At our May meeting, one member shared a great question: Has anyone had experience with falling backwards due to their lordosis? She didn’t feel any warning signals, and just fell back.

by Amanda Hill, FSHD Society

How do I find clinical trials that are recruiting participants?

Our website is one of the best ways to find clinical trials that are recruiting. We will always have current information for you on our Clinical Trials web page. Another great way to find clinical trials is at clinicaltrials.gov. This website is a database for clinical trials and other clinical research studies all over the world.

Taking charge of people with FSHD. Treatment, prospects and therapeutic opportunities. Two live webinars dedicated to patients and health professionals promoted by the EpiThe4FSHD project and the FSHD-UILMD Patient Group.

The interview is edited by Maria Giovanna Tortora for the FSHD Group of UILDM.

When will the trial start?

The NeMO Center in Milan is ready to start recruiting and we are currently awaiting the latest regulatory checks. Indicatively, the first patients will be enrolled between the end of April and the beginning of May.

 

How will patient recruitment be organized? What will be the criteria required to access the trial?

About 100 people with FSHD are regularly taken care for at the NeMO Center in Milan, some of whom have infantile-onset pathologies.

Recruitment will be dictated by the inclusion/exclusion criteria of the study protocol. In particular, in this case we refer to adult patients aged between 18 and 65 years, ambulatory, with a confirmed genetic diagnosis of either Type1 or Type2. The requirements of the study also provide that there are residual skills in the shoulder girdle and in the upper limbs, skills that will be defined according to an instrumental evaluation, confirmed on the basis of the requirements set by the trial.

Potential candidates meeting the above requirements will be contacted by the NeMO Milano team, who will verify the effective suitability of the criteria and, of course, the person's willingness to participate in the study.

 

Can you describe the Fulcrum trial? How will the NeMO Center in Milan be organized in order to host it?

Fulcrum is a double-blind, randomized controlled phase-3 drug trial. This means that neither the patient nor the clinical team will be aware of the nature of the administration - drug or placebo – so as to ensure the methodological correctness of the trial protocol.

The main objective of the study, which will last 48 weeks, is to verify the efficacy of the molecule losmapimod (in oral tablet) in blocking the DUX4 gene, a key gene in the etiology of the disease. From a clinical point of view, this means slowing down the progression of the disease, especially in the function of the upper limbs.

For this reason, the study provides for constant and regular clinical evaluations in order to monitor the progress of the pathology and the possible efficacy of the drug, while simultaneously recording any side effects. However, in relation to side effects, it is quite important to underline that the safety study has already been carried out and the drug has demonstrated a good safety and tolerability profile in the previous phases of the trial.

From an organizational point of view, NeMO of Milan, through its Clinical Research Center, has been following pharmacological trials since 2017, with a research team made up of professionals who have acquired experience in conducting studies in all neuromuscular pathologies. Therefore, there will also be a dedicated team for Fulcrum trial who will work in synergy with the national and international network

 

What does the trial pathway involve if the patient gets worse? And also, would you like to add anything else which you think important to know for the patients?

As previously said, the safety and tolerability studies of the drug have already been confirmed, therefore should any side effects occur, that will be reported to the study coordinator center and the scientific procedure will be promptly evaluated, as well as of course we will refer to the indications of clinical practice in use. It is always important to underline that, not only is each patient free to participate and/or interrupt the study at any moment of the process, but that this decision does not in any way compromise his/her taking charge at the Center itself. This latter objective represents a priority of our mission.

The aim of  this study is to slow the typical progression of FSHD, and it must be said that the drug is not expected to lead to a worsening of the disease.

While monitoring the progress of the disease, in case a functional worsening should be noted, we will need to understand - at the end of the 48 weeks of the study - how much this worsening can be related to the drug/placebo intake - which is not expected – rather than how much it can be dependent on the natural course of the pathology itself. In any case, should there be a deterioration owned to the natural progression of the disease, whether a reduced entity of such deterioration should be reached due to beneficial administration of Lospaminod, that would still be an excellent result for the study.

As a testament to the scientific ferment that FSHD research is experiencing today, it must be said that the trial on the molecule losmapimod represents only one of the studies currently underway or which are in the start-up phase. Indeed, there are various others; for example, at the NeMO Center in Milan recruitment is also being carried out for the phase 2 trial (double blind/placebo-controlled) to verify the efficacy of antimyostatin (RO7204239 - a monoclonal antibody that binds myostatin). The study will have a duration of 2 years and involves the recruitment of adult and ambulatory patients.

Furthermore, NeMO Milano is involved in the incoming phase 1/2 studies to verify safety and tolerability, as well as explore the efficacy of the intravenously administered AOC1020 molecule, which aims to block the DUX4 gene. The preliminary study protocol is currently being evaluated at the Center.

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Elena Carraro, medico fisiatra, referente area riabilitativa del Centro Clinico NeMO di Milano

Vania Campanella, study coordinator Clinical Research Center - Centro Clinico NeMO di Milano

Alessandra Di Bari, study coordinator Clinical Research Center - Centro Clinico NeMO di Milano

Monica Montuori, study coordinator Clinical Research Center - Centro Clinico NeMO di Milano

 

In the photo: top left Alessandra Di Bari, right Elena Carraro; bottom left Monica Montuori, right Vania Campanella 

Thanks the Communication Office of the NeMO clinical centers.

At our December 13 Wellness Hour, Jennifer Egert led a short session on mindfulness in which she asked people to focus their awareness on their body sitting.

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