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Trial record 1 of 1 for:    NCT00773227
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Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts (OPMD)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Association Française contre les Myopathies (AFM), Paris
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT00773227
First received: October 15, 2008
Last updated: November 14, 2013
Last verified: November 2013
  Purpose

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
Muscular Dystrophy, Oculopharyngeal
Procedure: Autologous myoblasts transplantation and myotomy
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts

Resource links provided by NLM:


Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • The principal evaluation of the efficiency of the graft will be based on the functional quality of the pharyngeal propulsion as determined by fibroscopy and videofluoroscopy of swallowing. [ Time Frame: before the graft and at 2, 6, 12, 18 and 24 months after the graft ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • on the global swallowing properties which will be evaluated by a quantitative test, by a questionnaire and by an evaluation of the tolerance. This evaluation will include a clinical examination at each visit consisting [ Time Frame: before the graft and at 2, 6, 12, 18 and 24 months after the graft ] [ Designated as safety issue: No ]

Enrollment: 30
Study Start Date: April 2004
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
All patients included
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)
Other Name: autograft and surgery

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Man or woman <18-75> years old
  • Oculopharyngeal muscular dystrophy confirmed by genetic diagnosis (mutation of the GCG) on the chromosome 14)
  • Oculopharyngeal muscular dystrophy with UES dysfunction

    1. salivary or alimentary stasis at fibroscopy of swallowing above the UES,
    2. decreased opening of the UES at videofluoroscopy of swallowing A decreased of the pharyngeal propulsion may be associated Written consent of the patient

Exclusion Criteria:

  • History of myotomy of the UES in the context of the Oculopharyngeal muscular
  • Dystrophy;
  • HIV, hepatitis B or C tuberculosis);
  • Lupus, rheumatoid polyarthritis, sarcoïdosis, collagenosis) ;
  • Other neuromuscular diseases ;
  • History of malignant tumor ;
  • History of neck radiotherapy ;
  • Renal failure (creatinine clearance <60ml/min)
  • Liver failure ;
  • Pregnancy ;
  • Follow up less than 24 months:
  • Patients who refuse to sign the consent;
  • No social security.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00773227

Locations
France
Hospital Tenon
Paris, France, 75020
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Association Française contre les Myopathies (AFM), Paris
Investigators
Principal Investigator: Jean Lacau-Saint Guily, MD, PH Assistance Publique - Hôpitaux de Paris
  More Information

No publications provided by Assistance Publique - Hôpitaux de Paris

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT00773227     History of Changes
Other Study ID Numbers: P020908, AOM02100
Study First Received: October 15, 2008
Last Updated: November 14, 2013
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
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

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Oculopharyngeal
Genetic Diseases, Inborn
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases

ClinicalTrials.gov processed this record on November 20, 2014