NCT01775618

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

Completed Phase 3 Last updated 21 August 2020

What this trial tests

Phase 3 trial testing BAY94-9027 in Hemophilia A in 73 participants. Completed in 19 February 2020.

Timeline

29 May 2013

Primary endpoint
19 March 2015

19 February 2020

Quick facts

Lead sponsor	Bayer
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	73
Start date	29 May 2013
Primary completion	19 March 2015
Estimated completion	19 February 2020
Sites	37 locations across Italy, New Zealand, Netherlands, Greece, Belgium, Austria, United Kingdom, Israel

Drugs / interventions tested

• BAY94-9027 — full drug profile →
• BAY94-9027 — full drug profile →
• BAY94-9027 — full drug profile →

Conditions studied

• Hemophilia A — all drugs for Hemophilia A →

Sponsor

Bayer — full company profile →

Who can join

Under 12, male only, with Hemophilia A. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.

In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, \< 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.

Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Outcome of Clinical Trials with New Extended Half-Life FVIII/IX Concentrates.
   Mancuso ME, Santagostino E. · · 2017 · cited 72× · PMID 28350322 · DOI 10.3390/jcm6040039
2. PROTECT VIII Kids: BAY 94-9027 (PEGylated Recombinant Factor VIII) safety and efficacy in previously treated children with severe haemophilia A.
   Santagostino E, Kenet G, Fischer K, Biss T, et al · · 2020 · cited 24× · PMID 32212300 · DOI 10.1111/hae.13963
3. PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A.
   Mancuso ME, Biss T, Fischer K, Maas Enriquez M, et al · · 2021 · cited 12× · PMID 33724632 · DOI 10.1111/hae.14294
4. Favorable Pharmacokinetic Characteristics of Extended-Half-Life Recombinant Factor VIII BAY 94-9027 Enable Robust Individual Profiling Using a Population Pharmacokinetic Approach.
   Solms A, Iorio A, Ahsman MJ, Vis P, et al · · 2020 · cited 11× · PMID 31749076 · DOI 10.1007/s40262-019-00832-7
5. Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review.
   Reding MT, Lalezari S, Kenet G, Di Minno G, et al · · 2024 · cited 4× · PMID 39162954 · DOI 10.1007/s40268-024-00481-7
6. Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to &lt; 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main Study.
   Ozelo MC, Luciani M, Glosli H, Kavakli K, et al · · 2026 · cited 1× · PMID 41486550 · DOI 10.1111/ejh.70059
7. A post hoc analysis of PROTECT VIII kids assessing long-term efficacy and safety of damoctocog alfa pegol in adolescents with severe haemophilia A.
   Ahuja S, Biss T, Maas Enriquez M, Mancuso ME, et al · · 2024 · cited 1× · PMID 38193596 · DOI 10.1111/ejh.14167