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Gene Therapy

AAV5-hFIXco-Padua (AMT-061) for Hemophilia B

Phase 2
Waitlist Available
Led By Steven Pipe, MD
Research Sponsored by CSL Behring
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
>20 previous exposure days of treatment with FIX protein
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years post-dose
Awards & highlights

Study Summary

This trial is a study to test a new gene therapy for hemophilia B.

Eligible Conditions
  • Hemophilia B

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You have been treated with FIX protein for more than 20 days in the past.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years post-dose
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years post-dose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Secondary outcome measures
Annualized Bleeding Rate (ABR)
Annualized Exogenous Factor IX Usage
Annualized Exogenous Factor IX Usage Post-Continuous Prophylaxis
+2 more

Side effects data

From 2023 Phase 2 trial • 3 Patients • NCT03489291
67%
Arthralgia
67%
Back pain
67%
Headache
33%
Asthenia
33%
Paraesthesia
33%
Chest pain
33%
Osteonecrosis
33%
Joint swelling
33%
Alanine aminotransferase increased
33%
Peripheral coldness
33%
Tachycardia
33%
Joint range of motion decreased
33%
Pneumonia
33%
Pain
33%
Bronchitis
33%
Blood creatine phosphokinase increased
33%
Upper respiratory tract infection
33%
Coccidioidomycosis
33%
Hypersensitivity
33%
Dizziness
33%
Dyspnoea
33%
Aspartate aminotransferase increased
33%
Hypertension
33%
Sinus headache
33%
Tongue disorder
33%
Influenza
33%
Blood potassium decreased
33%
C-reactive protein increased
33%
Transaminases increased
33%
Vertigo
33%
Erectile dysfunction
100%
80%
60%
40%
20%
0%
Study treatment Arm
AMT-061

Trial Design

1Treatment groups
Experimental Treatment
Group I: Single infusion of AMT-061Experimental Treatment1 Intervention
Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline. After IMP administration (post IMP), subjects will be monitored for tolerance to the IMP and detection of potential immediate AEs at the clinical trial site for 24 hours (overnight stay).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AAV5-hFIXco-Padua (AMT-061)
2018
Completed Phase 2
~10

Find a Location

Who is running the clinical trial?

CSL BehringLead Sponsor
194 Previous Clinical Trials
1,211,219 Total Patients Enrolled
18 Trials studying Hemophilia B
4,318 Patients Enrolled for Hemophilia B
UniQure Biopharma B.V.Lead Sponsor
10 Previous Clinical Trials
271 Total Patients Enrolled
2 Trials studying Hemophilia B
77 Patients Enrolled for Hemophilia B
Steven Pipe, MDPrincipal InvestigatorUniversity of Michigan
1 Previous Clinical Trials
67 Total Patients Enrolled
1 Trials studying Hemophilia B
67 Patients Enrolled for Hemophilia B

Media Library

AAV5-hFIXco-Padua (AMT-061) (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03489291 — Phase 2
Hemophilia B Research Study Groups: Single infusion of AMT-061
Hemophilia B Clinical Trial 2023: AAV5-hFIXco-Padua (AMT-061) Highlights & Side Effects. Trial Name: NCT03489291 — Phase 2
AAV5-hFIXco-Padua (AMT-061) (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03489291 — Phase 2

Frequently Asked Questions

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

Could you provide information regarding the scope of this research in North America?

"Currently, this research is operating out of 4 locations spread across the United States. Sites can be found in Sacramento, San Diego and Ann Arbor as well as another undisclosed site. It would be prudent to select a clinic that is closest to your home address so you can minimize travel needs if accepted into the study."

Answered by AI

Is AAV5-hFIXco-Padua (AMT-061) a safe treatment option for individuals?

"While Phase 2 trials provide some evidence of safety, they lack efficacy data. Thus, our internal team at Power assigned AAV5-hFIXco-Padua (AMT-061) a score of two on the safety scale from 1 to 3."

Answered by AI

Is this study currently seeking volunteers?

"Clinicaltrials.gov provides evidence that this study, which was first published on July 24th 2018 and last updated on May 17th 2022, is not currently recruiting patients. Nonetheless, 89 other trials are actively enlisting participants at the moment."

Answered by AI
~0 spots leftby Mar 2025