Update on Clinical Trial A083-02: a Phase 2 Clinical Study of ACE-083 in FSHD

The FSH Society has a long history of partnering with biotech and pharmaceutical companies to facilitate recruitment of patients and families for focus groups, provide patient input to clinical outcome measures, and participation in clinical trials. The Society also assists companies by providing connections, insights and scientific information in the research, therapeutic and clinical areas. For the ongoing trial of ACE-083, the FSH Society has worked with the drug’s developer, Acceleron Pharma, to better understand how FSHD affects patients through a survey (see story here) as well as to educate patients about the process of enrolling in the clinical trial. In response to the high degree of interest in the ACE-083 trial, Acceleron has worked with the FSH Society to provide the following update and FAQ. We thank Acceleron for the company’s commitment to patient education.

Acceleron Pharma has received a tremendous amount of interest in the Phase 2 study of its investigational drug, ACE-083, in patients with facioscapulohumeral muscular dystrophy (FSHD). In response to the robust interest in this clinical trial, we wanted to provide information for how best to inquire about participating.

The clinical trial consists of two parts. Part 1 of the study will include up to 36 patients at multiple sites in the United States. Part 2 will include up to 40 additional patients at sites in the United States, Canada and Spain. There are currently eight (8) U.S. sites actively enrolling patients with more sites to be added in the next two months. The clinical trial listing at www.clinicaltrials.gov, with study identifier NCT02927080, will be updated when new sites are added and with information regarding whether or not each site is actively enrolling new patients. Please check www.clinicaltrials.gov to see if a site near you is enrolling patients in this clinical trial.

Please understand that, like most phase 2 clinical studies, there are a limited number of patients to be enrolled and therefore not every interested patient will be able to participate in this particular study. If you believe that you meet the eligibility criteria, which can be found on www.clinicaltrials.gov and want to get involved in the study, please contact the site nearest to you directly. The individual study sites are responsible for all activities related to the execution of the study, including patient selection and administering therapy and tests. You should not contact Acceleron regarding eligibility and participation in the study as the company is not allowed to interact directly with patients on these matters.

Please click here to learn more about the trial and to find contact information for the site nearest to you. In addition, a list of Frequently Asked Questions (FAQ) and answers is provided below for your reference.

FAQ’s on Clinical Trial A083-02: a Phase 2 Clinical Study of ACE-083 in FSHD

1. What is the A083-02 clinical study?
This clinical study is designed to evaluate the safety, tolerability and efficacy of ACE-083 in up to 76 adult patients with facioscapulohumeral muscular dystrophy (FSHD). ACE-083 is an investigational protein therapeutic being developed by Acceleron Pharma, Cambridge, MA. The study will be conducted in two parts: Part 1 (up to 36 patients) will be used to assess the safety, tolerability and beneficial effects at various doses of ACE-083 in patients with FSHD. Following the completion of Part 1, Part 2 (up to 40 patients) will use the dose of ACE-083 chosen from Part 1 to determine whether muscle volume, strength, and function are increased when compared to treatment with a placebo.

Part 1 of the study will be conducted at multiple sites within the United States. Part 2 is planned to involve sites in the United States (12 sites), Canada (3 sites) and Spain (2 sites). Total study duration for each patient will be approximately 24 weeks, including a 4-week screening period, a 12-week treatment period, and an 8-week follow-up period after the last dose.

2. What is ACE-083 and what does it do?
ACE-083 is an investigational drug that interacts with selected proteins in the transforming growth factor-beta (TGF-β) superfamily involved in the regulation of muscle mass and strength. ACE-083 has been designed to increase muscle mass and strength selectively in the muscles into which the drug is injected. Acceleron is developing ACE-083 for diseases in which improved muscle strength in a specific set of muscles may provide a clinical benefit to patients, such as FSHD. As an “investigational” drug, ACE-083 is not approved by any regulatory agency for use in any country at this time.

3. Where can I find the latest information about ACE-083 and the A083-02 clinical study?
You can learn more about ACE-083 at the Acceleron website, www.acceleronpharma.com. You can find more information about the ACE-083 phase 2 clinical study in FSHD by visiting www.clinicaltrials.gov and searching for study identifier NCT02927080. This listing will contain the most current information regarding the trial and is updated regularly.

4. What are the criteria for participating in the A083-02 clinical study?
Patients who are 18 years of age or older and have genetically confirmed FSHD or patients with a clinical diagnosis of FSHD and a first-degree relative with genetically confirmed FSHD may be eligible to take part in the ACE-083 study. Eligible patients must also be experiencing mild to moderate weakness in their tibialis anterior and/or biceps brachii muscles. Several additional criteria are included in the study protocol. Patients must meet all inclusion and exclusion criteria, including those listed for this study on www.clincialtrials.gov, to participate. It is best to have your physician assess for eligibility criteria or to contact study sites directly.

5. What does mild to moderate weakness in the tibialis anterior muscle look like?
Mild to moderate weakness of the tibialis anterior (front shin muscle) means you have some degree of foot drop, but are still able to lift your foot towards your shin against gravity. You may have weakness at the ankle (e.g., you may catch your toe when walking, or have to lift your foot high in order not to trip) but you still have some movement of the ankle.

6. What does mild to moderate weakness in the biceps brachii muscle look like?
Mild to moderate weakness of the biceps means you have biceps weakness, but can still lift your hand to your face against gravity (using the biceps). You have difficulty lifting a gallon of house paint or milk, but can still bend your elbow.

7. What clinical trial sites are involved in the A083-02 clinical study?
Currently, study A083-02 is only being conducted at sites in the United States. A list of site can be found on www.clinicaltrials.gov. Sites in Canada and Spain are expected to open enrollment by the end of this year. As new sites become available, they will be updated on www.clinicaltrials.gov with study identifier NCT02927080.

8. Who can I contact to get involved with the A083-02 clinical study?
When considering participation in a clinical study, it is best to speak with your physician. If you meet the eligibility criteria, and want to get involved in the study, please review the list of sites on www.clinicaltrials.gov for the location nearest you. You or your physician must contact this site directly in order to be considered for the study.

9. Who is the A083-02 clinical study sponsor and can I contact them directly?
Acceleron Pharma is the sponsor of the A083-02 clinical study and is the inventor and manufacturer of ACE-083. Please note that Acceleron is not allowed to assess patient eligibility for clinical trials, cannot contact sites directly on behalf of patients, nor provide any medical advice. The individual study sites are responsible for all activities related to the execution of the study, including patient selection and administering therapy and tests. You should not contact Acceleron regarding eligibility and participation in the study as the company is not allowed to interact directly with patients on these matters.

10. Is it possible to obtain ACE-083?
ACE-083 is an investigational drug and is not commercially available in any country. It is only accessible to patients enrolled in a clinical study.

11. Will there be other opportunities to participate if I cannot be in the A083-02 study?
A083-02 is the first study of ACE-083 in patients with FSHD. If ACE-083 demonstrates positive outcomes with respect to safety and efficacy, then there may be opportunities for you to participate in future studies in FSHD. Acceleron will promptly provide information if new studies are initiated with ACE-083.