A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (SHORTEN)
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ClinicalTrials.gov Identifier: NCT06019728 |
Recruitment Status :
Recruiting
First Posted : August 31, 2023
Last Update Posted : March 6, 2024
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Fabry's Disease | Drug: AGALSIDASE BETA (GZ419828) Drug: Acetaminophen Drug: Diphenhydramine Drug: Dexamethasone Drug: Montelukast | Phase 4 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 18 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Study to Investigate Safety and Tolerability of Higher Infusion Rate to shORten the duraTion of FabrazymE Infusion |
Actual Study Start Date : | November 10, 2023 |
Estimated Primary Completion Date : | October 6, 2025 |
Estimated Study Completion Date : | October 6, 2025 |
Arm | Intervention/treatment |
---|---|
Experimental: agalsidase beta
agalsidase beta 1 mg/kg infusion once every other week
|
Drug: AGALSIDASE BETA (GZ419828)
Pharmaceutical form: Lyophilized powder for reconstitution Route of administration: IV infusion
Other Name: Fabrazyme Drug: Acetaminophen Tablet or solution; Oral Drug: Diphenhydramine Tablet or solution; Oral Drug: Dexamethasone Tablet or solution; Oral Drug: Montelukast Tablet or chewable tablet or oral granules; Oral |
- Reduction of infusion duration from pretrial average of recent 3 infusions [ Time Frame: Baseline to month 4 ]
- Reduction of infusion duration from initial 120 minutes [ Time Frame: Baseline to month 4 ]
- Shortest infusion duration each participant tolerates [ Time Frame: Baseline to month 4 ]
- Number of participants achieving the shortest planned duration of infusion time [ Time Frame: Baseline to month 4 ]
- Percentage of participants achieving the shortest planned duration of infusion time [ Time Frame: Baseline to month 4 ]
- Number of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [ Time Frame: Baseline to month 4 ]
- Percentage of participants achieving infusion duration shorter than 90 minutes without experiencing any or the second IAR [ Time Frame: Baseline to month 4 ]
- Number of participants achieving infusion duration without experiencing any IAR [ Time Frame: Baseline to month 4 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 2 Years to 65 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Participants with confirmed diagnosis of FD who are ≥2 and ≤65 years of age at the time of signing the informed consent form (ICF) or assent, if applicable.
- Cohort 1: female participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 2: non-classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 3: classic male participants with body weight ≥30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 4: participants with body weight <30 kg who have been treated with Fabrazyme for at least 3 months without IARs during the most recent 3 infusions.
- Cohort 5: ERT-naïve participants. Women of childbearing potential must use a highly effective method of contraception through the study.
Exclusion Criteria:
- Female participants who are pregnant or breastfeeding.
- History of significant allergic disease or hypersensitivity to Fabrazyme or other medicinal products.
- Contraindication to Fabrazyme or any of the premedications or rescue medications (diphenhydramine, acetaminophen, montelukast, dexamethasone).
- Any other medical condition considered to make the increased infusion rate not tolerable at the Investigator's discretion. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06019728
Contact: Trial Transparency email recommended (Toll free for US & Canada) | 800-633-1610 ext option 6 | Contact-US@sanofi.com |
United States, Michigan | |
Infusion Associates Site Number : 1001 | Recruiting |
Grand Rapids, Michigan, United States, 49525 | |
United States, New York | |
Metropolitan Hospital Center Site Number : 1004 | Recruiting |
New York, New York, United States, 10021 | |
United States, Virginia | |
Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 1002 | Recruiting |
Fairfax, Virginia, United States, 22030 |
Study Director: | Clinical Sciences & Operations | Sanofi |
Responsible Party: | Sanofi |
ClinicalTrials.gov Identifier: | NCT06019728 |
Other Study ID Numbers: |
LPS17726 U1111-1287-8570 ( Registry Identifier: ICTRP ) |
First Posted: | August 31, 2023 Key Record Dates |
Last Update Posted: | March 6, 2024 |
Last Verified: | March 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Vascular Diseases Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors |
Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders Acetaminophen Diphenhydramine Promethazine Dexamethasone Montelukast Anti-Inflammatory Agents Antiemetics Autonomic Agents Peripheral Nervous System Agents Physiological Effects of Drugs |