Trial Outcomes & Findings for Hematopoietic Stem Cell Therapy for Patients With Multiple Sclerosis (NCT NCT00278655)

Results Overview

Data are reporting number of participants with disease progression. Disease progression is defined as a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 3 months apart.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

21 participants

Primary outcome timeframe

3 years after transplant

Results posted on

2014-05-01

Participant Flow

Between January 2003 and February 2005 in Northwestern Memorial Hospital 21 patients with relapsing-remitting multiple sclerosis (MS) underwent autologous hematopoietic stem cell transplantation in order to evaluate the safety and clinical outcome of autologous non-myeloablative hematopoetic stem cell transplantation in MS.

21 eligible patients had relapsing remitting MS not responding to interferon and had had two corticosteroid -treated relapses within the previous 12 months or one relapse and gadolinium-enhancing lesions seen on MRI and separate from the relapse.

Participant milestones

Participant milestones
Measure	Autologous Hematopoietic Stem Cell Transplantation Patient's own blood stem cells collected after mobilization with cyclophosphamide (2.0 gm/m²)and granulocyte colony-stimulating factor (G-CSF) (5-10mcg/kg/day.Collected stem cells given back to the patient after receiving conditioning regimen (Cyclophosphamide 50 mg/kg/day for four days and either 20 mg alemtuzumab or 6mg/kg rabbit antithymocyte globulin.
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Hematopoietic Stem Cell Therapy for Patients With Multiple Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stem Cell Transplantation n=21 Participants All participants will undergo stem cell transplantation after receiving conditioning regimen. hematopoietic stem cell transplantation : Autologous hematopoietic stem cell transplantation
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	21 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	21 years STANDARD_DEVIATION 2 • n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 years after transplant

Data are reporting number of participants with disease progression. Disease progression is defined as a 1 point increase in the Expanded Disability Status Scale (EDSS) on consecutive evaluations at least 3 months apart.

Outcome measures

Outcome measures
Measure	Stem Cell Transplantation n=21 Participants Autologous hematopoietic stem cell transplantation Peripheral blood stem cells were mobilised with 2g per square m intravenous (IV) cyclophosphamide (CY) followed by 10 µg per kg subcutaneous filgrastim daily from day 5. After neutrophil recovery, the mobilised cells were collected by apheresis. The recovered cells were unselected and cryopreserved. There was at least three weeks between mobilisation and the conditioning regimen. The conditioning regimen used was 200 mg per kg intravenous CY , given in four equal fractions between day -5 and day -2 with IV mesna, and one 20mg dose of IV alemtuzumab (CAMPATH-1H) given on day -2 with 250 mg intravenous methyl-prednisolone as premedication. Alemtuzumab was changed to rabbit antithymocyte globulin rATG) in the last 4 patients, who received 6 mg per kg rATG over 5 days instead of alemtuzumab . Stem cells wer reinfused 36 h after completion of CY. 5 µg/kg/day filgrastim was given from day 5 until neutrophil recovery.
Disease Progression	4 participants

SECONDARY outcome

Timeframe: three years

Data are reporting the number of participants who survived three years after the transplant Survival of 21 participants was evaluated at three years after the transplant

Outcome measures

Outcome measures
Measure	Stem Cell Transplantation n=21 Participants Autologous hematopoietic stem cell transplantation Peripheral blood stem cells were mobilised with 2g per square m intravenous (IV) cyclophosphamide (CY) followed by 10 µg per kg subcutaneous filgrastim daily from day 5. After neutrophil recovery, the mobilised cells were collected by apheresis. The recovered cells were unselected and cryopreserved. There was at least three weeks between mobilisation and the conditioning regimen. The conditioning regimen used was 200 mg per kg intravenous CY , given in four equal fractions between day -5 and day -2 with IV mesna, and one 20mg dose of IV alemtuzumab (CAMPATH-1H) given on day -2 with 250 mg intravenous methyl-prednisolone as premedication. Alemtuzumab was changed to rabbit antithymocyte globulin rATG) in the last 4 patients, who received 6 mg per kg rATG over 5 days instead of alemtuzumab . Stem cells wer reinfused 36 h after completion of CY. 5 µg/kg/day filgrastim was given from day 5 until neutrophil recovery.
Survival	21 participants

Adverse Events

Stem Cell Transplantation

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Stem Cell Transplantation n=21 participants at risk All participants will undergo stem cell transplantation after receiving conditioning regimen. hematopoietic stem cell transplantation : Autologous hematopoietic stem cell transplantation
Nervous system disorders Transient neurological symptoms	4.8% 1/21 • Number of events 1 • three years
Infections and infestations Blood culture with coagulase-negative staphylococcus	4.8% 1/21 • Number of events 1 • three years
Infections and infestations Dermatomal zoster	9.5% 2/21 • Number of events 2 • three years
Gastrointestinal disorders Diarrhoea due to Clostridium difficile	4.8% 1/21 • Number of events 1 • three years
Blood and lymphatic system disorders Grade IV thrombocytopenia	9.5% 2/21 • Number of events 2 • three years

Additional Information

Dr Richard Burt

Northwestern University

Phone: 312-908-0059

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place