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Long Term Safety Study in Patients With HLH/MAS in Still's Disease Exposed to Gamifant.
Sponsor: Swedish Orphan Biovitrum
Summary
This study aims to evaluate safety information in patients with HLH/MAS in Still's disease exposed to Gamifant.
Official title: Long-term Safety of Gamifant® in Adult and Pediatric Patients With HLH/MAS in Still's Disease With Inadequate Response or Intolerance to Glucocorticoids, or With Recurrent MAS
Key Details
Gender
All
Age Range
Any - 80 Years
Study Type
OBSERVATIONAL
Enrollment
70
Start Date
2026-12
Completion Date
2030-12
Last Updated
2026-11-02
Healthy Volunteers
No
Interventions
Local laboratory testing
Protocol-mandated assessments include specified laboratory testing at baseline and during the initial 12 months of follow-up (Week 1, Week 2, Month 1, Month 3, Month 6, Month 12): complete blood counts, differential leukocyte count, serum biochemistry/liver function tests, CMV PCR. CMV serology is only required (per protocol) to be conducted at baseline. No laboratory testing is required per protocol after the 12-month visit.
Local Lab Tests
Protocol-mandated assessments include specified laboratory testing at baseline and during the initial 12 months of follow-up (Week 1, Week 2, Month 1, Month 3, Month 6, Month 12): complete blood counts, differential leukocyte count, serum biochemistry/liver function tests, CMV PCR. CMV serology is only required (per protocol) to be conducted at baseline. No laboratory testing is required per protocol after the 12-month visit.
Locations (18)
Children's of Alabama
Birmingham, Alabama, United States
Childrens Hospital Los Angeles - RHU
Los Angeles, California, United States
University of Miami
Miami, Florida, United States
John Hopkins all Children's Hospital
St. Petersburg, Florida, United States
Childrens Healthcare of Atlanta
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Mott Children's Hospital
Ann Arbor, Michigan, United States
University of Minnesota Medical Center
Minneapolis, Minnesota, United States
Morgan Stanley Children's Hospital
New York, New York, United States
Atrium Health Levine Childrens Cancer and Blood Disorders a Facility of Carolinas Medical Center
Charlotte, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Childrens Health Specialty Center Dallas
Dallas, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Rheumatology
Seattle, Washington, United States