Have you or your loved ones been diagnosed with hemophilia a?

You may be eligible to participate in a hemophilia a clinical trial.

Have you or your loved ones been diagnosed with hemophilia a? You may be eligible to participate in a hemophilia a clinical trial.

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Hemophilia A Clinical Trial

NCT02172950 | Phase 3 | Interventional

Have you or your loved ones been diagnosed with hemophilia a?

You may be eligible to participate in a hemophilia a clinical trial.

Have you or your loved ones been diagnosed with hemophilia a? You may be eligible to participate in a hemophilia a clinical trial.

Active not recruiting

Male

All ages

This study has recruited 245 Participants

This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII‑SingleChain for prophylaxis and on‑demand treatment of bleeding episodes in 50 previously untreated patients (PUPs) with no previous exposure to any FVIII product who achieve at least 50 EDs to rVIII-SingleChain in this study. A substudy (open to both PTPs and PUPs) will investigate the use of rVIII-SingleChain in surgery. A substudy (open to PUPs who develop an inhibitor to rVIII-SingleChain) will investigate the use of rVIII-SingleChain in immune tolerance induction (ITI) therapy.

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Available locations

Study Site 7240008

Active not recruiting A Coruna, ESP 15006

Study Site 5280006

Active not recruiting Amsterdam Zuidoost, NLD 1105 AZ

Study Site 8400241

Active not recruiting Aurora, CO, USA 80045

Study Site 7640001

Active not recruiting Bangkok, THA 10330

Study Site 7640005

Active not recruiting Bangkok, THA 10700

Study Site 7240021

Active not recruiting Barcelona, ESP 08035

Study Site 4220007

Active not recruiting Beirut, LBN 165191

Study Site 2760034

Active not recruiting Bonn, DEU 53127

Study Site 2500015

Active not recruiting Brest, FRA 29609

View all 65 locations

Details for the study

Brief Title

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

Official Title

A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects With Severe Hemophilia A

Brief Summary

This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs) with severe congenital hemophilia A and previous exposure to FVIII products who achieve at least 100 exposure days (EDs) to rVIII-SingleChain in this study, as well as in previously untreated patients (PUPs) with no previous exposure to any FVIII product who achieve at least 50 EDs to rVIII-SingleChain in this study. A substudy (open to both PTPs and PUPs) will investigate the use of rVIII-SingleChain in surgery. A substudy (open to PUPs who develop an inhibitor to rVIII-SingleChain) will investigate the use of rVIII-SingleChain in immune tolerance induction (ITI) therapy.

Treatments and/or Procedures

rVIII SingleChain

Recombinant single-chain coagulation factor VIII

Outcome Measures

Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.

Primary

Incidence of inhibitor formation to FVIII in PTPs with 100 EDs to rVIII-SingleChain

Number of PTPs (with 100 EDs to rVIII-SingleChain) with inhibitor formation to FVIII

At the closest visit after 100 EDs (up to 5 years).

Primary

Incidence of high-titer inhibitor formation to FVIII in PUPs with at least 50 EDs of rVIII-SingleChain

Number of PUPs (with at least 50 EDs of rVIII-SingleChain) with high-titer inhibitor formation to FVIII. 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).

Primary

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

Primary

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

Treatment success in PTPs

Up to 5 years

Secondary

Consumption of rVIII-SingleChain in PTPs and PUPs - injections

The number of rVIII-SingleChain injections per month and per year

Up to 5 years

Secondary

Consumption of rVIII-SingleChain in PTPs and PUPs - IU/kg

rVIII-SingleChain consumption (IU/kg) per month and per year, and per event during prophylaxis, on-demand, and surgery.

Up to 5 years

Secondary

Percentage of PUPs with clinically significant abnormal vital signs values after first rVIII-SingleChain injection

Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator

At 1, 2, 3, and 6 hours after first rVIII-SingleChain injection

Secondary

Treatment success for non-major bleeding episodes in PUPs

Percentage of 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". Non-major bleeding episodes are those not requiring treatment at the hemophilia center or not threatening subject's life or loss of limb.

Up to 5 years

Secondary

Incidence of total inhibitor formation to FVIII in PUPs

Number of PUPs (after 10 and 50 EDs of rVIII-SingleChain) with inhibitor formation (both low- and high-titer inhibitors) to FVIII.

At the closest visit after 10 and after 50 EDs (up to 5 years)

Secondary

Incidence of low-titer inhibitor formation to FVIII in PUPs

Number of PUPs (after 10 and 50 EDs of rVIII-SingleChain) with low-titer inhibitor formation to FVIII. Low-titer inhibitor is defined as an inhibitor titer of less than 5 Bethesda units/mL.

At the closest visit after 10 and after 50 EDs (up to 5 years)

Secondary

Incidence of high-titer inhibitor formation to FVIII in PUPs after 10 EDs with rVIII-SingleChain

Number of PUPs (after 10 EDs of rVIII-SingleChain) with high-titer inhibitor formation to FVIII. High-titer inhibitor is defined as an inhibitor titer of ≥ 5 Bethesda units/mL.

At the closest visit after 10 EDs

Secondary

Annualized bleeding rate in PTPs and PUPs

The annualized bleeding rate for PTPs and PUPs taking prophylaxis and on-demand treatment regimens

Up to 5 years

Secondary

Percentage of PUPs with treatment-emergent clinically significant abnormal vital signs values

Vital signs assessments include heart rate, blood pressure, and body temperature. Clinical significance of an abnormality will be assessed by the investigator

Up to 5 years

Secondary

Hemostatic efficacy of rVIII-SingleChain for PTPs and PUPs who undergo surgery

The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response".

From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)

Study Criteria

Inclusion Criteria:

PTPs (previously treated patients)

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.
Males up to 65 years of age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%), who have at least 50 EDs to any FVIII product, and who are not currently enrolled in a CSL-sponsored clinical study with rVIII-SingleChain.

PUPs (previously untreated patients)

Males up to 18 years of age who have been diagnosed with severe congenital hemophilia A (FVIII activity levels < 1%).
No prior exposure to any Factor VIII product (with the exception of short-term use of blood products).

ITI substudy (immune tolerance induction)

PUPs (previously untreated patients) who have developed a confirmed inhibitor to rVIII-SingleChain in the main study.

Exclusion Criteria:

Known or suspected hypersensitivity to rVIII‑SingleChain or to any excipients of rVIII‑SingleChain or Chinese hamster ovary (CHO) proteins.
Currently receiving a therapy not permitted during the study.
Serum creatinine > 2 x upper limit of normal, alanine aminotransferase or aspartate aminotransferase > 5 x upper limit of normal at Screening (if specified).
Any first-order family (eg, siblings) history of FVIII inhibitors.
For PTPs (previously treated patients) not rolling over directly from a CSL-sponsored clinical study with rVIII-SingleChain: any history of or current FVIII inhibitors.

Have you or your loved ones been diagnosed with hemophilia a?

Hemophilia A Clinical Trial

Have you or your loved ones been diagnosed with hemophilia a?

Available locations

Details for the study

Brief Title

Official Title

Brief Summary

Treatments and/or Procedures

Outcome Measures

Study Criteria

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