HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients



Status:Recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - Any
Updated:4/3/2019
Start Date:June 27, 2018
End Date:August 2024
Contact:Alison Long, MD
Email:a.long@uniqure.com
Phone:339-970-7578

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Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B

This is an open-label, single-dose, multi-center, multinational trial to demonstrate the
efficacy of AMT-061 and to further describe its safety profile.

The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant
adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a
codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a
liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous
infusion administered at a dose of 2 x 10^13 gc/kg.


Inclusion Criteria:

1. Male

2. Age ≥18 years

3. Subjects with congenital hemophilia B, classified as severe or moderately severe, and
are currently on factor IX prophylaxis

4. >150 previous exposure days of treatment with factor IX protein

Exclusion Criteria:

1. History of factor IX inhibitors

2. Positive factor IX inhibitor test at screening

3. Select screening laboratory value >2 times upper limit of normal

4. Positive human immunodeficiency virus (HIV) test at screening, not controlled with
anti-viral therapy

5. Active infection with hepatitis B or C virus at screening

6. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the
end of the lead-in phase

7. Previous gene therapy treatment

8. Receipt of an experimental agent within 60 days prior to screening

9. Current participation or anticipated participation within one year after study drug
administration in this trial in any other interventional clinical trial involving
drugs or devices
We found this trial at
18
sites
1 Children's Way
Little Rock, Arkansas 72202
(501) 364-1100
Arkansas Children's Hospital Arkansas Children's Hospital (ACH) is the only pediatric medical center in Arkansas...
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Chapel Hill, North Carolina 27599
(919) 962-2211
Phone: 919-966-4736
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Guy Young, MD
Phone: 323-361-3478
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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Nashville, TN
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Phone: 503-494-8716
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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Portland, OR
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Phone: 801-662-4700
University of Utah Research is a major component in the life of the U benefiting...
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Salt Lake City, UT
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4202 E Fowler Ave
Tampa, Florida 33620
(813) 974-2011
Phone: 813-974-4088
University of South Florida The University of South Florida is a high-impact, global research university...
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Tampa, FL
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500 S State St
Ann Arbor, Michigan 48109
(734) 764-1817
Phone: 734-764-9692
University of Michigan The University of Michigan was founded in 1817 as one of the...
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Ann Arbor, MI
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13001 E. 17th Pl
Aurora, Colorado 80045
303-724-5000
Phone: 303-724-3856
University of Colorado Denver The University of Colorado Denver | Anschutz Medical Campus provides a...
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Bruxelles,
Principal Investigator: C. Hermans, Ph.D., M.D.
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Bruxelles,
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936 Delaware Avenue
Buffalo, New York 14209
Principal Investigator: Adam Kotowski
Phone: 716-896-2470
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Buffalo, NY
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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East Lansing, Michigan 48824
Phone: 517-353-9385
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Houston, Texas 77030
Principal Investigator: Miguel Escobar, M.D.
Phone: 713-500-8360
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2501 South Hope Street
Los Angeles, California 90007
Principal Investigator: Doris Quon, MD
Phone: 213-742-1402
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Memphis, Tennessee 38163
Principal Investigator: Sandeep Rajan, MD
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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San Diego, California 92093
Principal Investigator: Annette von Drygalski
Phone: 858-657-6028
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