Tag Archives: mps 2

Hunter Syndrome Trial Update 3-18-2016

 

The Breaking Barriers Hunter Syndrome Trial has successfully completed enrollment for the first cohort/group of four patients! We are now enrolling for the second cohort. Armagen plans to enroll 12 patients that are 18 years and older into this study. If you would like to view the full study update, you can download it below:
Study Update from Armagen 3-18-16

 

Armagen is developing therapies to non-invasively deliver drugs across the blood-brain barrier, a filter that protect the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. Available medications for Hunter Syndrome/MPS II do not cross the blood-brain barrier in clinically relevant amounts and therefore do not address the progress neurological complications of the disease.

 

If you would like to enroll in the Breaking Barriers Hunter Syndrome Trial, you may sign up here.

Breaking Barriers Hunter Syndrome Trial

 

 

Researchers are conducting a Hunter Syndrome trial of AGT-182, an investigational treatment for people with mucopolysaccharidosis type II or MPS 2.

 

AGT-182 is an enzyme replacement therapy (ERT) designed to treat both the body-related (somatic) and central nervous system (CNS) symptoms and complications of Hunter syndrome.

 

Currently approved treatments for Hunter syndrome (or MPS 2) are unable to treat the brain (CNS) because of the enzyme’s inability to cross the blood-brain barrier (BBB). The BBB is a filter that protects the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. As a result, available therapies for MPS disease do not address many of the severe and progressive neurological complications of the Hunter Syndrome. AGT-182 is designed to cross the BBB in the same way insulin does.

 

To sign up for the Breaking Barriers Hunter Syndrome trial, sign up here.

Hunter Syndrome Support Groups

Hunter Syndrome (MPS 2) Support Groups

 

If you or a loved one has Hunter syndrome, there are many dedicated organizations, Hunter Syndrome Support groups and other resources that foster education, support and research for these diseases.

 

We encourage patients and caregivers living with Hunter syndrome to learn more about ongoing clinical trials for investigational therapies.”-  Barbara Wedehase, MSW, CGC, Executive Director of the National MPS Society

 

CLICK THE LOGOS ABOVE TO BE LINKED TO THEIR WEBSITES

Participating Physicians Hunter Syndrome Trials

Participating Physicians – Hunter Syndrome Trial aka Mucopolysaccharidosis II or MPS2

Oakland Children’s Hospital

Dr. Paul Harmatz

Contact: Jacqueline Madden, PNP
510-428-3885 ext 5745
jmadden@mail.cho.org

Oakland, CA

SEE IF YOU QUALIFY

Children’s Hospital of Orange County

Dr. Raymond Wang

Contact: Nina Movsesyan
714-509-3008
nmovsesyan@choc.org

Orange, CA

SEE IF YOU QUALIFY

Emory Lysosomal
Storage Disease Center

Dr. William R. Wilcox

Contact: Stephanie Cagle
404-778-8421
stephanie.cagle@emoryhealthcare.org

Decatur, GA

SEE IF YOU QUALIFY

Ann & Robert H. Lurie Children’s
Hospital of Chicago

Dr. Barbara K. Burton

Contact: Rachel Katz
312-227-6764
rkatz@luriechildrens.org

Chicago, IL

SEE IF YOU QUALIFY

University of Minnesota

Dr. Chester B. Whitley

Contact: Brenda Diethelm-Okita
612-625-1594
dieth001@umn.edu

Minneapolis, MN

SEE IF YOU QUALIFY

Children’s Hospital
of Pittsburgh of UPMC

Dr. Gerard Vockley

Contact: Nadene Henderson, MS, CGC
412-692-6065
Ndh3@Pitt.edu

Pittsburgh, PA

SEE IF YOU QUALIFY

O & O Alpan, LLC

Dr. Ozlem Goker-Alpan

Contact: Chidima Ioanou
571-308-1905

Fairfax, VA 22030

SEE IF YOU QUALIFY

University Medical Center
Hamburg Eppendorf

Dr. Nichole M. Muschol

Contact: Nicole M Muschol, MD
+49 40 7410 20400
muschol@uke.de

Hamburg, Germany

SEE IF YOU QUALIFY

ZKJM MC University of Mainz

Dr. Julia Hennermann

Contact: Julia Hennermann, MD
+49(0) 6131 175754
julia.hennermann@unimedizin-mainz.de

Mainz, Germany

SEE IF YOU QUALIFY

HSK Dr. Horst-Schmidt-Kliniken GmbH

Dr. Christina Lampe

Contact: Christina Lampe, MD
+49 (611) 43-2314
christina.lampe@helios-kliniken.de

Wiesbaden, Germany

SEE IF YOU QUALIFY

Erasmus Medical Center

Dr. JMP van den Hout

Contact: Hannerieke van den Hout, MD
+31 (0) 10 7037 278
j.vandenhout@erasmusmc.nl

Rotterdam, Netherlands

SEE IF YOU QUALIFY

Institute of Human Genetics

Dr. Chiong

Contact: Angela Q Villa
+63 2 310 1780 ext 108
aqviall@post.upm.edu.ph

Manila, Phillipines

SEE IF YOU QUALIFY

About Breaking Barriers Clinical Trial

The Breaking Barriers Clinical Trial of AGT-182 is a Phase 1 trial that will test the safety and determine a well-tolerated dose of AGT-182 for the treatment of patients with Hunter Syndrome.

Participating in the Trial

Physicians at leading research institutions who specialize in hunter syndrome treatment are conducting the AGT-182 clinical trial. If you or a loved one has been diagnosed with Hunters syndrome and would like to participate in the clinical trial, please view the “Participating Physicians” page for the study center nearest to you. To find out if you qualify for the Hunter Disease trial you may:

  • Contact the clinical site via the contact form through this website
  • Call the clinical site directly

Key participation criteria:

  • Ages 18 or older
  • Diagnosed with Hunter syndrome
  • Available for 8 weeks of investigational treatment
  • Willing to discontinue standard enzyme replacement therapy and switch to AGT-182 for the duration of their participation in the clinical trial.

OR

For more information on the trial, please view the “Frequently Asked Questions” page or visit:

Home

RECENT STUDY UPDATE! Click here to download the Newsletter:
Study Update from Armagen 3-18-16

Researchers are conducting a clinical trial of AGT-182, an investigational treatment for people with Hunter Syndrome aka mucopolysaccharidosis II or MPS 2.

AGT-182 is an enzyme replacement therapy (ERT) designed to treat both the body-related (somatic) and central nervous system (CNS) symptoms and complications of Hunter syndrome.

Currently approved treatments for Hunter syndrome (or MPS 2) are unable to treat the brain (CNS) because of the enzyme’s inability to cross the blood-brain barrier (BBB). The BBB is a filter that protects the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. As a result, available therapies for MPS disease do not address many of the severe and progressive neurological complications of the Hunter Syndrome. AGT-182 is designed to cross the BBB in the same way insulin does.

Please CLICK HERE to find out how you or a loved one can participate in this trial.

Safety Information
This Hunter Syndrome trial is monitored and approved by a Data Monitoring Committee (DMC) and safety will be reported to an institutional review board to ensure the safety of the participants.