baseline.
[ Time Frame: From baseline to Month 6 (duration: 6 months) ]

3. Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3).
[ Time Frame: From baseline to Month 6 (duration: 6 months) ]

Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  1. Signed and dated ICF prior to any study-mandated procedure;
  2. Male or female adult subjects;
  3. FD diagnosis confirmed with local genetic test results;
  4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
  5. Enzyme replacement therapy (ERT) status:
    1. Subject never treated with ERT; or
    2. Subject has not received ERT for at least 6 months prior to screening; or
    3. Subject treated with ERT since at least 12 months at the time of the screening visit, and agreeing to stop ERT for approximately 8 months.
  6. A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
  7. Subjects with moderate or severe neuropathic pain during the screening period.

Exclusion Criteria:

  1. Pregnant, planning to be become pregnant, or lactating subject.
  2. Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.
  3. Subject on regular dialysis for the treatment of chronic kidney disease.
  4. Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
  5. Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
  6. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.
Open or close this module Contacts/Locations
Central Contact Person: Fabrice Kramer, MD
Telephone: +41 58 844 00 00
Email: AC-069A301@idorsia.com
Study Officials: Fabrice Kramer, MD
Study Director
Idorsia Pharmaceuticals
Locations: United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Contact:Contact: Eric Wallace, Prof. ericlwallace@uab.edu
United States, California
University of California Irvine
Irvine, California, United States, 92696
Contact:Contact: Virginia Kimonis, Dr. vkimonis@uci.edu
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
Contact:Contact: John Bernat, Prof. john-bernat@uiowa.edu
United States, Kansas
University of Kansas Medical Center - Department of Neurology
Kansas City, Kansas, United States, 66160
Contact:Contact: Ahmad Tuffaha, Dr. atuffaha@kumc.edu
United States, Minnesota
University of Minnesota - Pediatric Gene Therapy
Minneapolis, Minnesota, United States, 55455
Contact:Contact: Chester Whitley, Prof. whitley@umn.edu
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Contact:Contact: Robert Hopkin, Prof. rob.hopkin@cchmc.org
United States, Pennsylvania
Children's Hospital of Pittsburgh (UPMC)
Pittsburgh, Pennsylvania, United States, 15224
Contact:Contact: Damara Ortiz, Dr. damara.ortiz@chp.edu
United States, Texas
Research Baylor Institute of Metabolic Disease
Dallas, Texas, United States, 75226
Contact:Contact: Raphael Schiffmann, Dr. raphael.schiffmann@bswhealth.org
United States, Utah
University of Iowa Hospitals and Clinics
Salt Lake City, Utah, United States, 84113
Contact:Contact: Nicola Longo, Prof. nicola.longo@hsc.utah.edu
United States, Virginia
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030
Contact:Contact: Ozlem Goker-Alpan, Dr. ogoker-alpan@ldrtc.org
Canada
Queen Elizabeth II Health Sciences Center Halifax Infirmary - Division of Neurosurgery
Halifax, Canada, B3H 3A7
Contact:Contact: Michael West, Prof.
Contact:Contact: MLWEST@dal.ca
Vancouver Hospital & Health Sciences - Vancouver General Hospital
Vancouver, Canada, V5Z 1M9
Contact:Contact: Sandra Sirrs, Prof. sandra.sirrs@vch.ca
Health Sciences Center Winnipeg
Winnipeg, Canada, R3A 1S1
Contact:Contact: Cheryl Greenberg, Prof. cgreenberg@hsc.mb.ca
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Contact:Contact: Aneal Khan, Dr. khaa@ucalgary.ca
Czechia
The Charles University
Praha, Czechia, 128 08
Contact:Contact: Ales Linhart, Prof. ales.linhart@lf1.cuni.cz
France
Université de Versailles - Saint Quentin en Yvelines
Garches, France, 92380
Contact:Contact: Dominique Germain, Prof. dominique.germain@rpc.aphp.fr
Germany
Charite Campus Virchow-Klinikum
Berlin, Germany, 10117
Contact:Contact: Sima Canaan-Kühl, Dr. sima.canaan-kuehl@charite.de
Fachinternistische Gemeinschaftspraxis Markgräferland
Mühlheim, Germany, 79379
Contact:Contact: Marcus Cybulla, Dr. dr.cybulla@f-g-m.de
Medizinische Klinik und Poliklinik I der Universität Schwerpunkt Nephrologie
Würzburg, Germany, 97080
Contact:Contact: Christoph Wanner, Prof. wanner_C@ukw.de
Netherlands
Academic Medical Center
Amsterdam, Netherlands, 22660
Contact:Contact: Carla Hollak, Prof. c.e.hollak@amc.uva.nl
United Kingdom
University Hospital Birmingham NHS Foundation Trust
Birmingham, United Kingdom, B15 2WB
Contact:Contact: Tarekegn Hiwot, Dr. tarekegn.geberhiwot@uhb.nhs.uk
Lysosomal Storage Disorders Unit, Department of Hematology
London, United Kingdom, NW3 2QG
Contact:Contact: Derralynn Hughes, Dr. derralynnhughes@nhs.net
National Hospital for Neurology and Neurosurgery
London, United Kingdom, WC1N3BG
Contact:Contact: Robin Lachmann, Dr. r.lachmann@nhs.net
Department of Endocrinology and Metabolic Medicine
Salford, United Kingdom, M6 8HD
Contact:Contact: Ana Jovanovic, Dr. ana.jovanovic@srft.nhs.uk
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Citations:
Links:
Available IPD/Information:

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