Autologous Stem Cell Transplant in other Neuro-Inflammatory diseases

Phase 2

• Conditions: euromyelitis Optica; Stiff Person Syndrome; Myasthenia Gravis; Central Nervous System Vasculitis; Neuromyelitis Optica; Inflammatory and Immune System - Other inflammatory or immune system disorders; Inflammatory and Immune System - Autoimmune diseases; Neurological - Other neurological disorders

• Registration Number: ACTRN12622000530729
• Lead Sponsor: St Vincent's Hospital, Sydney

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-04-04
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

HSCT patients with NID
- Age 18-65
- Age 65-70 (may be considered only if HCT-CI<3 and deemed fit both physically and cognitively by at least two investigators)
- Adequate organ function as measured by:
oCardiac LV Ejection Fraction > 45%
oTotal Lung Capacity > 60%
oDLCO/VA > 50%.
oNegative serology for active HBV, active HCV and HIV.
oNegative CT skeletal survey in patients with 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 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