Clinical Trial Details
— Status: Available
Administrative data
| NCT number |
NCT05592028 |
| Other study ID # |
DoN-001 |
| Secondary ID |
|
| Status |
Available |
| Phase |
|
| First received |
|
| Last updated |
|
Study information
| Verified date |
August 2022 |
| Source |
University of the Philippines Manila - Philippine General Hospital |
| Contact |
Roland Dominic G Jamora, MD, PhD |
| Phone |
+63 2 85242338 |
| Email |
rgjamora[@]up.edu.ph |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Expanded Access
|
Clinical Trial Summary
X-linked dystonia-parkinsonism (XDP) is a rare, X-linked, adult-onset, and progressive
movement disorder seen almost exclusively in men from Panay Island in the Philippines. The
disease is associated with mutations involving the DYT3/TAF1 gene, and all the cases
described so far have been linked to Filipino ancestry. Although XDP is very rare globally,
the prevalence is 5.74 per 100,000 individuals in Panay Island and 0.31 per 100,000 in the
Philippines as a whole. Majority of patients (95%) were males, and the mean age of onset was
39 years. The mean duration of illness was 16 years, and the mean age of death was 55.6
years.
Description:
To determine the outcomes of MRgFUS pallidothalamic tractotomy on adult XDP patients at the
Philippine General Hospital.
This is a prospective study that aims to determine the outcomes of MRgFUS pallidothalamic
tractotomy (both unilateral and bilateral) on adult XDP patients at the Philippine General
Hospital.
No sample size will be determined a priori. The study will be conducted over a period of five
years, and all eligible patients may be invited to join the study during this period.
The procedure will be performed inside the MRI suite, using a 1.5 Tesla MRI system (GE Optima
MR450, USA) and the ExAblate Neuro device (InSightec, Haifa, Israel) to deliver the focused
ultrasound. The patient's head will be shaved and immobilized using a stereotactic frame
under local anesthesia. An elastic diaphragm will be attached to the patient's head and
connected to the ultrasound transducer,8 and the patient will be positioned inside the bore
of the MRI. A reference MRI scan will be performed to position the transducer to the target,
after which the MRI images (T1-weighted axial and T2-weighted axial/coronal MRI at 1 mm
slices) will be fused to the preoperative CT scan for the skull-correction algorithm.8
Stereotactic treatment planning will then be performed to target the pallidothalamic tract.
It is usually located at the midcommisural line (0 to 1.0 mm posterior) in the
anterior-posterior direction, 8-10 mm lateral to the intercommisural line in the mediolateral
direction, 0-1.5 mm inferior to the intercommisural line in the dorsoventral direction, and
6.5-7.5 mm to the thalamo-ventricular border.7 The lesion will be made 3 mm lateral to the
mamillo-thalamic tract and superior and medial to the medial subthalamic nucleus.9,11,12 Once
the target is set, a series of low-power sonications producing temperatures between 40-45oC
and lasting 10-20 seconds will be performed.8 Clinical assessment for tremor suppression and
treatment side effects will be made after each sonication,8 and definitive non-reversible
thermal ablation will be performed only at the setting wherein the patient experiences tremor
reduction or suppression with no adverse effects.9 The final sonication temperature usually
ranges between 55-63oC.8 After the treatment, the stereotactic frame will be removed and the
patient will be monitored in the hospital overnight.
The patients will be assessed within 24 hours of treatment then asked to follow up with the
movement disorder neurologist (RDJ) at 2 weeks, 3 months, 6 months, 9 months, and 12 months
post-treatment through a clinic visit. Video recording will be taken on initial visit and at
each follow up visit. Another blinded assessor will score the videos of the patients. The
outcome will be assessed using the XDP-Movement Disorder Society of the Philippines
(XDP-MDSP) Scale at each visit, and comparing it to the pre-treatment baseline, as well as
using the XDP Clinical and Functional Staging. The pre- and post-treatment BFMDRS and UPDRS
scores will also be obtained.
For bilateral MRgFUS tractotomy, the contralateral lesion will be done at least 6 months
after the first procedure, provided that there are no serious adverse effects from the first
PTT lesioning.
Demographic, clinical, treatment-related, and outcome data of all study patients will be
collected prospectively by the investigators throughout the duration of the study.
