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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)

Primary Purpose

Hemophilia A, Hemophilia B

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

fitusiran

Bypassing agents

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring RNAi therapeutic, Hemophilia A, Hemophilia B, Hemophilia A, Severe, Hemophilia B, Severe, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Factor VIII, Factor IX, Inhibitor, Bypassing agents, Coagulants, Fitusiran

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Males, greater than or equal to (>=) 12 years of age.
Severe hemophilia A or B with inhibitors.
- (Severity confirmed by a central laboratory where coagulation factor VIII (FVIII) level was less than (<)1% or factor IX (FIX) level was less than or equal to [<=]2% at Screening; Inhibitors defined as inhibitor titer of >=0.6 Bethesda units per milliliter [BU/mL] or as evidenced by medical records).
A minimum of 6 bleeding episodes requiring BPA treatment within the last 6 months prior to screening.
Willing and able to comply with the study requirements and to provide written informed consent and assent.

Exclusion Criteria:

Known co-existing bleeding disorders other than hemophilia A or B.
Antithrombin (AT) activity <60% at Screening.
Co-existing thrombophilic disorder.
Clinically significant liver disease.
Active hepatitis C virus infection.
HIV positive with a cluster of differentiation-4 count of <200 cells/microliter.
History of arterial or venous thromboembolism.
Inadequate renal function.
History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine.
History of intolerance to SC injection(s).
Any other conditions or comorbidities that would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement.

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Experimental

Arm Label

Bypassing Agents (BPA) On-demand

Fitusiran 80 mg Prophylaxis

Arm Description

Participants received On-demand BPAs (use of these agents, as needed, for episodic bleeding episodes, and not on a regular regimen intended to prevent spontaneous bleeding) per Investigator discretion from Day 1 for treatment of breakthrough bleeding episodes, up to a total of 9 months.

Participants received Fitusiran 80 mg subcutaneously (SC) as prophylaxis once monthly from Day 1, along with the on-demand BPAs (per investigator's discretion and within bleeding dosing guidelines) for treatment of breakthrough bleeding episodes, up to a total of 9 months.

Outcomes

Primary Outcome Measures

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

ABR for a participant during efficacy period (EP) was defined as annualized number of bleeding episodes during EP annualized to a 1-year interval of time. Treated Bleeding episode: any occurrence of hemorrhage that required administration of BPA or factor. It started from first sign of a bleed and ended no more than 72 hours after last treatment for bleed, within which any symptoms of bleeding at same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. EP was defined as time duration starting from Day 29 when antithrombin (AT) lowering capacity of fitusiran had achieved therapeutic target range to the earliest of (Day 246 or last day of bleeding follow up) (maximum duration of EP: from Day 29 to Day 246). This outcome measure (OM) represents estimated results (i.e., results received by applying negative binomial [NB] regression model on data collected during EP).

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

ABR for a participant during EP was defined as annualized number of bleeding episodes during EP annualized to a 1-year interval of time. ABR= number of bleeding episodes during EP divided by total number of days during EP*365.25. A bleeding episode was defined as any occurrence of hemorrhage that required administration of BPA or factor. It started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. EP was defined as time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of (Day 246 or the last day of bleeding follow up) (maximum duration of EP: from Day 29 to Day 246). This OM represents observed results (i.e., descriptive statistics values based on the data which was collected during EP).

Secondary Outcome Measures

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

ABR for a participant during treatment period (TP) was defined as annualized number of bleeding episodes during TP annualized to a 1-year interval of time. Bleeding episode: any occurrence of hemorrhage that required administration of BPA or factor. It started from first sign of a bleed and ended no more than 72 hours after last treatment for bleed, within which any symptoms of bleeding at same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. TP was sum of onset period (first 28 days after first dose of fitusiran) and EP (starting on Day 29 when AT lowering capacity of fitusiran had achieved therapeutic target range to earliest of [Day 246 or last day of bleeding follow up])(maximum duration of TP: from Day 1 to Day 246). This OM represents estimated results (i.e., results received by applying NB regression model on data collected during TP).

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

ABR for a participant during TP was defined as annualized number of bleeding episodes during TP annualized to a 1-year interval of time. ABR= number of bleeding episodes during TP divided by total number of days during TP*365.25. Bleeding episode: any occurrence of hemorrhage that required administration of BPA or factor. It started from the first sign of a bleed and ended no more than 72 hours after last treatment for bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. TP was sum of onset period (first 28 days after the first dose of fitusiran) and EP (starting on Day 29 when AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of [Day 246 or the last day of bleeding follow up]) (maximum duration of TP: from Day 1 to Day 246). This OM represents observed results (i.e., descriptive statistics values based on data collected during TP).

Estimated Annualized Spontaneous Bleeding Rate for Treated Bleeds During Efficacy Period

Annualized spontaneous bleeding rate for a participant during EP was defined as annualized number of spontaneous bleeding episodes during EP annualized to a 1-year interval of time. Spontaneous bleeding episode was bleeding event that occurred for no apparent or known reason, particularly into joints, muscles, and soft tissues. It started from first sign of a bleed and ended no more than 72 hours after last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. EP was defined as time duration starting from Day 29 when AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of (Day 246 or last day of bleeding follow up) (maximum duration of EP: from Day 29 to Day 246). This OM represents estimated results (i.e., results received by applying NB regression model on data collected during EP).

Observed Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period

ABR for participant during EP was defined as annualized number of spontaneous bleeding episodes during EP annualized to a 1-year interval of time. ABR=number of treated spontaneous bleeding episodes during EP divided by total number of days during EP*365.25. Spontaneous bleeding episode was bleeding event that occurred for no apparent or known reason, into joints, muscles, and soft tissues. It started from the first sign of a bleed and ended no more than 72 hours after the last treatment for bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. EP: time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to earliest of (Day 246 or the last day of bleeding follow up) (maximum duration of EP: from Day 29 to Day 246). This OM represents observed results (i.e., descriptive statistics values based on data collected during EP).

Estimated Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Annualized joint bleeding rate for a participant during EP was defined as annualized number of bleeding episodes during EP annualized to a 1-year interval of time. Joint bleeding episode was characterized by an unusual sensation in the joint ("aura") in combination with 1) increasing swelling or warmth over the skin, joint, 2) increased pain, or 3) progressive loss of range of motion or difficulty in using the limb as compared with Baseline. It started from first sign of a bleed and ended no more than 72 hours after last treatment for the bleed. EP was defined as time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of (Day 246 or the last day of bleeding follow up) (maximum duration of EP: from Day 29 to Day 246). This OM presents estimated results (i.e., results received by applying NB regression model on data collected during EP).

Observed Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Annualized joint bleeding rate for participant during EP was defined as annualized number of joint bleeding episodes during EP annualized to 1-year interval of time. ABR= number of treated joint bleeding episodes during EP divided by total number of days during EP*365.25. A joint bleeding episode was characterized by an unusual sensation in joint ("aura") in combination with 1) increasing swelling or warmth over skin, joint, 2) increased pain, or 3) progressive loss of range of motion or difficulty in using limb as compared with Baseline. It started from first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed. EP: time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of (Day 246 or last day of bleeding follow up) (maximum duration of EP: from Day 29 to Day 246). This OM presents observed results (i.e., descriptive statistics values based on data collected during EP).

Health-related Quality of Life (HRQOL): Change From Baseline in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Domain Score at Month 9

Haem-A-QoL: participant-reported questionnaire designed for adult participants (>=17 years of age) with hemophilia; and consisted of 46 items comprising 10 domains (physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality). Items were rated along five response options: never, rarely, sometimes, often, or all the time. Change from baseline in physical Health domain score was reported in this outcome measure. Raw score for physical health domain were transformed to a scale ranged from 0 to 100, where lower scores denoted better physical health.

Health-related Quality of Life (HRQOL): Change From Baseline in Haem-A-QOL Total Score at Month 9

Haem-A-QoL: participant-reported questionnaire designed for adult participants (>=17 years of age) with hemophilia; and consisted of 46 items comprising 10 domains (physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality). Items were rated along five response options: never, rarely, sometimes, often, or all the time. Raw score for each domain were transformed to a scale ranged from 0 to 100, where lower scores denoted better health. Haem-A-QoL Total Score was average of all domain scores and ranged from 0 to 100, where lower scores denoted better quality of life.

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Onset Period

ABR was annualized number of bleeding episodes during onset period per participant annualized to a 1-year interval of time. A treated bleeding episode was defined as any occurrence of hemorrhage that may required administration of BPA or factor. It started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. The onset period was defined as time interval from Day 1 to the earlier of Day 28 or the last day of bleeding follow up. This OM represents estimated results (i.e., results received by applying NB regression model on data collected during onset period).

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

An adverse Event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Treatment emergent AEs were defined as any AE with onset date after first dose of fitusiran in the fitusiran prophylaxis arm or after Day 1 visit in the BPA on-demand arm. A Serious AE (SAE) was defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability or incapacity, was a congenital anomaly or birth defect, or was a medically important event.

Full Information

NCT ID

NCT03417102

First Posted

January 25, 2018

Last Updated

March 15, 2022

Sponsor

Genzyme, a Sanofi Company

1. Study Identification

Unique Protocol Identification Number

NCT03417102

Brief Title

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

Acronym

ATLAS-INH

Official Title

ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, With Inhibitory Antibodies to Factor VIII or IX

Study Type

Interventional

2. Study Status

Record Verification Date

March 2022

Overall Recruitment Status

Completed

Study Start Date

February 14, 2018 (Actual)

Primary Completion Date

November 25, 2020 (Actual)

Study Completion Date

June 23, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Genzyme, a Sanofi Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular medication. In addition, the study assessed safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).

Detailed Description

The duration of treatment with fitusiran was 9 months. The estimated total time on study, inclusive of screening, for each participant was up to 11 months for all participants who enroll in the extension study and participants in the on-demand arm who did not enroll in the extension study. The estimated total time on study was up to 17 months in fitusiran treatment arm participants who did not enroll in the extension study due to the requirement for up to an additional 6 months of follow-up monitoring for antithrombin levels.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A, Hemophilia B

Keywords

RNAi therapeutic, Hemophilia A, Hemophilia B, Hemophilia A, Severe, Hemophilia B, Severe, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Factor VIII, Factor IX, Inhibitor, Bypassing agents, Coagulants, Fitusiran

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

60 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Bypassing Agents (BPA) On-demand

Arm Type

Active Comparator

Arm Description

Arm Title

Fitusiran 80 mg Prophylaxis

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

fitusiran

Intervention Description

solution for injection; by subcutaneous (SC) injection

Intervention Type

Drug

Intervention Name(s)

Bypassing agents

Intervention Description

solution for injection; by intravenous (IV) injection

Primary Outcome Measure Information:

Title

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Secondary Outcome Measure Information:

Title

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

Description

Time Frame

From Day 1 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

Description

Time Frame

From Day 1 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Estimated Annualized Spontaneous Bleeding Rate for Treated Bleeds During Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Estimated Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Health-related Quality of Life (HRQOL): Change From Baseline in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Domain Score at Month 9

Description

Time Frame

Baseline (Day 1), Month 9

Title

Health-related Quality of Life (HRQOL): Change From Baseline in Haem-A-QOL Total Score at Month 9

Description

Haem-A-QoL: participant-reported questionnaire designed for adult participants (>=17 years of age) with hemophilia; and consisted of 46 items comprising 10 domains (physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality). Items were rated along five response options: never, rarely, sometimes, often, or all the time. Raw score for each domain were transformed to a scale ranged from 0 to 100, where lower scores denoted better health. Haem-A-QoL Total Score was average of all domain scores and ranged from 0 to 100, where lower scores denoted better quality of life.

Time Frame

Baseline (Day 1), Month 9

Title

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Onset Period

Description

Time Frame

From Day 1 up to Day 28 or up to the last day of bleeding follow up (any day up to Day 28), whichever was the earliest

Title

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

Description

Time Frame

From Baseline (Day 1) up to 15 months (i.e. 9 months treatment period + 6-months follow-up)

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Males, greater than or equal to (>=) 12 years of age. Severe hemophilia A or B with inhibitors. (Severity confirmed by a central laboratory where coagulation factor VIII (FVIII) level was less than (<)1% or factor IX (FIX) level was less than or equal to [<=]2% at Screening; Inhibitors defined as inhibitor titer of >=0.6 Bethesda units per milliliter [BU/mL] or as evidenced by medical records). A minimum of 6 bleeding episodes requiring BPA treatment within the last 6 months prior to screening. Willing and able to comply with the study requirements and to provide written informed consent and assent. Exclusion Criteria: Known co-existing bleeding disorders other than hemophilia A or B. Antithrombin (AT) activity <60% at Screening. Co-existing thrombophilic disorder. Clinically significant liver disease. Active hepatitis C virus infection. HIV positive with a cluster of differentiation-4 count of <200 cells/microliter. History of arterial or venous thromboembolism. Inadequate renal function. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine. History of intolerance to SC injection(s). Any other conditions or comorbidities that would make the participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Sciences & Operations, MD

Organizational Affiliation

Sanofi

Official's Role

Study Director

Facility Information:

Facility Name

Investigational Site Number 0117

City

Phoenix

State/Province

Arizona

ZIP/Postal Code

85016

Country

United States

Facility Name

Investigational Site Number 0139

City

Los Angeles

State/Province

California

ZIP/Postal Code

90027

Country

United States

Facility Name

Investigational Site Number 0135

City

Orange

State/Province

California

ZIP/Postal Code

92868

Country

United States

Facility Name

Investigational Site Number 0137

City

San Diego

State/Province

California

ZIP/Postal Code

92123

Country

United States

Facility Name

Investigational Site Number 0128

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32610

Country

United States

Facility Name

Investigational Site Number 0115

City

Jacksonville

State/Province

Florida

ZIP/Postal Code

32207

Country

United States

Facility Name

Investigational Site Number 0105

City

Miami

State/Province

Florida

ZIP/Postal Code

33136

Country

United States

Facility Name

Investigational Site Number 0103

City

Tampa

State/Province

Florida

ZIP/Postal Code

33607

Country

United States

Facility Name

Investigational Site Number 0119

City

New Orleans

State/Province

Louisiana

ZIP/Postal Code

70112

Country

United States

Facility Name

Investigational Site Number 0136

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Investigational Site Number 0111

City

Las Vegas

State/Province

Nevada

ZIP/Postal Code

89135

Country

United States

Facility Name

Investigational Site Number 0104

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104-5127

Country

United States

Facility Name

Investigational Site Number 6101

City

Camperdown

ZIP/Postal Code

2050

Country

Australia

Facility Name

Investigational Site Number 6104

City

Clayton

ZIP/Postal Code

3168

Country

Australia

Facility Name

Investigational Site Number 1102

City

Montreal

ZIP/Postal Code

H1T 2M4

Country

Canada

Facility Name

Investigational Site Number 8604

City

Beijing

ZIP/Postal Code

100045

Country

China

Facility Name

Investigational Site Number 8602

City

Guangzhou

ZIP/Postal Code

510515

Country

China

Facility Name

Investigational Site Number 8605

City

Hangzhou

ZIP/Postal Code

310003

Country

China

Facility Name

Investigational Site Number 8603

City

Shanghai

ZIP/Postal Code

200025

Country

China

Facility Name

Investigational Site Number 8601

City

Tianjin

ZIP/Postal Code

300020

Country

China

Facility Name

Investigational Site Number 3303

City

Lyon

ZIP/Postal Code

69677

Country

France

Facility Name

Investigational Site Number 3301

City

Rouen

ZIP/Postal Code

76038

Country

France

Facility Name

Investigational Site Number 4905

City

Frankfurt Am Main

ZIP/Postal Code

60590

Country

Germany

Facility Name

Investigational Site Number 4906

City

Leipzig

ZIP/Postal Code

4103

Country

Germany

Facility Name

Investigational Site Number 9102

City

Bangalore

ZIP/Postal Code

560034

Country

India

Facility Name

Investigational Site Number 9108

City

India

Country

India

Facility Name

Investigational Site Number 9104

City

Jaipur

ZIP/Postal Code

302017

Country

India

Facility Name

Investigational Site Number 9106

City

Lucknow

ZIP/Postal Code

226003

Country

India

Facility Name

Investigational Site Number 9103

City

Pune

ZIP/Postal Code

411001

Country

India

Facility Name

Investigational Site Number 9111

City

Pune

ZIP/Postal Code

411004

Country

India

Facility Name

Investigational Site Number 9105

City

Vellore

ZIP/Postal Code

632004

Country

India

Facility Name

Investigational Site Number 3901

City

Florence

ZIP/Postal Code

50134

Country

Italy

Facility Name

Investigational Site Number 3904

City

Padua

ZIP/Postal Code

35128

Country

Italy

Facility Name

Investigational Site Number 8110

City

Japan

Country

Japan

Facility Name

Investigational Site Number 8103

City

Kita Kyushu-Shi

Country

Japan

Facility Name

Investigational Site Number 8202

City

Daejeon

ZIP/Postal Code

35233

Country

Korea, Republic of

Facility Name

Investigational Site Number 8203

City

Seoul

ZIP/Postal Code

3722

Country

Korea, Republic of

Facility Name

Investigational Site Number 8204

City

Seoul

Country

Korea, Republic of

Facility Name

Investigational Site Number 6003

City

Kota Kinabalu

ZIP/Postal Code

88586

Country

Malaysia

Facility Name

Investigational Site Number 6004

City

Malaysia

Country

Malaysia

Facility Name

Investigational Site Number 2701

City

Parktown

ZIP/Postal Code

2193

Country

South Africa

Facility Name

Investigational Site Number 2703

City

Polokwane

ZIP/Postal Code

699

Country

South Africa

Facility Name

Investigational Site Number 2702

City

Port Elizabeth

ZIP/Postal Code

6001

Country

South Africa

Facility Name

Investigational Site Number 3402

City

Madrid

ZIP/Postal Code

28046

Country

Spain

Facility Name

Investigational Site Number 8803

City

Changhua

ZIP/Postal Code

500

Country

Taiwan

Facility Name

Investigational Site Number 8801

City

Taipei

ZIP/Postal Code

110

Country

Taiwan

Facility Name

Investigational Site Number 8804

City

Taiwan

Country

Taiwan

Facility Name

Investigational Site Number 8805

City

Taiwan

Country

Taiwan

Facility Name

Investigational Site Number 9002

City

Adana

ZIP/Postal Code

?01130

Country

Turkey

Facility Name

Investigational Site Number 9004

City

Akdeniz

ZIP/Postal Code

07059

Country

Turkey

Facility Name

Investigational Site Number 9001

City

Ankara

ZIP/Postal Code

06100

Country

Turkey

Facility Name

Investigational Site Number 9005

City

Istanbul

ZIP/Postal Code

34093

Country

Turkey

Facility Name

Investigational Site Number 9003

City

Izmir

ZIP/Postal Code

35100

Country

Turkey

Facility Name

Investigational Site Number 9006

City

Turkey

Country

Turkey

Facility Name

Investigational Site Number 8003

City

Kyiv

ZIP/Postal Code

01135

Country

Ukraine

Facility Name

Investigational Site Number 8001

City

Kyiv

ZIP/Postal Code

04060

Country

Ukraine

Facility Name

Investigational Site Number 8002

City

Lviv

ZIP/Postal Code

79044

Country

Ukraine

Facility Name

Investigational Site Number 4407

City

London

ZIP/Postal Code

E1 2ES

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing 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://vivli.org

Citations:

PubMed Identifier

35114155

Citation

The Lancet Haematology. The role of conferences in tackling inequalities. Lancet Haematol. 2022 Feb;9(2):e81. doi: 10.1016/S2352-3026(22)00008-4. No abstract available.

Results Reference

derived

PubMed Identifier

34922648

Citation

Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available.

Results Reference

derived

Learn more about this trial

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)

Hemophilia A, Hemophilia B

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

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