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Efficacy and Safety of Radiofrequency Pallidotomy in the Management of Dystonia

Primary Purpose

Dystonia

Status
Not yet recruiting
Phase
Not Applicable
Locations
India
Study Type
Interventional
Intervention
Radiofrequency Pallidotomy
Sponsored by
All India Institute of Medical Sciences, New Delhi
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dystonia focused on measuring Dystonia, Segmental dystonia, Generalized dystonia

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patients with generalized, segmental or focal dystonias who are being considered for simultaneous bilateral radiofrequency pallidotomy. Of all ages and gender Exclusion Criteria: Those who deny consent Pregnant ladies and women of childbearing potential without adequate contraception Those who have undergone botulinum toxin injection in the last 12 weeks or those who are being planned for the same in the upcoming 12 weeks.

Sites / Locations

  • All India Institute of Medical Sciences, New Delhi

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Early intervention arm

Delayed intervention arm

Arm Description

Radiofrequency Pallidotomy at earliest available date

Radiofrequency pallidotomy offered at 12 weeks following agreeing to participate in the study

Outcomes

Primary Outcome Measures

BFM Dystonia disability scale
To compare the BFM Dystonia disability scale 6 weeks following bilateral simultaneous radiofrequency pallidotomy (early treatment arm) with the BFM Dystonia disability scale 6 weeks following acceptance of the procedure in those who were randomized to undergo the procedure atleast 12 weeks after accepting the the procedure (delayed treatment arm)
Adverse effects: New onset or worsening Dysphagia or dysarthria
To study the incidence of new onset or worsening dysphagia or dysarthria in patients with dystonia undergoing bilateral simultaneous pallidotomy

Secondary Outcome Measures

20% improvement in BFM Dystonia disability scale
To compare the proportion of patients having atleast 20% improvement in BFM Dystonia disability scale from baseline to 6 weeks following bilateral simultaneous radiofrequency pallidotomy(early treatment arm) with the proportion of patients with similar improvement from baseline to 6 weeks following acceptance of procedure in those who undergo surgery atleast 12 weeks after accepting the procedure (delayed treatment arm)
20% improvement in BFM Dystonia movement scale
To compare the proportion of patients having atleast 20% improvement in BFM Dystonia movement scale from baseline to 6 weeks following bilateral simultaneous radiofrequency pallidotomy (early treatment arm) with the proportion of patients with similar improvement from baseline to 6 weeks following acceptance of procedure in those who undergo surgery atleast 3 months after accepting the procedure (delayed treatment arm)
Adverse effects
To study the proportion of patients in early and delayed treatment arm who develop adverse effects during the 12 weeks following surgery or agreeing for surgery respectively.

Full Information

First Posted
September 8, 2023
Last Updated
September 8, 2023
Sponsor
All India Institute of Medical Sciences, New Delhi
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1. Study Identification

Unique Protocol Identification Number
NCT06038097
Brief Title
Efficacy and Safety of Radiofrequency Pallidotomy in the Management of Dystonia
Official Title
Efficacy and Safety of Radiofrequency Pallidotomy in the Management of Dystonia - A Delayed Start Randomized Controlled Trial
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
October 1, 2023 (Anticipated)
Primary Completion Date
December 31, 2026 (Anticipated)
Study Completion Date
March 31, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
All India Institute of Medical Sciences, New Delhi

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Generalized dystonia is treated with pallidotomy. This is based on observational data which is significantly limited by publication bias and there are no RCTs. The case reports focus on successful outcomes and case series have an inherent selection bias. Bilateral pallidotomy has been used in our institute in a series of patients with generalized and segmental dystonia and have been seen to show good efficacy. However, the existing literature suggests that it is also associated with dysphagia and dysarthria in some cases and thus simultaneous bilateral pallidotomy is not preferred in several centres. However, our center routinely performs simultaneous bilateral pallidotomy. The response rates and compliations of the procedure have not been systematically studied in RCT and we need to generate data on the efficacy and safety of Pallidotomy on generalized and segmental dystonia. This randomized controlled trial will fill the void in knowledge in this field.
Detailed Description
Deep brain stimulation surgery (DBS) has become the standard of care for the management of generalized dystonia It has been shown in Randomized controlled trials to be effective and safe.1-3 Since the last 30 years, DBS has been used to treat a variety of patients with dystonias, which are of varying etiologies. However, the DBS is an expensive system, needs hardware implantation and regular programming. It is associated with surgical implantation which may lead to complications such as infection of the hardware, leads and electrodes. Since it is expensive, it is out of reach for the majority of the patients who pay out of pocket. It leads to increased healthcare costs because of the initial expenses for the implant as well as regular follow ups for programming. The programming is currently labour intensive and needs long hours of observation with trial and error to find the optimum programming settings for the individual patient. This may need the patient to be admitted in the inpatient setting or multiple outpatient visits for the patient, which can lead to loss of working hours and burden on the healthcare system. Before the introduction of DBS by Benabid, functional neurosurgery with lesioning procedures such as pallidotomy, thalamotomy, subthalamotomy were used to manage movement disorders such as dystonia, tremors and Parkinson disease. However after the advent of DBS, these procedures have taken a backseat. Also there are no randomized trials for the safety and efficacy of lesioning procedures in dystonias. The current practice in various centers of the world is to do unilateral pallidotomy in most cases of dystonia. Simultaneous bilateral pallidotomy is not preferred by several practitioners due to the risk of bulbar symptoms which may be irreversible in certain cases. However, our experience in our Institute suggests good outcomes in patients with various forms of focal and generalized dystonias undergoing simultaneous bilateral pallidotomy using radiofrequency ablation. Similarly we have done pallidotomy for genetic dystonias such as NBIAs, Wilson's disease who had disabling dystonias or medically refractory status dystonicus.4 However, the long term efficacy and safety of these patients is not clear as there are no registries or long term follow up data. We need a well conducted Randomized controlled trial to systematically collect data on the efficacy and safety of the intervention to generate meaningful information for patients and clinicians to understand the risks and benefits of the procedures and offer relevant information for clinical decision making. Problem statement Evidence based management requires robust data to make clinical decisions. This data is derived from studies which may be observational or interventional. In the evidence pyramid, meta-analysis of randomized controlled trials is considered to be of the highest quality of evidence. Generalized dystonia is treated with pallidotomy. This is based on observational data which is significantly limited by publication bias and there are no RCTs. The case reports focus on successful outcomes and case series have an inherent selection bias. Bilateral pallidotomy has been used in our institute in a series of patients with generalized and segmental dystonia and have been seen to show good efficacy. However, the existing literature suggests that it is also associated with dysphagia and dysarthria in some cases and thus simultaneous bilateral pallidotomy is not preferred in several centres. However, our center routinely performs simultaneous bilateral pallidotomy. The response rates and compliations of the procedure have not been systematically studied in RCT and we need to generate data on the efficacy and safety of Pallidotomy on generalized and segmental dystonia. This randomized controlled trial will fill the void in knowledge in this field. Review of literature There are no randomized controlled trials to study the efficacy and safety of pallidotomy or thalamotomy in the management of dystonia. The case series and case reports regarding pallidotomy in dystonia describe a heterogenous population of patients who had undergone the procedure.5-10 Most of the case series have a bias towards reporting favourable outcomes. We found a meta-analysis of 100 patients who underwent bilateral procedures for dystonia.11 This meta-analysis described 33 studies with varying indications such as generalized dystonia, dystonic storm and focal dystonias. The majority of the patients had genetic causes of dystonia with DYT1 being the most common known genetic cause. Some studies reported a staged bilateral procedures while some simultaneous bilateral pallidotomy. The median time of follow up was 12 months with a range of 2-180 months. This meta-analysis revealed that 8% patients had transient adverse effects while 11% had permanent adverse effects. The commonest adverse effects that were permanent involved bulbar dysfunction with dysarthria anarthria or mutism. Most of the patients had a lasting beneficial effect while 19% patients had relapse of symptoms on prolonged follow up. The time to relapse of symptoms ranged from 3 weeks to 4.5 years. The predictors of response or relapse were not apparent from this meta-analysis. Moreover, why pallidotomy was preferred in contrast to DBS is not clear from the reports. Another case series describing 89 patients who underwent radiofrequency pallidotomy suggested unacceptable rates of complications with bilateral pallidotomy such as medically refractory parkinsonism, dysarthria and dysphagia.12 However these are retrospective review of data and the adverse effects may have been selectively reported in both unilateral and bilateral cases. In our center 10 children underwent bilateral simultaneous pallidotomy over a 7 year period and it was found that two patients had undergone the procedure for medically refractory status dystonicus and both of them had resolution of the status dystonicus. Two patients with generalized dystonia had recurrence of symptoms over a follow up of 4.5 years while three had a sustained improvement of more than 40% improvement in BFMDRS.4

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dystonia
Keywords
Dystonia, Segmental dystonia, Generalized dystonia

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
Outcomes Assessor
Allocation
Randomized
Enrollment
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Early intervention arm
Arm Type
Experimental
Arm Description
Radiofrequency Pallidotomy at earliest available date
Arm Title
Delayed intervention arm
Arm Type
No Intervention
Arm Description
Radiofrequency pallidotomy offered at 12 weeks following agreeing to participate in the study
Intervention Type
Procedure
Intervention Name(s)
Radiofrequency Pallidotomy
Intervention Description
Radiofrequency bilateral simultaneous pallidotomy
Primary Outcome Measure Information:
Title
BFM Dystonia disability scale
Description
To compare the BFM Dystonia disability scale 6 weeks following bilateral simultaneous radiofrequency pallidotomy (early treatment arm) with the BFM Dystonia disability scale 6 weeks following acceptance of the procedure in those who were randomized to undergo the procedure atleast 12 weeks after accepting the the procedure (delayed treatment arm)
Time Frame
6 weeks
Title
Adverse effects: New onset or worsening Dysphagia or dysarthria
Description
To study the incidence of new onset or worsening dysphagia or dysarthria in patients with dystonia undergoing bilateral simultaneous pallidotomy
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
20% improvement in BFM Dystonia disability scale
Description
To compare the proportion of patients having atleast 20% improvement in BFM Dystonia disability scale from baseline to 6 weeks following bilateral simultaneous radiofrequency pallidotomy(early treatment arm) with the proportion of patients with similar improvement from baseline to 6 weeks following acceptance of procedure in those who undergo surgery atleast 12 weeks after accepting the procedure (delayed treatment arm)
Time Frame
6 weeks
Title
20% improvement in BFM Dystonia movement scale
Description
To compare the proportion of patients having atleast 20% improvement in BFM Dystonia movement scale from baseline to 6 weeks following bilateral simultaneous radiofrequency pallidotomy (early treatment arm) with the proportion of patients with similar improvement from baseline to 6 weeks following acceptance of procedure in those who undergo surgery atleast 3 months after accepting the procedure (delayed treatment arm)
Time Frame
6 weeks
Title
Adverse effects
Description
To study the proportion of patients in early and delayed treatment arm who develop adverse effects during the 12 weeks following surgery or agreeing for surgery respectively.
Time Frame
12 weeks

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients with generalized, segmental or focal dystonias who are being considered for simultaneous bilateral radiofrequency pallidotomy. Of all ages and gender Exclusion Criteria: Those who deny consent Pregnant ladies and women of childbearing potential without adequate contraception Those who have undergone botulinum toxin injection in the last 12 weeks or those who are being planned for the same in the upcoming 12 weeks.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Arunmozhimaran Elavarasi, MD DM
Phone
+919013844274
Email
arun_ela@yahoo.com
First Name & Middle Initial & Last Name or Official Title & Degree
Padma Srivastava MV, MD DM
Phone
+919868398261
Email
vasanthapadma123@gmail.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Arunmozhimaran Elavarasi, MD DM
Organizational Affiliation
All India Institute of Medical Sciences, New Delhi
Official's Role
Principal Investigator
Facility Information:
Facility Name
All India Institute of Medical Sciences, New Delhi
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110029
Country
India

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
De-identified patient data will be shared on approaching through proper channel through ethics committee AIIMS, New Delhi
IPD Sharing Time Frame
Will become available after the project is completed, will be available for 5 years atleast after completition of the trial
IPD Sharing Access Criteria
De-identified patient data will be shared on approaching through proper channel through ethics committee AIIMS, New Delhi

Learn more about this trial

Efficacy and Safety of Radiofrequency Pallidotomy in the Management of Dystonia

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