Trial Outcomes & Findings for Posterior Fossa Decompression With or Without Duraplasty for Chiari Type I Malformation With Syringomyelia (NCT NCT02669836)

Last Updated: 2024-10-31

Results Overview

The number of participants with complications such as: Cerebrospinal fluid (CSF)-related complications ≤6 months (e.g. CSF leak, pseudomeningocele, aseptic meningitis, infection, hydrocephalus), and the requirement for additional surgery for wound revision or CSF diversion

Recruitment status

COMPLETED

Study phase

Target enrollment

162 participants

Primary outcome timeframe

less then or equal to 6 months from decompression

Results posted on

2024-10-31

Participant Flow

Participant milestones

Participant milestones
Measure	Posterior Fossa Decompression Surgery The bone is surgically removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected Posterior fossa decompression: Planned areas of bone removal from the suboccipital region of the skull and cervical l1 lamina. Then, the constricting epidural band at the level of the foramen magnum is resected.	Dural Augmentation Surgery The bone is removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected. Then, the dura is opened. Microsurgical dissection is performed and the dura is sewn closed. Dural Augmentation: The dura is opened sharply, exposing the cerebellar tonsils, brainstem, and upper spinal cord. After microsurgical dissection, the dura is sewn closed with a dural graft.
Overall Study STARTED	84	78
Overall Study 3-6 Month Follow up	60	63
Overall Study COMPLETED	53	61
Overall Study NOT COMPLETED	31	17

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Posterior Fossa Decompression With or Without Duraplasty for Chiari Type I Malformation With Syringomyelia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Posterior Fossa Decompression Surgery n=84 Participants The bone is surgically removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected Posterior fossa decompression: Planned areas of bone removal from the suboccipital region of the skull and cervical l1 lamina. Then, the constricting epidural band at the level of the foramen magnum is resected.	Dural Augmentation Surgery n=78 Participants The bone is removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected. Then, the dura is opened. Microsurgical dissection is performed and the dura is sewn closed. Dural Augmentation: The dura is opened sharply, exposing the cerebellar tonsils, brainstem, and upper spinal cord. After microsurgical dissection, the dura is sewn closed with a dural graft.	Total n=162 Participants Total of all reporting groups
Age, Categorical <=18 years	80 Participants n=5 Participants	74 Participants n=7 Participants	154 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	9.94 years STANDARD_DEVIATION 5.59 • n=5 Participants	10.77 years STANDARD_DEVIATION 5.37 • n=7 Participants	10.34 years STANDARD_DEVIATION 5.48 • n=5 Participants
Sex: Female, Male Female	48 Participants n=5 Participants	39 Participants n=7 Participants	87 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants	39 Participants n=7 Participants	75 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants	9 Participants n=7 Participants	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	78 Participants n=5 Participants	69 Participants n=7 Participants	147 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants	10 Participants n=7 Participants	15 Participants n=5 Participants
Race (NIH/OMB) White	72 Participants n=5 Participants	59 Participants n=7 Participants	131 Participants n=5 Participants
Race (NIH/OMB) More than one race	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Size of Syringomyelia Syrinx AP Diameter (mm) 3-6 mm	65 Participants n=5 Participants	37 Participants n=7 Participants	102 Participants n=5 Participants
Size of Syringomyelia Syrinx AP Diameter (mm) 7-9 mm	19 Participants n=5 Participants	41 Participants n=7 Participants	60 Participants n=5 Participants

PRIMARY outcome

Timeframe: less then or equal to 6 months from decompression

Population: Note: values represent reporting by ITT, i.e. split by randomization, not necessarily treatment received.

Outcome measures

Outcome measures
Measure	Posterior Fossa Decompression Surgery n=84 Participants The bone is surgically removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected Posterior fossa decompression: Planned areas of bone removal from the suboccipital region of the skull and cervical l1 lamina. Then, the constricting epidural band at the level of the foramen magnum is resected.	Dural Augmentation Surgery n=78 Participants The bone is removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected. Then, the dura is opened. Microsurgical dissection is performed and the dura is sewn closed. Dural Augmentation: The dura is opened sharply, exposing the cerebellar tonsils, brainstem, and upper spinal cord. After microsurgical dissection, the dura is sewn closed with a dural graft.
The Number of Participants With Surgical Complications	8 Participants	10 Participants

SECONDARY outcome

Timeframe: 10-14 months

Population: Note: values represent reporting by ITT, i.e. split by randomization, not necessarily treatment received

Number of patients who experienced improvement in clinical or neurological symptoms-such as; headaches, pain, numbness or tingling, nausea/ vomiting, itching, weakness, difficulty swallowing, difficulty sleeping and cranial nerve assessments.

Outcome measures

Outcome measures
Measure	Posterior Fossa Decompression Surgery n=53 Participants The bone is surgically removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected Posterior fossa decompression: Planned areas of bone removal from the suboccipital region of the skull and cervical l1 lamina. Then, the constricting epidural band at the level of the foramen magnum is resected.	Dural Augmentation Surgery n=61 Participants The bone is removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected. Then, the dura is opened. Microsurgical dissection is performed and the dura is sewn closed. Dural Augmentation: The dura is opened sharply, exposing the cerebellar tonsils, brainstem, and upper spinal cord. After microsurgical dissection, the dura is sewn closed with a dural graft.
Aim 2: The Number of Patients With Clinical Improvement	43 Participants	43 Participants

SECONDARY outcome

Timeframe: 10-14 months

Population: Values represent reporting by ITT not treatment received

Syrinx regression comparison between posterior fossa decompression surgery and dural augmentation surgery cohorts based on ITT.

Outcome measures

Outcome measures
Measure	Posterior Fossa Decompression Surgery n=52 Participants The bone is surgically removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected Posterior fossa decompression: Planned areas of bone removal from the suboccipital region of the skull and cervical l1 lamina. Then, the constricting epidural band at the level of the foramen magnum is resected.	Dural Augmentation Surgery n=60 Participants The bone is removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected. Then, the dura is opened. Microsurgical dissection is performed and the dura is sewn closed. Dural Augmentation: The dura is opened sharply, exposing the cerebellar tonsils, brainstem, and upper spinal cord. After microsurgical dissection, the dura is sewn closed with a dural graft.
Aim 2: The Mean Number of Syrinx Reduction in Millimeters Less Than 24 Months Post Surgery.	1.06 millimeters Standard Deviation 1.73	3.07 millimeters Standard Deviation 2.47

OTHER_PRE_SPECIFIED outcome

Timeframe: 10-14 Months from Decompression

Population: Note: values represent reporting by ITT, i.e. split by randomization, not necessarily treatment received

Quality of Life (QOL) was assessed using the Chiari Health Index Pediatrics (CHIP), and the Health Utilities-3 (HUI-3). The CHIP is a 5-point response scale. Within the physical symptoms' domain, 5 questions regarding pain severity are alternatively scaled. The Health Utilities Index Mark 3 (HUI3) is a generic status classification system, generalizable in pediatric patients with illness. The HUI3 assesses domains including vision, hearing, speech, ambulation, dexterity, emotion, cognition, and pain. We used a non-inferiority approach in the analysis. The score is corrected for missing values and scaled to a range of scores between 0 and 1. An increasing score represents increasing HRQOL. Increase/decrease in the quality of life metric (on a scale from 0-1, with 0 representing no quality of life and 1 representing 100% quality of life).

Outcome measures

Outcome measures
Measure	Posterior Fossa Decompression Surgery n=28 Participants The bone is surgically removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected Posterior fossa decompression: Planned areas of bone removal from the suboccipital region of the skull and cervical l1 lamina. Then, the constricting epidural band at the level of the foramen magnum is resected.	Dural Augmentation Surgery n=29 Participants The bone is removed from the suboccipital region of the skull and Cervical 1 lamina so the constricting epidural band can be resected. Then, the dura is opened. Microsurgical dissection is performed and the dura is sewn closed. Dural Augmentation: The dura is opened sharply, exposing the cerebellar tonsils, brainstem, and upper spinal cord. After microsurgical dissection, the dura is sewn closed with a dural graft.
Aim 3 : Mean Change in Quality of Life Measurement After Surgery Relative to Baseline.	0.02 score on a scale Standard Deviation 0.16	0.04 score on a scale Standard Deviation 0.17

Adverse Events

Posterior Fossa Decompression Surgery

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Dural Augmentation Surgery

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

David D. Limbrick MD

Washington University

Phone: 314-454-3212

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place