Observational Study in Multiple Sclerosis Patients Treated With Autologous Hematopoietic Stem Cell Transplantation

NCT04674280 | Unknown

• 1 other identifier: 8410-016
• Study Type: observational
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

Observational prospective , multi-center study Primary objective : To gain further homogenous evidence for clinical efficacy of aHSCT in patients undergoing aHSCT for MS as primary indication. Secondary objectives:

• Safety, tolerability and toxicity of aHSCT in MS
• Quality of life and long-term disability after aHSCT
• MRI outcome after aHSCT Primary endpoint : Time to failure to maintain a NEDA status Secondary endpoints:
• Overall survival
• Transplant related mortality
• MRI Assessment including lesions
• Treatment-related complications . • Quality of life through the MS QL 54 standard assessment
• Improvement of disability Inclusion criteria:
• Diagnosis of MS according to the 2010 revision McDonald's criteria
• Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, previous treatments administered
• Patients aged 18yrs or over at the time of the first aHSCT Exclusion criteria:
• Lack of one of the above criteria
• Physical, mental, or social condition which could affect the patient from returning for follow-up visits
• Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care. Recruitment: 50 patients Recruitment period: 2 years starting from the inclusion of the 1st patient Follow-up duration: 2 years

Conditions

Multiple Sclerosis

Outcome Measures

Primary Outcomes (1)

• Time to NEDA-3 failure

  NEDA-3 is defined as a composite endpoint including survival without relapses, disability progression and MRI activity (new or enlarging T2 lesions or Gd-enhancing lesions on MRI).

  4 years

Secondary Outcomes (2)

• Overall survival

  4 years

• Transplant related mortality

  4 years

Interventions

aHSCT PROCEDURE

Autologous haematopoietic stem cell transplantation

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients wiht multiple sclerosis treated with autologous heamatopoietic stem cell transplantation

You may qualify if:

• Diagnosis of MS according to McDonald's criteria
• Availability of a detailed clinical history about MS, including progression of disability and relapse rate in the previous 2 years, treatments administered before aHSCT, and categorization of the sub-types of MS according to current EBMT guidelines
• Highly active relapsing remitting MS failing DMTs
• Progressive MS with active inflammatory component. In accordance with the EBMT guidelines, this category includes both secondary and primary progressive MS with active inflammatory component.
• Aggressive (malignant) MS not previously treated with a full course of DMT
• Patients aged 18 or over at time of first aHSCT
• Signed informed consent

You may not qualify if:

• Lack of one of the above criteria
• Progressive MS without active inflammatory component, as this indication is assigned 'GNR' according to EBMT guidelines, irrespective of whether it is secondary or primary progressive.
• Unwillingness or inability to comply with the requirements of this protocol including the presence of any condition (physical, mental, or social) that is likely to affect the patient be able to return for the scheduled follow-up visits.
• Patients with cognitive impairments, who are unable to provide written, informed consent prior to any testing under this protocol, including screening and baseline investigations that are considered part of routine patient care.

Sponsors & Collaborators

• European Society for Blood and Marrow Transplantation: lead

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Riccardo Saccardi, MD

  European Society for Blood and Marrow Transplantation

  PRINCIPAL INVESTIGATOR

Central Study Contacts

EBMT ADWP- OMST study coordinator

CONTACT

+33.1.70.64.24.16; manuela.badoglio@upmc.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 14, 2020
• First Posted: December 19, 2020
• Study Start: January 1, 2021
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: December 21, 2020

Record last verified: 2020-12

Data Sharing

• IPD Sharing: Will not share