Trial Outcomes & Findings for Stem Cell Therapy for Patients With Multiple Sclerosis Failing Alternate Approved Therapy- A Randomized Study (NCT NCT00273364)
NCT ID: NCT00273364
Last Updated: 2020-08-12
Results Overview
The EDSS scale ranges from 0 to 10 in 0.5 increments that represent higher levels of disability. Improvement in EDSS is defined by both a 0.5 or 1.0 points sustained for more than 6 months.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
110 participants
Primary outcome timeframe
Pre Treatment, 6 and 12 months Post Treatment
Results posted on
2020-08-12
Participant Flow
Participant milestones
| Measure |
Hematopoietic Stem Cell Transplantation
Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG
Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen
|
Standard Therapy for MS
Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)
Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)
|
|---|---|---|
|
Overall Study
STARTED
|
55
|
55
|
|
Overall Study
COMPLETED
|
55
|
55
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
The number analyzed in rows differs from the overall enrolled because EDSS were not analyzed on patients who did not receive intervention or who were lost to follow up.
Baseline characteristics by cohort
| Measure |
Hematopoietic Stem Cell Transplantation
n=55 Participants
Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG
Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen
|
Standard Therapy for MS
n=55 Participants
Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)
Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)
|
Total
n=110 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
35.6 years
n=55 Participants
|
35.6 years
n=55 Participants
|
35.6 years
n=110 Participants
|
|
Sex: Female, Male
Female
|
34 Participants
n=55 Participants
|
39 Participants
n=55 Participants
|
73 Participants
n=110 Participants
|
|
Sex: Female, Male
Male
|
21 Participants
n=55 Participants
|
16 Participants
n=55 Participants
|
37 Participants
n=110 Participants
|
|
Expanded Disability Status Scale (EDSS)
|
3.4 units on a scale
STANDARD_DEVIATION 1.2 • n=52 Participants • The number analyzed in rows differs from the overall enrolled because EDSS were not analyzed on patients who did not receive intervention or who were lost to follow up.
|
3.3 units on a scale
STANDARD_DEVIATION 1.0 • n=54 Participants • The number analyzed in rows differs from the overall enrolled because EDSS were not analyzed on patients who did not receive intervention or who were lost to follow up.
|
3.35 units on a scale
STANDARD_DEVIATION .05 • n=106 Participants • The number analyzed in rows differs from the overall enrolled because EDSS were not analyzed on patients who did not receive intervention or who were lost to follow up.
|
PRIMARY outcome
Timeframe: Pre Treatment, 6 and 12 months Post TreatmentPopulation: The number analyzed in one or more rows differs from the overall number analyzed because not all patients had their EDSS evaluated and some patients were lost to followed up.
The EDSS scale ranges from 0 to 10 in 0.5 increments that represent higher levels of disability. Improvement in EDSS is defined by both a 0.5 or 1.0 points sustained for more than 6 months.
Outcome measures
| Measure |
Hematopoietic Stem Cell Transplantation
n=52 Participants
Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG
Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen
|
Standard Therapy for MS
n=54 Participants
Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)
Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)
|
|---|---|---|
|
Expanded Disability Status Scale (EDSS) Improvement
Pre Treatment
|
3.4 units on a scale
Standard Deviation 1.2
|
3.3 units on a scale
Standard Deviation 1.0
|
|
Expanded Disability Status Scale (EDSS) Improvement
6 Months Post Treatment
|
2.5 units on a scale
Standard Deviation 1.4
|
3.7 units on a scale
Standard Deviation 1.5
|
|
Expanded Disability Status Scale (EDSS) Improvement
12 Months Post Treatment
|
2.4 units on a scale
Standard Deviation 1.4
|
4 units on a scale
Standard Deviation 1.7
|
Adverse Events
Hematopoietic Stem Cell Transplantation
Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths
Standard Therapy for MS
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Hematopoietic Stem Cell Transplantation
n=52 participants at risk
Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with Cyclophosphamide and rATG
Hematopoietic Stem Cell Therapy: After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen
|
Standard Therapy for MS
n=55 participants at risk
Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), Gilenya (fingolimod) or Dimethyl fumarate (Tecfidera or BG-12)
Standard treatment with a conventional drug: Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)
|
|---|---|---|
|
General disorders
Febrile Neutropenia (culture negative)
|
25.0%
13/52 • Number of events 13 • Adverse events were collected during transplant and at 1 year post Treatment
|
0.00%
0/55 • Adverse events were collected during transplant and at 1 year post Treatment
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
32.7%
17/52 • Number of events 17 • Adverse events were collected during transplant and at 1 year post Treatment
|
0.00%
0/55 • Adverse events were collected during transplant and at 1 year post Treatment
|
|
Metabolism and nutrition disorders
Hypokalemia
|
25.0%
13/52 • Number of events 13 • Adverse events were collected during transplant and at 1 year post Treatment
|
0.00%
0/55 • Adverse events were collected during transplant and at 1 year post Treatment
|
|
Metabolism and nutrition disorders
Hypergylcemia
|
9.6%
5/52 • Number of events 5 • Adverse events were collected during transplant and at 1 year post Treatment
|
0.00%
0/55 • Adverse events were collected during transplant and at 1 year post Treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place