Active not recruitingPHASE3INTERVENTIONAL

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors

This important study is testing a medicine called emicizumab in very young children, from newborns up to 12 months of age, who have severe haemophilia A. Haemophilia A is a condition where blood doesn't clot properly, leading to bleeding. Emicizumab is designed to help blood clot and reduce bleeding episodes. The study wants to find out how effective this medicine is, if it's safe for babies, and how their bodies react to it. Participants will receive the medicine for a year, and then continue to be followed for several more years to see its long-term effects. This research could help improve treatments for babies born with this condition.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Hoffmann-La Roche

Enrolment target

Start

04 Feb 2021

Estimated completion

18 May 2030

Severe Hemophilia A

Results

Results from this study

Posted June 2024

Results have been published for this study.

Primary outcome

Model-Based Annualized Bleeding Rate for Treated Bleeds

The number of treated bleeds over the efficacy period was estimated as an annualized bleeding rate (ABR) using a negative binomial regression model, which accounts for different follow-up times. A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed". The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed. Bleeds due to surgery/procedure were excluded.

Full results on the registry

What is this study about?

This study is focusing on a new way to help babies with severe haemophilia A, a condition that makes them bleed easily. Normally, our blood contains a protein called Factor VIII that helps it clot. In people with severe haemophilia A, there isn't enough of this protein, making them at risk of bleeding into joints, muscles, or even the brain. This study is testing a medicine called emicizumab, which works differently from traditional treatments by helping the blood clotting process happen more easily.

The main goal is to understand if emicizumab is a safe and effective treatment for these very young patients. Researchers will be looking at how well it prevents bleeding episodes, how the babies' bodies handle the medicine, and if there are any side effects. This is a "Phase IIIb" study, which means it's one of the final stages of testing a new medicine before it might be available more widely. The study will last for a year, with participants receiving the medicine regularly, followed by a longer period of up to seven years to see the long-term benefits and safety.

Developing new treatments for babies is crucial because they are at a vulnerable stage, and preventing early bleeds can make a big difference to their health as they grow. This research aims to provide valuable information that could help doctors choose the best care for infants with severe haemophilia A, potentially improving their quality of life by reducing bleeding complications.

Key takeaways

This study is for babies with severe haemophilia A, from birth up to 12 months old.
It tests a medicine called emicizumab to prevent bleeding.
The treatment involves regular injections under the skin.
The study includes a one-year treatment phase followed by up to seven years of monitoring.
Regular clinic visits and blood tests are required to monitor health and treatment effectiveness.

Who may be eligible?

This study is looking for very specific patients. It's for babies who are newborn up to 12 months old when they join, and they must weigh at least 3 kilograms (about 6.6 pounds). They need to have been diagnosed with severe haemophilia A, meaning their bodies produce very little of the important Factor VIII protein. It's also important that they haven't developed 'inhibitors' – these are like antibodies that can stop standard Factor VIII treatments from working.

The study is for babies who haven't had much previous treatment for their haemophilia. This means they should either be completely new to treatment or have only had very limited exposure (up to 5 days) to haemophilia medicines. Babies with other bleeding disorders or those who have had certain other medications are not able to join. All parents or caregivers must be willing and able to follow the study's requirements closely.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your baby 12 months old or younger?
Does your baby weigh at least 3 kilograms (about 6.6 pounds)?
Has your baby been diagnosed with severe haemophilia A?
Has your baby received very little or no previous haemophilia treatment?
Do you have a clear understanding of the study requirements and feel able to commit to them?

Answer every question to see your result.

What does participation involve?

If your baby takes part in this study, they will receive the study medicine, emicizumab, as an injection under the skin every two weeks for the first year. After this first year, they will continue to receive the medicine, but the dose and how often it's given might change (either weekly, every two weeks, or every four weeks) for up to an additional seven years. This means the study could last for a total of eight years.

Throughout the study, your baby will have regular visits to the clinic. At these visits, doctors will check their health, do blood tests to see how the medicine is working and to monitor for any side effects, and track any bleeding episodes. You will also be asked to fill out questionnaires. It's important to attend all scheduled appointments and follow the doctor's instructions carefully.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit of receiving a new treatment that could help prevent bleeding episodes in your baby with severe haemophilia A. However, like all medicines, emicizumab has potential risks and side effects, which will be carefully monitored by the study team. These potential risks will be fully explained to you before you decide to take part. You are free to withdraw your baby from the study at any time, for any reason, without affecting their future medical care.

Locations (32)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Phoenix Children's Hospital
Verified postcode
Phoenix, United States
Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States
University of Colorado Denver, Children's Hospital
Verified postcode
Aurora, United States
Tulane University Health Sciences Center
Verified postcode
New Orleans, United States
University of Michigan
Verified postcode
Ann Arbor, United States
Seattle Children's Hospital
Verified postcode
Seattle, United States
The Children's Hospital at Westmead
Verified postcode
Westmead, Australia
Royal Children's Hospital
Verified postcode
Parkville, Australia
Perth Children's Hospital
Verified postcode
Nedlands, Australia
Medizinische Universität Wien
Verified postcode
Vienna, Austria
Cliniques Universitaires St-Luc
Verified postcode
Brussels, Belgium
UZ Leuven Gasthuisberg
Verified postcode
Leuven, Belgium

Common questions

What is haemophilia A?

Haemophilia A is a genetic condition where a person's blood doesn't clot properly because they don't have enough of a protein called Factor VIII, leading to easy bleeding.

What is emicizumab?

Emicizumab is a medicine designed to help the blood clotting process in people with haemophilia A, aiming to reduce the number of bleeding episodes.

How will my baby receive the medicine?

The medicine, emicizumab, will be given to your baby as an injection just under the skin, similar to a regular jab.

How long will the study last?

The initial treatment period is for one year, but participants will then be followed for up to an additional seven years, making the total study duration up to eight years.

Can we stop participating at any time?

Yes, you have the right to withdraw your baby from the study at any point, and this will not affect their regular medical care.