Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)
RESIST EXP
Immune Tolerance Induction Study in Patients With Severe Type A Haemophilia With Inhibitor After Failure of a Previous Induction of Immune Tolerance With FVII Concentrates Without Von Willebrand Factor Rescue
2 other identifiers
interventional
3
1 country
1
Brief Summary
The purpose of this study is to evaluate whether a concentrate containing both FVIII and von Willebrand Factor (VWF) given at a high dose will induce immune tolerance in subjects who have already experienced and failed ITI with VWF-free FVIII concentrates. The treatment on this study is expected to last up to 33 months.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Nov 2009
Longer than P75 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 3, 2009
CompletedFirst Submitted
Initial submission to the registry
January 15, 2010
CompletedFirst Posted
Study publicly available on registry
January 18, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 21, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
October 21, 2020
CompletedAugust 23, 2021
December 1, 2020
11 years
January 15, 2010
August 18, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Achievement of an inhibitor titer of <0.6 BU/failure to achieve an inhibitor titer of <0.6 BU within 33 months, or failure to decrease inhibitor titer by at least 20% compared to titer in prior 6 months, beginning at 3 months after starting ITI
Primary end point is the achievement of an inhibitor titer of less than 0.6 BU or failure to achieve an inhibitor titer of less than 0.6 BU within 33 months of treatment, or failure to decrease the inhibitor titer by at least 20% compared to the titer in the prior 6 months, beginning at 3 months after starting ITI
3 years
Secondary Outcomes (3)
Time to achieve success- either partial or complete.
33 months
Safety - assessment of adverse events through treatment and compliance with prolonged regimen.
33 months
Cost of care.
Up to 45 months
Study Arms (1)
Factor VIII and von Willebrand Factor
EXPERIMENTALInterventions
200 IU/Kg by one or two bolus injections daily. After successful confirmation the dose will be tailed off progressively until discontinuation. Patients will be treated with a VWF/FVIII concentrates according to physician/patients preference.
Eligibility Criteria
You may qualify if:
- severe hemophilia A (FVIII\<1%) with high responding inhibitors (peak levels \>5 BU)
- male, any age;
- any inhibitor level at study enrollment;
- ability and willingness to participate to the study;
- previous ITI course of at least 9 months with a VWF-free FVIII concentrate at any dosage, such as recombinant FVIII and/or monoclonally purified FVIII.
You may not qualify if:
- concomitant systemic treatment with immunosuppressive drugs;
- concomitant experimental treatment;
- previous history of myocardial infarction and/or cerebral stroke
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- City of Hope Medical Centerlead
- Charta Foundationcollaborator
- Grifols Biologicals, LLCcollaborator
- CSL Behringcollaborator
- Biotest Pharmaceuticals Corporationcollaborator
- Grifols Therapeutics LLCcollaborator
Study Sites (1)
City of Hope Medical Center
Duarte, California, 91010, United States
Related Publications (3)
Berntorp E, Ekman M, Gunnarsson M, Nilsson IM. Variation in factor VIII inhibitor reactivity with different commercial factor VIII preparations. Haemophilia. 1996 Apr;2(2):95-9. doi: 10.1111/j.1365-2516.1996.tb00022.x.
PMID: 27214015BACKGROUNDKreuz W, Steiner J, Auerswald G, Beeg T, Becker S. Successful immunetolerance therapy of FVIII-inhibitor in chldren after changing from high to intermediate purity FVIII concentrate. Ann Hematol 1996; 72 (suppl 1).
BACKGROUNDGringeri A, Musso R, Mazzucconi MG, Piseddu G, Schiavoni M, Pignoloni P, Mannucci PM; RITS-FITNHES Study Group. Immune tolerance induction with a high purity von Willebrand factor/VIII complex concentrate in haemophilia A patients with inhibitors at high risk of a poor response. Haemophilia. 2007 Jul;13(4):373-9. doi: 10.1111/j.1365-2516.2007.01484.x.
PMID: 17610550BACKGROUND
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Nadia P. Ewing, MD
Clinical Professor of Pediatrics, City of Hope National Medical Center, Dept. of Pediatrics, 1500 E. Duarte Rd. Duarte, CA 91010
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 15, 2010
First Posted
January 18, 2010
Study Start
November 3, 2009
Primary Completion
October 21, 2020
Study Completion
October 21, 2020
Last Updated
August 23, 2021
Record last verified: 2020-12