Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03754790 |
Recruitment Status :
Recruiting
First Posted : November 27, 2018
Last Update Posted : March 3, 2021
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Primary Objective:
To characterize the long-term safety and tolerability of fitusiran
Secondary Objectives:
-
To characterize the long-term efficacy of fitusiran as assessed by the frequency of:
- Bleeding episodes
- Spontaneous bleeding episodes
- Target joint bleeding episodes
- To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hemophilia | Drug: Fitusiran | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 244 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX |
Actual Study Start Date : | January 9, 2019 |
Estimated Primary Completion Date : | January 2026 |
Estimated Study Completion Date : | January 2026 |

Arm | Intervention/treatment |
---|---|
Experimental: Fitusiran
Participants will be administered fitusiran as an subcutaneous injection once monthly or every other month for 48 months
|
Drug: Fitusiran
Pharmaceutical form:solution for injection Route of administration: subcutaneous |
- Number of patients with adverse events [ Time Frame: From day -30 (screening) to day 673 ]
- Annualized bleeding rate (ABR) [ Time Frame: From day 29 to day 673 ]Annualized bleeding rate (ABR) in the treatment period
- Annualized spontaneous bleeding rate [ Time Frame: From day 29 to day 673 ]Annualized spontaneous bleeding rate in the treatment period
- Annualized joint bleeding rate [ Time Frame: From day 29 to day 673 ]Annualized joint bleeding rate in the treatment period
- Changes in Haem-A-quality of life (QoL) score [ Time Frame: On day 1 (baseline), day 169, day 337, day 505 and day 673 ]Change in Haem A QoL physical health score and total score in the treatment period (in participants ≥17 years of age) from baseline (day 1) to day 169 (post-restart for patients on the modified dosing regimen), day 337, day 505 and day 673

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion criteria :
- Participant must be at least 12 years of age inclusive, at the time of signing the informed consent
- Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial
- Male
- Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative
Exclusion criteria:
- Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis
- Current participation in immune tolerance induction treatment (ITI)
- Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes
- Use of compounds other than factor concentrates or BPAs for hemophilia treatment
- Current or prior participation in a gene therapy trial
- Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) >1.5 × upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST > 5 x ULN for patients who were in the fitusiran arm in the parent study
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03754790
Contact: Trial Transparency email recommended (Toll free number for US & Canada) | 800-633-1610 ext 1 then # | Contact-US@sanofi.com |

Study Director: | Clinical Sciences & Operations | Sanofi |
Responsible Party: | Genzyme, a Sanofi Company |
ClinicalTrials.gov Identifier: | NCT03754790 |
Other Study ID Numbers: |
LTE15174 2018-002880-25 ( EudraCT Number ) U1111-1210-0018 ( Other Identifier: UTN ) |
First Posted: | November 27, 2018 Key Record Dates |
Last Update Posted: | March 3, 2021 |
Last Verified: | March 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/ |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | No |
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |