Autologous Stem Cell Transplant in Neuro-Inflammatory diseases other than multiple sclerosis

Not Applicable

Not yet recruiting

• Conditions: euro-Inflammatory disease; Neuro-Inflammatory disease; Neurological - Other neurological disorders

• Registration Number: ACTRN12622001186741
• Lead Sponsor: The Alfred- Melbourne

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-09-05
• Last Update Posted: 12 September 2022

Recruitment & Eligibility

• Status: Not yet recruiting
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

•Age 18-65
oAge 65-70 (may be considered only if HCT-CI (Haemopoietic cell transplantation – comorbidity index) <3 and deemed fit both physically and cognitively by at least two investigators)
•Adequate organ function as measured by:
oCardiac Left Ventricle Ejection Fraction greater than 45%
oTotal Lung Capacity of at least 60%
oDLCO/VA (Diffusing Capacity Of The Lungs For Carbon Monoxide) of at least 50%.
oNegative serology for active Hepatitis B, active Hepatitis C and Human Immunodeficiency Virus.
oNegative CT skeletal survey in patients with Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and a para-protein
oSerological assessments of haematology, liver, kidney and thyroid function reviewed by transplant physician and specialty input sought were required.
•No evidence of chronic infection or significant systemic illness where a treating specialist has concerns about HSCT.
•Clearance from treating physician in the case of prior or co-existent malignancy
•No current history of substance abuse (drug or alcohol) or other factor (eg: serious psychiatric impairment) that may interfere with patient’s ability to comply with the study procedure and follow up.
•Negative pregnancy test.
•Sperm collection or ova cryopreservation is to be offered prior to HSCT in those of child-bearing age.
•Patients must agree to use a form of effective contraception (either i.e. partner) during and for 3 months after HSCT (females that are either post-menopausal for 12 months prior to randomization or surgically sterile [through hysterectomy or bilateral oophorectomy] are not required to use birth control).
•Able to provide informed consent and the absence of mental and cognitive deficits which can interfere with the capability of providing the informed consent.
•AHSCT deemed an appropriate high-intensity immunotherapeutic treatment in the opinion of the referring physician.
•Published data to support the role of AHSCT for the disease.
•Suitability for AHSCT will be determined by a multidisciplinary HSCT panel including a neurologist and haematologists/transplant physicians.
•If suitability is contended an expert opinion from and alternate national or international centre involved in AHSCT for AID may be sought.

Exclusion Criteria

•Any patient during the screening phase whilst being considered for HSCT arm who does not meet inclusion criteria
•Any patient on the study treatment arm deemed not suitable for transplant by a consensus of HSCT specialists as determined at the HSCT MDT.
•Any patient unable to understand the purpose and risks of the study or adhere to the post-transplant management including medication adherence and appointment attendance.
•Patients with a predominately progressive form of disease.
•Patients where mimics have not been adequately excluded.
•Patients unable to undergo MRI scans.
•Patients with advanced NID where the risks of transplant are deemed to outweigh potential benefits.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The primary objective of this submission is to assess safety and tolerability of HSCT in NID. This will be determined by length of stay in hospital from day 0 until discharge as well as time to engraftment (number of days from stem cell infusion until neutrophil engraftment). This will be assessed by inpatient medical records at the Alfred Hospital. The Investigators will be involved with patient discharge. Engraftment will be assessed by blood tests and physical exam during hospital admission by investigators. <br><br>This is a composite primary objective.<br><br><br><br>[One-off intervention with post discharge follow ups for 10 years. Assessed at time of discharge, then at follow up visits will be performed 3 months, 6 months, and 12 months post transplant then yearly until 10 years post transplant. ]