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RNAi Therapeutics

Fitusiran for Pediatric Hemophilia (ATLAS-PEDS Trial)

Q: Who would be an ideal candidate for this clinical trial?

Those with hemophilia a between the ages of 1 and 11 may be eligible for this clinical trial. So far, 32 individuals have been accepted into the study.

Q: Are people with the specific illness needed for this research currently being accepted?

That is correct, the clinical trial detailed on clinicaltrials.gov is still recruiting patients. The study was first announced on January 28th, 2020 with the most recent update coming April 7th, 2022. They are currently looking for 32 individuals that meet the criteria from 5 different locations.

Q: Does this kind of clinical trial have precedent?

Fitusiran has undergone 4 clinical trials since 2015, with the most recent study still ongoing. 37 patients participated in the first trial which was sponsored by Genzyme Corporation and completed its Phase 1 &#x26; 2 stages successfully. The current live studies involve 34 cities across 24 countries.

Q: What is the global distribution of this clinical trial?

So far, this study has enrolled patients from 5 sites: 8400008 in Hackensack, 8400002 in Los Angeles, 8400007 in Cleveland, and other locations.

Q: Does this research project require participants to be over a certain age?

This particular study is for children aged 1 year to 11. In total, there are 43 clinical trials underway for patients younger than 18 and 69 different trials for seniors.

Q: How many individuals will be included in this research project?

The trial's sponsor, Genzyme (a Sanofi Company), needs to enrol 32 patients that fit the clinical profile for the study. This will be conducted from different sites including Hackensack, <a href="https://www.withpower.com/clinical-trials/new-jersey">New jersey</a> and Los Angeles, <a href="https://www.withpower.com/clinical-trials/ohio">Ohio</a>.

Q: Could you please compare and contrast the results of this clinical trial with others involving Fitusiran?

There are 3 ongoing studies related to Fitusiran, 2 of which are Phase 3 trials. The capital cities of Ukraine and the United Kingdom host the majority of these medical investigations, but there are a total of 184 research sites worldwide.

Phase 3

Waitlist Available

Research Sponsored by Genzyme, a Sanofi Company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL

Weight requirements at the time of enrollment: 8 to <45 kg

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up)

Awards & highlights

ATLAS-PEDS Trial Summary

This trial is testing a new drug for hemophilia in children. The goal is to see if it is safe and effective.

Who is the study for?

This trial is for male children aged 1 to less than 12 with severe hemophilia A or B. They must have a history of inhibitor antibodies and meet specific blood test criteria. Participants need good vein access for blood draws, weigh between 8 to <45 kg, and be able to follow the study plan with parental consent.Check my eligibility

What is being tested?

The trial is testing appropriate dose levels of Fitusiran in young boys with severe hemophilia A or B. It aims to confirm dosing, assess safety and tolerability, and measure how much drug stays in the bloodstream over time.See study design

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions at injection sites, possible liver issues due to altered clotting factor production, allergic responses, or other unforeseen complications related to Fitusiran.

ATLAS-PEDS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My latest inhibitor test result is below 0.6 BU/mL, but I have records showing two previous results of 0.6 BU/mL or higher.

Select...

My weight is between 8 and 45 kg.

Select...

I have antibodies against clotting factors and meet specific test criteria.

Select...

I am a boy aged between 1 and 11 years.

ATLAS-PEDS Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up)

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of at follow up) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Plasma antithrombin (AT) activity levels

Secondary outcome measures

Fitusiran plasma concentrations

Number of participants reported with adverse events

Side effects data

From 2022 Phase 3 trial • 80 Patients • NCT03549871

27%

Alanine Aminotransferase Increased

12%

Nasopharyngitis

Upper Respiratory Tract Infection

Fibrin D Dimer Increased

Arthralgia

Cholelithiasis

Injection Site Pain

Cholecystitis

Aspartate Aminotransferase Increased

Cough

Abdominal Pain

Injection Site Erythema

Hepatic Steatosis

Toothache

Haemophilic Arthropathy

Influenza

Asthma Late Onset

Vomiting

Femur Fracture

Cerebrovascular Accident

Stevens-Johnson Syndrome

Central Venous Catheter Removal

Biliary Neoplasm

Headache

Joint Swelling

Synovitis

C-Reactive Protein Increased

Pancreatitis Acute

Vascular Device Infection

Arthropod Bite

Fall

100%

80%

60%

40%

20%

Study treatment Arm

Overall: SAS 1 - Fitusiran 80 mg QM

Cohort A: SAS 1 - Fitusiran 80 mg QM

Cohort A: SAS 2 - Fitusiran 50 mg Q2M

Overall: SAS 1 - Factor/BPA Prophylaxis

Cohort A: SAS 1 - BPA Prophylaxis

Cohort B: SAS 1 - Factor Prophylaxis

Cohort B: SAS 1 - Fitusiran 80 mg QM

Cohort A: SAS 2 - BPA Prophylaxis

ATLAS-PEDS Trial Design

1Treatment groups

Experimental Treatment

Group I: FitusiranExperimental Treatment1 Intervention

Participants will receive a selected dose of fitusiran at regular intervals, as per study protocol

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fitusiran

2018

Completed Phase 3

~80

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor

524 Previous Clinical Trials

85,597 Total Patients Enrolled

2 Trials studying Hemophilia

361 Patients Enrolled for Hemophilia

Clinical Sciences & OperationsStudy DirectorSanofi

862 Previous Clinical Trials

2,019,806 Total Patients Enrolled

3 Trials studying Hemophilia

436 Patients Enrolled for Hemophilia

Media Library

Fitusiran (RNAi Therapeutics) Clinical Trial Eligibility Overview. Trial Name: NCT03974113 — Phase 3

Hemophilia Research Study Groups: Fitusiran

Hemophilia Clinical Trial 2023: Fitusiran Highlights & Side Effects. Trial Name: NCT03974113 — Phase 3

Fitusiran (RNAi Therapeutics) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03974113 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who would be an ideal candidate for this clinical trial?

"Those with hemophilia a between the ages of 1 and 11 may be eligible for this clinical trial. So far, 32 individuals have been accepted into the study."

Answered by AI

Are people with the specific illness needed for this research currently being accepted?

"That is correct, the clinical trial detailed on clinicaltrials.gov is still recruiting patients. The study was first announced on January 28th, 2020 with the most recent update coming April 7th, 2022. They are currently looking for 32 individuals that meet the criteria from 5 different locations."

Answered by AI

Does this kind of clinical trial have precedent?

"Fitusiran has undergone 4 clinical trials since 2015, with the most recent study still ongoing. 37 patients participated in the first trial which was sponsored by Genzyme Corporation and completed its Phase 1 & 2 stages successfully. The current live studies involve 34 cities across 24 countries."

Answered by AI

What is the global distribution of this clinical trial?

"So far, this study has enrolled patients from 5 sites: 8400008 in Hackensack, 8400002 in Los Angeles, 8400007 in Cleveland, and other locations."

Answered by AI

Does this research project require participants to be over a certain age?

"This particular study is for children aged 1 year to 11. In total, there are 43 clinical trials underway for patients younger than 18 and 69 different trials for seniors."

Answered by AI

How many individuals will be included in this research project?

"The trial's sponsor, Genzyme (a Sanofi Company), needs to enrol 32 patients that fit the clinical profile for the study. This will be conducted from different sites including Hackensack, New jersey and Los Angeles, Ohio."

Answered by AI

Could you please compare and contrast the results of this clinical trial with others involving Fitusiran?

"There are 3 ongoing studies related to Fitusiran, 2 of which are Phase 3 trials. The capital cities of Ukraine and the United Kingdom host the majority of these medical investigations, but there are a total of 184 research sites worldwide."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

2020. "Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03974113.

All > Hemophilia A > Hemophilia