Trials Sponsors Conditions Drugs Pricing

Conditions

Hemophilia A Severe Hemophilia A

Outcome Measures

Primary Outcomes (4)

Incidence of Inhibitor Formation to FVIII in Previously Treated Patients (PTPs) With 100 Exposure Days (EDs) to CSL627
At the closest visit after 100 EDs (up to 5 years).
Number of Previously Untreated Patients (PUPs) With High-titer Inhibitor Formation to FVIII With at Least 50 EDs to CSL627
High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.
At the closest visit after 50 EDs (up to 5 years).
Percent Treatment Success for Major Bleeding Episodes in PUPs
Percentage of major bleeding episodes treated successfully where treatment success for a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". Major bleeding episodes are defined as bleeding episodes for which a subject is required to seek treatment at the hemophilia center or that threatens the subject's life or loss of limb.
Up to 5 years
Annualized Spontaneous Bleeding Rate in PUPs
The annualized spontaneous bleeding rate for PUPs taking prophylaxis and on-demand treatment regimens.
Up to 5 years

Secondary Outcomes (18)

Percentage of Bleeding Episodes Treated Successfully in PTPs
Up to 5 years
Annualized Bleeding Rate in PTPs and PUPs
Up to 5 years
Percentage of Bleeding Episodes Requiring 1, 2, 3, or > 3 Injections of CSL627 to Achieve Hemostasis in PTPs and PUPs
Up to 5 years
Mean Number of On-demand Infusions of CSL627
Up to 5 years
Mean On-demand Dose Administered of CSL627
Up to 5 years
+13 more secondary outcomes

Study Arms (2)

Previously treated patients (PTPs)

EXPERIMENTAL

The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule for the subject based upon the subject's pharmacokinetic (PK) profile, rVIII-SingleChain PK data, previous FVIII treatment regimen, and bleeding phenotype, if available.

Biological: rVIII-SingleChain

Previously untreated patients (PUPs)

EXPERIMENTAL

The investigator will assign subjects to either prophylaxis or on-demand treatment regimens for rVIII-SingleChain by intravenous injection. The investigator will determine the rVIII-SingleChain dose and dosing schedule at their discretion, taking into consideration the World Federation of Hemophilia (WFH) guidelines, the type of bleeding episode, location of the bleeding, subject's age, and other disease characteristics.

Biological: rVIII-SingleChain

Interventions

rVIII-SingleChainBiological

Recombinant single-chain coagulation factor VIII

Also known as: CSL627

Previously treated patients (PTPs)Previously untreated patients (PUPs)

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

PTPs:
Males of any age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels \< 1%) and who participated in a previous CSL-sponsored clinical study with rVIII-SingleChain.

Sponsors & Collaborators

CSL Behring
Industrylead

Study Sites (64)

Study Site 8400213

San Diego, California, 92103-8651, United States

Study Site 8400241

Aurora, Colorado, 80045, United States

Study Site 8400118

Hartford, Connecticut, 06106, United States

Study Site 8400116

Miami, Florida, 33136, United States

Study Site 8400184

Chicago, Illinois, 60612, United States

Study Site 8400204

New Orleans, Louisiana, 70112, United States

Study Site 8400240

Dallas, Texas, 75390-9016, United States

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title: Clinical Trial Disclosure Manager
Organization: CSL Behring

Study Officials

Program Director
CSL Behring
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Enrollment

246 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 3
Allocation: NON RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

June 23, 2014

First Posted

June 24, 2014

Study Start

October 13, 2014

Date type not reported

This date has not been verified as actual or estimated.

An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (4)

Secondary Outcomes (18)

Study Arms (2)

Previously treated patients (PTPs)

Previously untreated patients (PUPs)

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (64)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations

An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (4)

Secondary Outcomes (18)

Study Arms (2)

Previously treated patients (PTPs)

Previously untreated patients (PUPs)

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (64)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Results Point of Contact

Study Officials

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Documents

Locations