Alphanate in Immune Tolerance Induction Therapy

Stopped early · Phase 2

Conditions studied: Hemophilia A, Congenital

In brief

This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study, planned in of approximately 25 male participants with congenital hemophilia A who will receive their first (primary) immune tolerance induction (ITI) treatment with alphanate. The study consists of 2 phases: * An ITI Treatment Phase in which all eligible participants will receive ITI treatment with alphanate for a period of up to 33 months. Upon confirmation of complete immune tolerization, participants will then enter a 12-month Prophylactic Phase. If, after 33 months of ITI, a participants has achieved partial immune tolerance, the participants will enter a 12-month Prophylactic Phase. * A 12-month Prophylactic Phase for all participants who meet the criteria for complete or partial success to continue on a prophylactic dosing regimen of alphanate. Due to limited enrollment, this study was early terminated.

Key facts

Study ID

NCT03095287

Run by

Grifols Therapeutics LLC

People needed

2

Starts

2018-01-03

Expected to finish

2020-09-18

Last updated by the study team

2021-11-23

Who can join

Age: any, up to 12. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• The subject has a documented diagnosis of severe congenital hemophilia A with FVIII:C <1% of normal.
• The subject is a male <12 years (and at least 2 years of age if in India) at the Baseline Visit.
• The subject's documented historical peak inhibitor titer is ≥5 BU and ≤200 BU.
• The subject has an inhibitor titer >0.6 BU and <10 BU at Screening.
• The subject has had a delay ≤24 months from the date of diagnosis of the inhibitor to the start of the subject's ITI treatment.

You may not qualify if…

• The subject has acquired factor VIII (FVIII) deficiency.
• The subject has previously received ITI treatment.
• The subject has a recent (within 1 month) history of central line infection at the time of Screening.
• The subject has a high risk of cardiovascular, cerebrovascular, or thromboembolic event as judged by the investigator.
• The subject is currently undergoing treatment with immunosuppressive drugs (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin, interferon, or the use of a protein A column or plasmapheresis and is unwilling to discontinue these treatments starting at the screening visit.
• The subject has a known infection with human immunodeficiency virus (HIV) or has clinical signs and symptoms consistent with current HIV infection.
• The subject has a known previous infection with hepatitis B virus (HBV) or hepatitis C virus (HCV) or has clinical signs and symptoms consistent with current HBV or HCV infection.
• The subject has significant proteinuria, has a history of acute renal failure or severe renal impairment (blood urea nitrogen or creatinine >2 times the upper limit of normal), or is receiving dialysis at Screening.
• The subject has a value of aspartate transaminase or alanine aminotransferase >2 times the upper limit of normal at Screening.
• The subject has clinical evidence of any significant acute or chronic disease that, in the opinion of the investigator, may interfere with successful completion of the trial or place the subject at undue medical risk.
• The subject has a history of anaphylaxis or severe systemic reaction to any plasma derived or other blood products.

Where it is running

• Emory University — Atlanta, Georgia, United States
• University of Kentucky — Lexington, Kentucky, United States
• Childrens Hospital and Clinics of Minnesota — Minneapolis, Minnesota, United States
• The Childrens Mercy Hospital — Kansas City, Missouri, United States
• Robert Wood Johnson Medical Group — New Brunswick, New Jersey, United States
• Newark Beth Israel Medical Center & Children's Hospital of New Jersey — Newark, New Jersey, United States
• University of North Carolina at Chapel Hill, Hemophilia and Thrombosis Center — Chapel Hill, North Carolina, United States
• Seattle Children's Hospital, Seattle Children's Research Institute — Seattle, Washington, United States
• McMaster Children's Hospital — Hamilton, Ontario, Canada
• Lokmanya Tilak Municipal Medical College & General Hospital — Mumbai, Maharashtra, India
• B. J. Govt. Medical College & Sassoon Hospital — Pune, India
• A.O.U. Santa Maria della Misericordia Perugia — Perugia, Umbria, Italy
• Azienda Ospedaliera Universitaria Careggi — Florence, Italy
• Universita degli Studi di Roma La Sapienza — Roma, Italy
• Kemerovo Regional Clinical Hospital — Kemerovo, Russia
• FGUs Hospital - Kirov Scientific Research Institute — Kirov, Russia
• Center for Hemophilia Treatment St.-Petersburg — Saint Petersburg, Russia
• Hospital Universitari i Politecnic La Fe — Valencia, Autonomous Community of Valencia, Spain
• Hospital Universitario La Paz — Madrid, Spain
• Hospital Universitario Virgen del Rocio — Seville, Spain

Full record on ClinicalTrials.gov

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