Hemophilia ANCT03154437
An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
A study testing Emicizumab in people with Hemophilia A, run by Genentech, Inc. at 14 sites in United States.
Trial summary
This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Emicizumab
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 14 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is [i.e.], greater than or equal to [>/=] 5 Bethesda Units)
- History of treatment with episodic or prophylactic bypassing agents for at least the last 24 weeks
- >/=6 (if on an episodic bypassing agent regimen) or >/=2 (if on a prophylactic bypassing agent regimen) bleeds within 24 weeks prior to screening
- Currently using recombinant activated factor VII (rFVIIa) or are willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds
- Adequate hematologic function, defined as platelet count >/= 100,000 per microliters (mcL) and hemoglobin >/=8 grams per deciliter (g/dL) at screening
- Adequate hepatic and renal function
Who cannot join
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy or prophylaxis with FVIII with the exception of participants who have received a treatment regimen of FVIII prophylaxis with concurrent bypassing agent prophylaxis
- Treatment for thromboembolic disease within 12 months before Day 1 (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
- Other conditions (example [e.g.], certain autoimmune diseases) that may increase the risk of bleeding or thrombosis
- High risk for thrombotic microangiopathy (TMA), in the investigator's judgment
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
Show all 9Show fewer
- Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned use during the study, with the exception of antiretroviral therapy
- Treatment with any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Day 1; A non-hemophilia-related investigational drug within the last 30 days or 5 half-lives before Day 1, whichever is longer; An investigational drug concurrently
- Any serious medical condition, treatment, or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in the study
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
University of Colorado Denver, Children's Hospital
Aurora, Colorado, 80045
Boston Childrens Hospital
Boston, Massachusetts, 02115
Rush Medical Center
Chicago, Illinois, 60612
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Texas Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, 75235
University of Texas Medical School
Houston, Texas, 77030
Children's Mercy Hosp Clinics
Kansas City, Missouri, 64108
University of Miami Miller School of Medicine
Miami, Florida, 33136
University of Minnesota
Minneapolis, Minnesota, 55455
Tulane Medical Center; Investigational/Research Pharmacy
New Orleans, Louisiana, 70112
Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
Newark, New Jersey, 07112
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
University of Utah
Salt Lake City, Utah, 84132
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
Seattle, Washington, 98104
About this study
Official title: An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors
This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.
- Conditions
- Hemophilia A
- Being tested
- Emicizumab
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmicizumabBiological
Also called ACE910, CH5534262
Participants will receive emicizumab at a loading dose of 3 milligrams per kilogram (mg/kg) per week subcutaneously (SC) for 4 weeks, followed by a maintenance dose of 1.5 mg/kg per week SC thereafter. Treatment with emicizumab will continue until unacceptable toxicity, withdrawal of consent, participant or physician decision to discontinue treatment, death, the participant is able to obtain commercial drug after emicizumab becomes commercially available, or the sponsor decides to discontinue emicizumab clinical development, whichever occurs first.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Genentech, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Genentech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03154437
- Sponsor's protocol ID
- ML39356
- Status last verified
- Mar 2018
- First submitted
- 12 May 2017
- Last updated
- 29 Mar 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors
- ClinicalTrials.gov ID
- NCT03154437
- Acronym / sponsor ID
- ML39356
- Conditions
- Hemophilia A
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Genentech, Inc.Industry (pharma, biotech, devices)
- First posted
- 16 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.
Study design
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is [i.e.], greater than or equal to [>/=] 5 Bethesda Units)
- History of treatment with episodic or prophylactic bypassing agents for at least the last 24 weeks
- >/=6 (if on an episodic bypassing agent regimen) or >/=2 (if on a prophylactic bypassing agent regimen) bleeds within 24 weeks prior to screening
- Currently using recombinant activated factor VII (rFVIIa) or are willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds
- Adequate hematologic function, defined as platelet count >/= 100,000 per microliters (mcL) and hemoglobin >/=8 grams per deciliter (g/dL) at screening
Key exclusion criteria (9 listed)
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy or prophylaxis with FVIII with the exception of participants who have received a treatment regimen of FVIII prophylaxis with concurrent bypassing agent prophylaxis
- Treatment for thromboembolic disease within 12 months before Day 1 (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
- Other conditions (example [e.g.], certain autoimmune diseases) that may increase the risk of bleeding or thrombosis
- High risk for thrombotic microangiopathy (TMA), in the investigator's judgment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmicizumabAlso: ACE910, CH5534262Participants will receive emicizumab at a loading dose of 3 milligrams per kilogram (mg/kg) per week subcutaneously (SC) for 4 weeks, followed by a maintenance dose of 1.5 mg/kg per week SC thereafter. Treatment with emicizumab will continue until unacceptable toxicity, withdrawal of consent, participant or physician decision to discontinue treatment, death, the participant is able to obtain commercial drug after emicizumab becomes commercially available, or the sponsor decides to discontinue emicizumab clinical development, whichever occurs first. | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Emicizumab in people with Hemophilia A, run by Genentech, Inc. at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2018.