Muscular Dystrophy, OculopharyngealPhase 2OPMDNCT00773227
Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts
A Phase 2 study testing Autologous myoblasts transplantation and myotomy in people with Muscular Dystrophy, Oculopharyngeal, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Muscular Dystrophy, Oculopharyngeal
- What is being tested?
- Autologous myoblasts transplantation and myotomy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- Apr 2004to Oct 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
- salivary or alimentary stasis at fibroscopy of swallowing above the UES,
- decreased opening of the UES at videofluoroscopy of swallowing A decreased of the pharyngeal propulsion may be associated Written consent of the patient
Who cannot join
- 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 ;
Show all 13Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read more
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.
Read the detailed description
OCULO-Pharyngeal Muscular Dystrophy (OPMD) is characterised by the selective affection of the superior sphincter muscles of the oesophagus (SSO) and the pharyngeal.muscles resulting in dysphagia. The most common treatment for the dysphagia induced by this disease is a myotomy. However, although this will relax the constriction and improve swallowing it will not prevent the progressive degradation of the pharyngeal muscles. This progressive loss of contractility will eventually result in false routes and severe difficulty in swallowing, increased risk of pulmonary infection and severe weight loss which are the most common causes of mortality in these patients.
Concept:
The protocol which we are proposing is a pilot study in which autologous myoblasts isolated from unaffected limb muscles will be grafted into the pharyngeal constrictor muscles of patients diagnosed as suffering from OPMD. Our aim is to improve both swallowing and the contractile deficit generated by the dystrophic pharyngeal constrictor muscles. A myotomy of the SSO will be carried out at the same time as the myoblast transplantation, since we have already validated the improvement resulting from this surgery even though we know that this will provide only a partial and transitory improvement.
Type of trial: This is a multicentric trial with a direct benefit for the patient in which 10 patients will receive an autologous transplantation of myoblasts. Due to the possibility that a certain number of patients may withdraw from the study we have decided to include initially a maximum of 15 patients. The PHRC for this clinical trial was accepted in 2002 and the official promotor is the AP-HP. The patients will be selected by the different promotors but will be followed in the 'Service d'ORL of TENON Hospital '. The proliferative capacity of the myoblasts isolated from the muscle biopsies will be carried out by Vincent MOULY and KAMEL MANCHAOUI in the UMR 7000 CNRS directed by Dr Butler-Browne and the cells will be amplified prior to grafting in the Cellular laboratory therapy of Dr MAROLLEAU; The clinical trial will be controlled by a specifically selected committee of five.
Description of the trial In the first step of this study patients will be selected for inclusion in the trial according to the following criteria : 1: men or women aged between 18 and 75 years ; 2: genetic confirmation and characterisation of OPMD 3: SSO dysfunction confirmed by the stasis of saliva or food above the sphincter visualised by fibroscopy and a defect in the opening of the SSO also confirmed by videofluoroscopy of swallowing; the SSO defect may be associated with a reduction in pharyngeal propulsion as revealed by fibroscopy and videofluoroscopy of swallowing 4: official signed consent of the patients to participate in this clinical trial.
The next step will be the selection of the unaffected muscle to be used for the production of the autologous myoblasts to be used for transplantation. Muscles will be selected for engraftment on the basis of the proliferative capacity of the cultures made from biopsies (0.5-1gm) obtained following local anesthesia of either the quadriceps or the STERNO-CLEIDO-MASTOIDIEN muscles. The muscle which is able to produce the largest number of myoblasts will be biopsied a second time using the same conditions. Two grams of muscle will be removed and the myoblasts will be isolated and cultured until there are about 100 million myoblasts at which point the cells can be injected into the pharyngeal muscles of the patient. If there is no difference between the two different muscle biopsies then we will biopsy preferentially the STERNO-CLEIDO-MASTOIDIEN muscle since it is situated directly in the trajectory that will be used when grafting the myoblasts. The operation by lateral cervicotomy will be carried out under general anesthesia, in order to expose the SSO and the pharyngeal muscles. The graft will be carried out by injecting the myoblasts into about 20 different sites in the pharyngeal constrictor muscles above the site of the associated myotomy of the SSO. The next step will involve a two year evaluation of the trial. For each patient a descriptive analysis will be carried out based on the comparison of the different results. 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. A secondary evaluation will be based 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 of the examination of the pharynx in order to confirm the absence of muscle tumors and a cervical palpation in order to determine the volume of the muscle and to survey the site of myoblasts implantation. A rigid tube pharyngeal endoscopy which will be carried out under general anesthesia 6 to 12 months after the graft in case of abnormal pharynx at fibroscopy. In addition, a global neuromuscular examination will be carried out on an annual basis to follow the general evolution of the myopathy. The fibroscopy to determine the swallowing function, the global quantitative tesy of swallowing and the questionnaire will be carried out at 2, 6 and 24 months after the graft. A videofluoroscopy of swallowing will be carried out at 2 months then at 1 and 2 years after the graft.
Risks and Benefits:
The principal benefits to the patients which could be envisioned by this procedure is a delay in the deterioration of the pharyngeal muscles associated with this disease and consequently an improvement in the difficulties in swallowing usually associated with this disease. The potential risk associated with this type of operation are an inflammatory reaction, the appearance of a tumor or the lack of improvement of the symptoms associated with the disease.
A parallel preclinical study has been carried out in the dog in order to evaluate the tolerance and feasibility of the procedure for autografting myoblasts in the pharyngeal muscles.
- Conditions
- Muscular Dystrophy, Oculopharyngeal
- Being tested
- Autologous myoblasts transplantation and myotomy
- Keywords
- 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
- Medical subject headings
- Muscular Dystrophy, Oculopharyngeal, Deglutition Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous myoblasts transplantation and myotomyProcedure or surgery
Also called autograft and surgery
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)
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | All patients included Receives: Procedure: Autologous myoblasts transplantation and myotomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: before the graft and at 2, 6, 12, 18 and 24 months after the graft
Secondary outcomes (1)
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
Measured: before the graft and at 2, 6, 12, 18 and 24 months after the graft
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2008 (estimated)
- Study startApr 2004
- Primary completionJul 2015
- Study completionOct 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Dystrophy, OculopharyngealStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Association Française contre les Myopathies (AFM), Paris
- Responsible party
- Sponsor
- Investigators
- Jean Lacau-Saint Guily, MD, PH, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00773227
- Sponsor's protocol ID
- P020908
- Other IDs
- AOM02100
- Acronym
- OPMD
- Status last verified
- Nov 2015
- First submitted
- 15 Oct 2008
- Last updated
- 20 Nov 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Perie S, Trollet C, Mouly V, Vanneaux V, Mamchaoui K, Bouazza B, Marolleau JP, Laforet P, Chapon F, Eymard B, Butler-Browne G, Larghero J, St Guily JL. Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: a phase I/IIa clinical study. Mol Ther. 2014 Jan;22(1):219-25. doi: 10.1038/mt.2013.155. Epub 2013 Jul 8.
PubMed 23831596
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts
- ClinicalTrials.gov ID
- NCT00773227
- Acronym / sponsor ID
- OPMD / P020908
- Conditions
- Muscular Dystrophy, Oculopharyngeal
- Keywords
- Oculopharyngeal muscular dystrophy, Swallowing disorders, dysphagia, Pharyngeal propulsion, Upper oesophageal sphincter, Cricopharyngeal muscle, Autologous myoblasts, Autograft, Myotomy of the upper oesophageal sphincter
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Association Française contre les Myopathies (AFM), Paris
- Study start
- Apr 2004
- Primary completion
- Jul 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 16 Oct 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
OCULO-Pharyngeal Muscular Dystrophy (OPMD) is characterised by the selective affection of the superior sphincter muscles of the oesophagus (SSO) and the pharyngeal.muscles resulting in dysphagia. The most common treatment for the dysphagia induced by this disease is a myotomy. However, although this will relax the constriction and improve swallowing it will not prevent the progressive degradation of the pharyngeal muscles. This progressive loss of contractility will eventually result in false routes and severe difficulty in swallowing, increased risk of pulmonary infection and severe weight loss which are the most common causes of mortality in these patients.
Concept:
The protocol which we are proposing is a pilot study in which autologous myoblasts isolated from unaffected limb muscles will be grafted into the pharyngeal constrictor muscles of patients diagnosed as suffering from OPMD. Our aim is to improve both swallowing and the contractile deficit generated by the dystrophic pharyngeal constrictor muscles. A myotomy of the SSO will be carried out at the same time as the myoblast transplantation, since we have already validated the improvement resulting from this surgery even though we know that this will provide only a partial and transitory improvement.
Type of trial: This is a multicentric trial with a direct benefit for the patient in which 10 patients will receive an autologous transplantation of myoblasts. Due to the possibility that a certain number of patients may withdraw from the study we have decided to include initially a maximum of 15 patients. The PHRC for this clinical trial was accepted in 2002 and the official promotor is the AP-HP. The patients will be selected by the different promotors but will be followed in the 'Service d'ORL of TENON Hospital '. The proliferative capacity of the myoblasts isolated from the muscle biopsies will be carried out by Vincent MOULY and KAMEL MANCHAOUI in the UMR 7000 CNRS directed by Dr Butler-Browne and the cells will be amplified prior to grafting in the Cellular laboratory therapy of Dr MAROLLEAU; The clinical trial will be controlled by a specifically selected committee of five.
Description of the trial In the first step of this study patients will be selected for inclusion in the trial according to the following criteria : 1: men or women aged between 18 and 75 years ; 2: genetic confirmation and characterisation of OPMD 3: SSO dysfunction confirmed by the stasis of saliva or food above the sphincter visualised by fibroscopy and a defect in the opening of the SSO also confirmed by videofluoroscopy of swallowing; the SSO defect may be associated with a reduction in pharyngeal propulsion as revealed by fibroscopy and videofluoroscopy of swallowing 4: official signed consent of the patients to participate in this clinical trial.
The next step will be the selection of the unaffected muscle to be used for the production of the autologous myoblasts to be used for transplantation. Muscles will be selected for engraftment on the basis of the proliferative capacity of the cultures made from biopsies (0.5-1gm) obtained following local anesthesia of either the quadriceps or the STERNO-CLEIDO-MASTOIDIEN muscles. The muscle which is able to produce the largest number of myoblasts will be biopsied a second time using the same conditions. Two grams of muscle will be removed and the myoblasts will be isolated and cultured until there are about 100 million myoblasts at which point the cells can be injected into the pharyngeal muscles of the patient. If there is no difference between the two different muscle biopsies then we will biopsy preferentially the STERNO-CLEIDO-MASTOIDIEN muscle since it is situated directly in the trajectory that will be used when grafting the myoblasts. The operation by lateral cervicotomy will be carried out under general anesthesia, in order to expose the SSO and the pharyngeal muscles. The graft will be carried out by injecting the myoblasts into about 20 different sites in the pharyngeal constrictor muscles above the site of the associated myotomy of the SSO. The next step will involve a two year evaluation of the trial. For each patient a descriptive analysis will be carried out based on the comparison of the different results. 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. A secondary evaluation will be based 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 of the examination of the pharynx in order to confirm the absence of muscle tumors and a cervical palpation in order to determine the volume of the muscle and to survey the site of myoblasts implantation. A rigid tube pharyngeal endoscopy which will be carried out under general anesthesia 6 to 12 months after the graft in case of abnormal pharynx at fibroscopy. In addition, a global neuromuscular examination will be carried out on an annual basis to follow the general evolution of the myopathy. The fibroscopy to determine the swallowing function, the global quantitative tesy of swallowing and the questionnaire will be carried out at 2, 6 and 24 months after the graft. A videofluoroscopy of swallowing will be carried out at 2 months then at 1 and 2 years after the graft.
Risks and Benefits:
The principal benefits to the patients which could be envisioned by this procedure is a delay in the deterioration of the pharyngeal muscles associated with this disease and consequently an improvement in the difficulties in swallowing usually associated with this disease. The potential risk associated with this type of operation are an inflammatory reaction, the appearance of a tumor or the lack of improvement of the symptoms associated with the disease.
A parallel preclinical study has been carried out in the dog in order to evaluate the tolerance and feasibility of the procedure for autografting myoblasts in the pharyngeal muscles.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Procedure: Autologous myoblasts transplantation and myotomyAll patients included |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (13 listed)
- 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 ;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous myoblasts transplantation and myotomyAlso: autograft and surgeryGraft 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) | Procedure or surgery | 1 |
Primary outcome
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
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2015.
- Derived from this study: PubMed 23831596Perie S, Trollet C, Mouly V, Vanneaux V, Mamchaoui K, Bouazza B, Marolleau JP, Laforet P, Chapon F, Eymard B, Butler-Browne G, Larghero J, St Guily JL. Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: a phase I/IIa clinical study. Mol Ther. 2014 Jan;22(1):219-25. doi: 10.1038/mt.2013.155. Epub 2013 Jul 8.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jean Lacau-Saint Guily, MD, PH principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Autologous myoblasts transplantation and myotomy in people with Muscular Dystrophy, Oculopharyngeal, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00773227, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2015.