Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

Official title:

An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 or VBP15-006 Studies

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT03863119
• Other study ID #: VBP15-EAP
• Status: Available

Study information

• Verified date: September 2023
• Source: Santhera Pharmaceuticals
• Contact: Ana de Vera, MD
• Phone: +41619068
• Email: ana.devera[@]santhera.com
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

The intent of this protocol is to provide continued access to vamorolone for subjects in the United States who Have Completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.

Description:

Vamorolone will be shipped to the subject's family by the study site. The patient will receive standard of care treatment and procedures for management of DMD. Treating Physicians participating in the expanded access program are required to collect and document any physician, patient, or caregiver reported safety events and report to the Sponsor. The subject's dose of vamorolone may be increased or decreased within a range of 2.0 to 6.0 mg/kg/day (only doses of 2 mg/kg, 4 mg/kg and 6 mg/kg are allowed), given once daily. Administration of vamorolone (taken with an 8 ounce (240 ml) glass of full fat milk, or equivalent high-fat food portion) will be unchanged from the VBP15-LTE, VBP15-004, or VBP15-006 studies. In the absence of safety concerns, and while this Expanded Access protocol isactive, vamorolone may be provided indefinitely or until approval, providedthat the Treating Physician and family agrees that continued administrationof vamorolone is in the best interest of the child.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: Male
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Subject's parent or legal guardian has provided written informed consent/HIPAA authorization
• Subject has previously completed at a participating US or Canada study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, VBP15-006 up to and including the Week 12 assessment
• Subject and parent/guardian are willing and able to comply with recommended study drug administration plan, and standard of care follow-up and monitoring as recommended by their Treating Physician

Exclusion Criteria:

• Subject had a serious or severe adverse event in study VBP15-LTE or VBP15-004 or VBP15-006 that, in the opinion of the Treating Physician and Sponsor, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this expanded access program
• Subject and/or parent/guardian are unable and/or unwilling to comply with regular medical care and follow-up as recommended by their Treating Physician throughout participation in the VBP15-EAP

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Vamorolone
2.0 mg/kg/day, 4.0 mg/kg/day, or 6.0 mg/kg/day at physician discretion

Locations

Country	Name	City	State
Canada	Alberta's Children Hospital	Calgary	Alberta
Canada	The Montreal Children's Hospital	Montréal	Quebec
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario
Canada	The Hospital for Sick Children	Toronto	Ontario
Canada	British Columbia Children's Hospital	Vancouver	British Columbia
Israel	Schneider Children's Medical Center	Petah Tikvah
United States	urie Children's Hospital of Chicago	Chicago	Illinois
United States	University of Texas Southwestern Medical Center	Dallas	Texas
United States	University of California Davis	Davis	California
United States	Duke University	Durham	North Carolina
United States	Nemours Children's Hospital	Orlando	Florida
United States	Seattle Children's	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada,  Israel, 

External Links

•   ReveraGen BioPharma
•   Vamorolone Phase IIa trial in Duchenne Muscular Dystrophy