Trial Outcomes & Findings for Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy (NCT NCT00278629)

NCT ID: NCT00278629

Last Updated: 2020-07-23

Results Overview

Survival of participants who survived the treatment and up to 5 years post treatment.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 80 participants
• Primary outcome timeframe: up to 5 years
• Results posted on: 2020-07-23

Participant Flow

Participant milestones

Measure	Hematopoietic Stem Cell Transplantation Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Overall Study STARTED	80
Overall Study COMPLETED	66
Overall Study NOT COMPLETED	14

Reasons for withdrawal

Measure	Hematopoietic Stem Cell Transplantation Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Overall Study Non-CIDP Immune-Mediated Peripheral Neur	14

Baseline Characteristics

The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.

Baseline characteristics by cohort

Measure	Hematopoietic Stem Cell Transplantation for CIDP n=66 Participants Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Age, Categorical <=18 years	0 Participants n=60 Participants • The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Age, Categorical Between 18 and 65 years	60 Participants n=60 Participants • The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Age, Categorical >=65 years	0 Participants n=60 Participants • The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Age, Continuous	43 years n=60 Participants • The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Sex: Female, Male Female	37 Participants n=60 Participants • The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Sex: Female, Male Male	23 Participants n=60 Participants • The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.
Region of Enrollment United States	60 participants n=60 Participants • The number analyzed differs from the overall because patients did not follow up and were subsequently excluded from the analysis.

PRIMARY outcome

Timeframe: up to 5 years

Population: The reason that the number analyzed is different from the overall number of participants anaylyzed is because 2 patients died from non treatment related conditions that were present before the transplant. Both deaths occurred more than 100 days after the hospital discharge from treatment.

Survival of participants who survived the treatment and up to 5 years post treatment.

Outcome measures

Measure	Hematopoietic Stem Cell Transplantation for CIDP n=66 Participants Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV
Number of Participants With Survival Treatment Survival	66 Participants
Number of Participants With Survival Post Treament Survival	64 Participants

SECONDARY outcome

Timeframe: 6 months, 1, 2, 3, 4 and 5 years post transplant

Population: The number analyzed in one or more rows differs from the over all number because not all patients returned for follow up.

Post transplant immune medication-free remission with stable or improving neurological exam

Outcome measures

Measure	Hematopoietic Stem Cell Transplantation for CIDP n=60 Participants Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV
Disease Improvement - Medication Free Remission 6 months post transplant	48 participants
Disease Improvement - Medication Free Remission 1 year post transplant	48 participants
Disease Improvement - Medication Free Remission 2 years after transplant	47 participants
Disease Improvement - Medication Free Remission 3 years post transplant	45 participants
Disease Improvement - Medication Free Remission 4 years post transplant	39 participants
Disease Improvement - Medication Free Remission 5 years post transplant	35 participants

SECONDARY outcome

Timeframe: pre transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant

Population: The number analyzed in one or more rows differs from the overall number analyzed because not all patients returned for followed up testing.

Disease Improvement - Requires No Assistance to Ambulate

Outcome measures

Measure	Hematopoietic Stem Cell Transplantation for CIDP n=60 Participants Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV
Disease Improvement - Ambulatory Assistance 6 months post transplant	41 Participants
Disease Improvement - Ambulatory Assistance 1 year post transplant	49 Participants
Disease Improvement - Ambulatory Assistance pre transplant	19 Participants
Disease Improvement - Ambulatory Assistance 2 years post transplant	48 Participants
Disease Improvement - Ambulatory Assistance 3 years post transplant	48 Participants
Disease Improvement - Ambulatory Assistance 4 years post transplant	43 Participants
Disease Improvement - Ambulatory Assistance 5 years post transplant	35 Participants

SECONDARY outcome

Timeframe: Pre Transplant, 6 months, 1, 2, 3, 4 and 5 years post transplant

Population: The reason the number analyzed in one or more rows differs from the overall number analyzed is because not all patients returned for followed up testing.

Change in the Medical Research Council (MRC) Scale: range is 0 (quadriplegic) - 60 (normal).

Outcome measures

Measure	Hematopoietic Stem Cell Transplantation for CIDP n=60 Participants Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV
Change in Disability and Strength Functional Scales Pre Transplant	51.8 score on a scale Standard Deviation 6.3
Change in Disability and Strength Functional Scales 6 Months Post Transplant	54.8 score on a scale Standard Deviation 5.6
Change in Disability and Strength Functional Scales 1 year post transplant	57 score on a scale Standard Deviation 4.18
Change in Disability and Strength Functional Scales 2 years post transplant	57 score on a scale Standard Deviation 4.3
Change in Disability and Strength Functional Scales 3 years post transplant	57 score on a scale Standard Deviation 3.9
Change in Disability and Strength Functional Scales 4 years post transplant	57 score on a scale Standard Deviation 3.9
Change in Disability and Strength Functional Scales 5 years post transplant	57 score on a scale Standard Deviation 4.3

Adverse Events

Hematopoietic Stem Cell Transplantation for CIDP

Serious events: 2 serious events

Other events: 29 other events

Deaths: 2 deaths

Serious adverse events

Measure	Hematopoietic Stem Cell Transplantation for CIDP n=66 participants at risk Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Gastrointestinal disorders Total Parenteral Nutrition - due to vomiting	1.5% 1/66 • Up to 5 years
Metabolism and nutrition disorders Hypokalemia	1.5% 1/66 • Up to 5 years
Respiratory, thoracic and mediastinal disorders Transient Dyspnea	1.5% 1/66 • Number of events 1 • Up to 5 years
Cardiac disorders Non Transplant Related Death	1.5% 1/66 • Number of events 1 • Up to 5 years
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Non Transplant Related Death	1.5% 1/66 • Number of events 1 • Up to 5 years

Other adverse events

Measure	Hematopoietic Stem Cell Transplantation for CIDP n=66 participants at risk Autologous hematopoietic stem cell transplantation will be performed after conditioning regimen of cyclophosphamide 200 mg/kg/intravenously(IV), rATG(thymoglobulin) 5.5 mg/kg/IV and rituximab 1000mg/IV.
Metabolism and nutrition disorders Hypophosphatemia	43.9% 29/66 • Number of events 29 • Up to 5 years
General disorders Febrile Neutropenia	39.4% 26/66 • Number of events 26 • Up to 5 years
Metabolism and nutrition disorders Hypokalemia	28.8% 19/66 • Number of events 19 • Up to 5 years
Respiratory, thoracic and mediastinal disorders Dyspnea	12.1% 8/66 • Number of events 8 • Up to 5 years
Infections and infestations Infections	12.1% 8/66 • Number of events 8 • Up to 5 years
Infections and infestations Clostridium Difficile Diarrhea	6.1% 4/66 • Number of events 4 • Up to 5 years
Metabolism and nutrition disorders Hypocalcemia	7.6% 5/66 • Number of events 5 • Up to 5 years
Metabolism and nutrition disorders Hyponatremia	7.6% 5/66 • Number of events 5 • Up to 5 years

Additional Information

Kathleen Quigley

Northwestern University

Phone: 312-695-8192

Email: k-quigley@northwestern.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place