Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01064284 |
|
Recruitment Status :
Completed
First Posted : February 8, 2010
Results First Posted : April 7, 2017
Last Update Posted : August 25, 2017
|
- Study Details
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in previously untreated patients (PUPs) or minimally blood component-treated (MBCTPs) in the first 50 EDs or in the first 3 years from enrollment, whichever occurs first.
.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia A | Drug: PLASMA DERIVED Factor VIII Drug: Recombinant FVIII | Phase 4 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 303 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Basic Science |
| Official Title: | Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) When Exposed to Plasma-derived Von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical Trial |
| Study Start Date : | January 2010 |
| Actual Primary Completion Date : | May 2015 |
| Actual Study Completion Date : | May 2015 |
| Arm | Intervention/treatment |
|---|---|
|
Active Comparator: PLASMA DERIVED Factor VIII
Plasma-derived vWF/FVIII
|
Drug: PLASMA DERIVED Factor VIII
Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding
Other Name: ALPHANATE |
|
Active Comparator: rFVIII
Recombinant FVIII
|
Drug: Recombinant FVIII
Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding
Other Name: ADVATE |
- To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the First 3 Years From Enrolment, Whichever Comes First in PUPs and MBCTs [ Time Frame: During the first 50 exposure days or first 3 years of enrollment, whichever occurs first ]
Expressed with the numebr of patients for each group who developed FVIII inhibitors.
PUPs: Previously Untreated Patients MBCTPs: Minimally Blood Component-Treated Patients
- To Evaluate the Anamnestic Response of Inhibitor Patients [ Time Frame: During the first 50 exposure days or first 3 years of enrollment, whichever occurs first ]
- To Evaluate the Frequency of Transient Inhibitors [ Time Frame: In the 6 months after inhibitor development ]Number of participants for each group who developed transient inhibitors (this means, those inhibitors which disappeared spontaneously within 6 months without immunotolerance treatment).
- To Evaluate the Modality of Occurrence of Inhibitors (Number of EDs) [ Time Frame: During the first 50 exposure days or first 3 years of enrollment, whichever occurs first ]Number of EDs: Number of Exposure Days (EDs) after which the inhibitors develop
- To Evaluate the Modality of Occurrence of Inhibitors (Titre at Onset) [ Time Frame: During 6 months of observation, from the inhibitor occurrence ]Inhibitor Titre at Onset
- To Evaluate Clinical Factors Potentially Associated to Inhibitor Development [ Time Frame: During the first 50 exposure days or first 3 years of enrollment, whichever occurs first ]
- To Evaluate Laboratory Factors Potentially Associated to Inhibitor Development [ Time Frame: During the first 50 exposure days or first 3 years of enrollment, whichever occurs first ]
- To Evaluate the Incidence of All Other Adverse Events Related and Not Related to the Products Used [ Time Frame: During the first 50 exposure days or first 3 years of enrollment, whichever occurs first ]
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: | 1 Minute to 6 Years (Child) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male subjects
- Any ethnicity
- Age <6 years
-
Severe haemophilia A (FVIII:C <1%), as confirmed at enrolment by the central laboratory.
o Those patients diagnosed locally as severe but subsequently found to have FVIII levels >= 1% on testing at the central laboratory will be separately recorded in the screening list.
-
Previously untreated (0 EDs to any FVIII concentrates or blood products) or minimally treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate.
o Patients not meeting these criteria will be separately recorded in the screening list.
- Negative inhibitor measurement at both local and central laboratory at screening
- Ability to comply with study requirements
- Signed informed consent of legal tutors o Patients who will not accept to enter into the study or to be randomized will be separately recorded.
Exclusion Criteria:
- Previous history of FVIII inhibitor
- Other congenital or acquired bleeding defects
-
Plasma FVIII level >= 1%, as assayed at the central laboratory
o Those patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
- Concomitant congenital or acquired immunodeficiency
- Concomitant treatment with systemic immunosuppressive drugs
- Concomitant treatment with any investigational drug
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): NCT01064284
Show 48 study locations
| Principal Investigator: | Pier M. Mannucci, Professor | Fondazione Ca' Granda Ospedale Maggiore Policlinico Milano | |
| Principal Investigator: | Flora Peyvandi, Professor | Fondazione Ca' Granda Ospedale Maggiore Policlinico Milano |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Fondazione Angelo Bianchi Bonomi |
| ClinicalTrials.gov Identifier: | NCT01064284 |
| Other Study ID Numbers: |
ABB - 09 - 001 2009-011186-88 ( EudraCT Number ) |
| First Posted: | February 8, 2010 Key Record Dates |
| Results First Posted: | April 7, 2017 |
| Last Update Posted: | August 25, 2017 |
| Last Verified: | July 2017 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Individual participant data (IPD) are available to other researchers through the publication of an article. |
|
Hemophilia A Factor VIII inhibitors vWF/FVIII rFVIII |
|
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders |
Hemorrhagic Disorders Genetic Diseases, Inborn Factor VIII Coagulants |