Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation (BMT) in Patients With Multiple Sclerosis (MS)

Multiple Sclerosis Clinical Trial

Official title:

Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation in Patients With Multiple Sclerosis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02529839
• Other study ID #: 0105-15-HMO-CTIL
• Status: Not yet recruiting
• Phase: N/A
• First received: June 10, 2015
• Last updated: August 19, 2015
• Start date: October 2015
• Est. completion date: October 2017

Study information

• Verified date: April 2015
• Source: Hadassah Medical Organization
• Contact: Igor Resnick, Prof.
• Phone: 972-50-787-4663
• Email: gashka.resnick[@]gmail.com
• Is FDA regulated: No
• Health authority: Israel: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety and efficacy of an immunoablative nonmyeloablative conditioning protocol for autologous bone marrow transplantation in patients with Multiple Sclerosis. Patients meeting inclusion and exclusion criteria will start an immunoablative nonmyeloablative conditioning regimen followed by autologous bone marrow transplantation. Patients will be followed for one year by a neurologist to evaluate the course of the disease after treatment.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: October 2017
• Est. primary completion date: October 2017
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

1. Consenting patients fulfilling the Poser's clinical criteria for definite MS

2. Age: 18-65, males and females

3. Relapsing and secondary progressive forms of MS with evidence of significant activity of MS (clinical and on the MRI).

4. EDSS score of 2.0 to 7.0 (see table 1).

5. Failure to at least one line of the currently available treatment, registered treatments (i.e. interferons, Copaxone, Tysabri, Gilenya, Tecfidera, immunosuppression) for MS. The lack of response to these treatments will be determined/defined by either an increase (deterioration) of one degree (or more) in the EDSS score, when baseline EDSS is less than 5.0 or 0.5 degree, when baseline EDSS is 5.0 or more, during the last year or the appearance of one major relapse of MS during the same period of time (under treatment), or evidence for new activity of MS (new T2 lesions or gadolinium enhancing lesions) during the last 12 months.

6. Duration of disease: >2 years, except cases with rapid progression, i.e. annual relapse rate =2 per 2 years on a conventional treatment or malignant multiple sclerosis with very intense symptoms (types is in most cases deadly).

Exclusion Criteria:

1. Patients suffering from significant cardiac, renal or hepatic failure or any other disease that may risk the patient or interfere with the ability to undergo high dose immunosuppression associated toxicities (according to the existing limitations for autologous transplantation).

2. Patients with active infections.

3. Patients with severe cognitive decline or inability to understand and sign the informed consent.

4. Patients who were treated with investigational protocols during the last 3 months prior to the inclusion.

5. Patients who received high dose immunosuppression with autologous stem cell rescue in the past with no effect.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Multiple Sclerosis
• Sclerosis

Intervention

Procedure:
• Autologous bone marrow transplantation
IMMUNOABLATIVE NONMYELOABLATIVE AUTOLOGOUS BONE MARROW TRANSPLANTATION on Day 0

Drug:
• Fludarabine
30mg/m2 on days -6 through -3
• Cyclophosphamide
50mg/kg on days -5 through -4
• Alemtuzumab
3mg on day -3, 9mg on day -2, 12 mg on day -1

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Hadassah Medical Organization

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Engraftment parameters of Neutrophils and Platelets	Absolute Neutrophil count >500 /microliter, Platelets>20,000/microliter	1 year	Yes
Primary	Transplant related mortality by Day 100		Day 100	Yes
Secondary	Changes in the Expanded Disability Status Scale (EDSS score, as compared to baseline)		1 year	No
Secondary	Overall survival (OS) at 1 year		1 year	No
Secondary	Progression-free survival (PFS) at 1 year		1 year	No
Secondary	Changes in MRI activity	T2 number	1 year	No
Secondary	Changes in MRI activity	Volume of gadolinium enhancing lesions	1 year	No