In an effort to keep the Duchenne muscular dystrophy community informed, Acceleron and Shire wish to provide an update about the status of the ACE-031 clinical program. During the course of clinical trials in healthy adults and in DMD boys, some participants experienced minor nosebleeds, gum bleeding, and/or small dilated blood vessels within the skin. These events all resolved fully upon discontinuation of treatment. By themselves, the minor bleeding events and dilated blood vessels were not considered to be a serious safety concern for study subjects. However, based on review of these safety data with the FDA and Health Canada, Acceleron has terminated the A031-03 DMD study and has suspended enrollment and dosing in the follow-on extension study A031-06.

Acceleron and Shire remain committed to the global DMD clinical program and the development of ACE-031. To that end, it is our intention to start new studies of ACE-031 in DMD with appropriate safety monitoring following discussions with regulatory agencies. In the coming months, we will provide updates to the DMD community as appropriate.

 

 

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I really hope that they find a way to minimize these side effects... I feel terrible thinking at our sons having all these side effects from different new drugs on top of the steroid side effects... I hope the 4 week dosing does the trick...

Moein said:

Ofelia,My sonis on the waiting list,and his neurologist who directs the trial (first site)called me to update me.

My son is supposed to enter the trial maximum in fall.

my  son was one of the ones getting nosebleeds, and I would not call them minor.    He had a couple gushers.

I have been told that they are trying to resolve the bleeding problems,but how
?

Jason, I hope your son is doing well.  I comprehend how difficult it is to be involved in an experimental process and very much appreciate his, and your bravery.  Thank you!!  You guys are champs!!!
Jason, I had no idea that it was so bad...they made it sound like something minor. :(

 

Jason first of all thank you, your son is helping all of us like everyone taking part on trials, did you see any benefits or did you son´s muscles grew while he was taking ACE-031?

Would you say it was worth the bleedings?, please give us some feedback about the drug itself.

 

greetings

JP.

 

Hi All,

Just wanted to check if anybody has update. Is there any chances for the trial to restart?

Thanks,Amrit

 

 

A Phase 2, Randomized, Placebo-Controlled, Multiple Ascending-Dose Study of ACE-031, a Soluble Activin Receptor Type IIB, in Boys with Duchenne Muscular Dystrophy (DMD)

Craig Campbell, London, ON, Canada, Diana Escolar, Baltimore, MD, Jean Mah, Calgary, AB, Canada, Mark Tarnopolsky, Hamilton, ON, Canada, Kathy Selby, Vancouver, BC, Canada, Hugh McMillan, Ottawa, ON, Canada, Yijun Yang, Dawn Wilson, Rachel Barger, Matthew Sherman, Kenneth Attie, Cambridge, MA 

OBJECTIVE: To evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic (PD) effects of ACE-031 in DMD. BACKGROUND: ACE-031 is a soluble activin type IIB receptor (ActRIIB-IgG1) that binds negative regulators of muscle, including myostatin and activin. In phase 1 studies in healthy adults, ACE-031 treatment resulted in dose-dependent increases in lean mass. DESIGN/METHODS: Ambulatory (30ft walk/run <12sec), steroid-treated DMD boys, age ≥4yr, were randomized to 12 weeks treatment with either escalating doses of ACE-031 or placebo (9:3 in each cohort), followed by 12-weeks follow-up. The primary objective was safety/tolerability. PD endpoints included lean/fat/bone mass (DXA), thigh muscle, fat volume (MRI), strength, 6-minute-walk-test-distance (6MWD), and other motor/pulmonary function tests. RESULTS: Two cohorts completed treatment (C1: 0.5mg/kg SC q4weeks, C2: 1mg/kg SC q2weeks). Mean age was 10.3 years (range 6-17). There were no AEs that were serious, severe, or resulted in discontinuation. Reversible telangiectasias and mild epistaxis were reported in C2. Mean total body lean mass by DXA increased 5.2% in C2 (p=0.015 vs baseline) vs 2.6% in placebo (p=0.20) after 12 weeks. Thigh MRI (C2) showed a trend for decreased muscle volume in placebo group which was attenuated in ACE-031 group. The least-squares mean 
Δ6MWD was +12meters in combined ACE-031 groups vs -30meters in placebo group after 24 weeks (p=0.06, ANCOVA). The %change in 6MWD correlated with that for 2MWD (r=0.67, p<0.001) and 10m walk/run (r=-0.53, p=0.009) after 24 weeks. Dose-related trends for decreased gain in fat mass and increased lumbar spine BMD by DXA were seen after 12 and 24 weeks. CONCLUSIONS: In this phase 2 study of ACE-031 in steroid-treated DMD boys, reversible telangiectasias and mild epistaxis were reported at higher dose. Significant dose-dependent increases in lean mass were observed. There were trends for maintained 6MWD, decreased gain in fat mass, and increased lumbar spine BMD with ACE-031 treatment. Supported by: MDA, PPMD.


http://www.distrofiamuscular.net/abstracts27.htm

Any update on ACE 031?  Been quiet out there for a while.

Ofelia - are these the results from the study in Canada that was terminated last year? They seem to be.

If so it makes it all the more baffling that another study has not yet been announced.

 

Yes, they are. They look so positive, so frustrating to see that almost a year passed and they did not come out with a solution to minimize the side effects and progress to the next trial. I just cannot believe that our sons need to lose so much time when something with this potential is out there.

David said:

Ofelia - are these the results from the study in Canada that was terminated last year? They seem to be.

If so it makes it all the more baffling that another study has not yet been announced.

 

Almost ONE YEAR and nothing from these people. How frustrating is this? Are they still working on this? do they have very clear in mind what 1 year means for our boys?

http://quest.mda.org/news/ace-031-clinical-trials-duchenne-md-stopp...

Ofelia Marin said:

Yes, they are. They look so positive, so frustrating to see that almost a year passed and they did not come out with a solution to minimize the side effects and progress to the next trial. I just cannot believe that our sons need to lose so much time when something with this potential is out there.

David said:

Ofelia - are these the results from the study in Canada that was terminated last year? They seem to be.

If so it makes it all the more baffling that another study has not yet been announced.

 

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