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Sponsors and Collaborators: |
Assistance Publique - Hôpitaux de Paris Association Française contre les Myopathies (AFM), Paris |
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Information provided by: | Assistance Publique - Hôpitaux de Paris |
ClinicalTrials.gov Identifier: | NCT00773227 |
The OCULO-Pharyngeal Muscular Dystrophy (OPMD) is a late onset hereditary muscle disease which is characterised by the selective affection of the pharyngeal muscles resulting in swallowing disorders, and by a ptosis from the dysfunction of the levator palpebral superiors muscles. Swallowing disorders are determinant in the prognosis of the disease, and potentially life-threatening deglutition, due to aspiration and denutrition. Degenerative dystrophy of the pharyngeal muscles causes difficulties to prepulse the food bolus in the pharynx, and the decreased relaxation of the cricopharyngeal muscle induced by the disease leads to blockage of food in the upper esophageal sphincter. The most common treatment for the dysphagia in OPMD is a myotomy of the upper esophageal sphincter muscles. However, although this will relax the constriction of the upper esophageal sphincter muscles and improve transitory the swallowing, it will not prevent the progressive degradation of the pharyngeal muscles. This progressive loss of contractility will eventually result in aspiration and severe difficulty in swallowing, increasing risk of aspiration pneumonia and severe weight loss which are the most common causes of mortality in OPMD patients.
The protocol which we are proposing is a graft of autologous cell muscles (myoblasts) isolated from unaffected limb muscles into the pharyngeal muscles of patients diagnosed as suffering from OPMD. Our aim is to improve both swallowing and the contractile deficit generated by the dystrophic pharyngeal muscles. A myotomy of the upper esophageal sphincter will be carried out at the same time as the myoblast transplantation, since we have already validated the improvement resulting from this surgery. Advantages of this new therapy in OPMD is the autograft, without risks of rejection, and the graft of myoblasts into the dystrophic pharyngeal muscles, above the myotomy of the upper esophageal sphincter muscles.
This model of cellular therapy has been studied through a preclinical study performed in dogs, allowing to valid the procedure and its safety, as well as to study the survival myoblasts grafted in the pharyngeal muscles.
This protocol is proposed for OPMD patients; it is firstly a safety study of both autograft and surgical procedure. In addition, the autograft may improve the swallowing disorders and life-threatening complications induced by aspiration and weight loss, resulting in a potential individual benefit.
Condition | Intervention | Phase |
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Muscular Dystrophy, Oculopharyngeal |
Procedure: Autologous myoblasts transplantation and myotomy |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts |
Estimated Enrollment: | 10 |
Study Start Date: | April 2004 |
Estimated Study Completion Date: | April 2011 |
Estimated Primary Completion Date: | October 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental
All patients included
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Procedure: Autologous myoblasts transplantation and myotomy
Graft of autologous cell muscles (myoblasts) isolated from unaffected limb muscles into the pharyngeal muscles of patients diagnosed as suffering from OPMD and surgical procedure (myotomy)
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Ages Eligible for Study: | 18 Years to 75 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Oculopharyngeal muscular dystrophy with UES dysfunction
Exclusion Criteria:
France | |
Hospital Tenon | |
Paris, France, 75020 |
Principal Investigator: | Jean Lacau-Saint Guily, MD, PH | Assistance Publique - Hôpitaux de Paris |
Responsible Party: | Department Clinical Research of Developpement ( Valerie Millul ) |
Study ID Numbers: | P020908, AOM02100 |
Study First Received: | October 15, 2008 |
Last Updated: | October 15, 2008 |
ClinicalTrials.gov Identifier: | NCT00773227 |
Health Authority: | France: Ministry of Health |
Oculopharyngeal muscular dystrophy Swallowing disorders, dysphagia Pharyngeal propulsion Upper oesophageal sphincter Cricopharyngeal muscle |
Autologous myoblasts Autograft Myotomy of the upper oesophageal sphincter Fibroscopy of swallowing Videofluoroscopy of swallowing |
Otorhinolaryngologic Diseases Esophageal disorder Gastrointestinal Diseases Pharyngeal Diseases Deglutition Disorders Oculopharyngeal muscular dystrophy Muscular Dystrophies Digestive System Diseases Muscular Diseases |
Genetic Diseases, Inborn Muscular Disorders, Atrophic Musculoskeletal Diseases Neuromuscular Diseases Atrophy Muscular dystrophy Esophageal Diseases Muscular Dystrophy, Oculopharyngeal |
Nervous System Diseases |