Trial Outcomes & Findings for Cardiac Safe Transplants for Systemic Sclerosis (NCT NCT03593902)

NCT ID: NCT03593902

Last Updated: 2020-08-12

Results Overview

Defined by at least a 25% improvement (decline) in skin score by modified Rodnan skin score (mRSS) if skin score is greater than 14 on enrollment. If skin score is less than 14 on enrollment, improvement is defined by at least a 5% improvement on mRSS. The modified Rodnan skin score (MRSS) is a measure for skin disease in scleroderma and is calculated by summation of skin thickness in 17 different body sites. The scale ranges from at total score of normal skin thickness (0) to severe thickness (51).

• Recruitment status: TERMINATED
• Study phase: PHASE2/PHASE3
• Target enrollment: 9 participants
• Primary outcome timeframe: Pre Treatment and 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 rituximab, fludarabine, cyclophosphamide, Mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) and intravenous immunoglobulin (IVIg) will be administered post-transplant. Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer Fludarabine: A chemotherapy medication commonly used in the treatment of leukemia and lymphoma Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder rATG: A rabbit polyclonal antibody to lymphocytes Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cardiac Safe Transplants for Systemic Sclerosis

Baseline characteristics by cohort

Measure	Hematopoietic Stem Cell Transplantation n=9 Participants Hematopoietic Stem Cell Therapy will be performed: Autologous stem cells will be infused after conditioning with rituximab, fludarabine, cyclophosphamide, Mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) and intravenous immunoglobulin (IVIg) will be administered post-transplant. Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer Fludarabine: A chemotherapy medication commonly used in the treatment of leukemia and lymphoma Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder rATG: A rabbit polyclonal antibody to lymphocytes Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Modified Rodnan skin score	25 units on a scale n=5 Participants

PRIMARY outcome

Timeframe: Pre Treatment and Post Treatment

Population: The number analyzed in one or more rows differs from the overall number analyzed because some patients did not follow up after the treatment.

Defined by at least a 25% improvement (decline) in skin score by modified Rodnan skin score (mRSS) if skin score is greater than 14 on enrollment. If skin score is less than 14 on enrollment, improvement is defined by at least a 5% improvement on mRSS. The modified Rodnan skin score (MRSS) is a measure for skin disease in scleroderma and is calculated by summation of skin thickness in 17 different body sites. The scale ranges from at total score of normal skin thickness (0) to severe thickness (51).

Outcome measures

Measure	Hematopoietic Stem Cell Transplantation n=9 Participants Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with rituximab, fludarabine, cyclophosphamide, Mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) and intravenous immunoglobulin (IVIg) will be administered post-transplant. Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer Fludarabine: A chemotherapy medication commonly used in the treatment of leukemia and lymphoma Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder rATG: A rabbit polyclonal antibody to lymphocytes Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and
Change in Skin Score by mRSS Pre Treatment	25 units on a scale Interval 4.0 to 48.0
Change in Skin Score by mRSS Post Treatment	16 units on a scale Interval 2.0 to 30.0

SECONDARY outcome

Timeframe: During Treatment and Post Treatment up to 1 year

Survival of Hematopoietic Stem Cell Transplant during treatment and post treatment up to 1 year.

Outcome measures

Measure

Hematopoietic Stem Cell Transplantation n=9 Participants Hematopoietic Stem Cell Therapy will be performed as follows: Autologous stem cells will be infused after conditioning with rituximab, fludarabine, cyclophosphamide, Mesna, rATG (rabbit), and methylprednisolone. Granulocyte-colony stimulating factor (G-CSF) and intravenous immunoglobulin (IVIg) will be administered post-transplant. Rituximab: Monoclonal antibody therapy used to treat certain autoimmune diseases and types of cancer Fludarabine: A chemotherapy medication commonly used in the treatment of leukemia and lymphoma Cyclophosphamide: A medication used as chemotherapy and to suppress the immune system Mesna: A medication used in those taking cyclophosphamide or ifosfamide to decrease the risk of bleeding from the bladder rATG: A rabbit polyclonal antibody to lymphocytes Methylprednisolone: A corticosteroid medication used to suppress the immune system and decrease inflammation G-CSF: A glycoprotein that stimulates the bone marrow to produce granulocytes and

Survival of Treatment

9 Participants

Adverse Events

Hematopoietic Stem Cell Transplantation

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Kathleen Quigley

Northwestern University

Phone: 312-695-8192

Email: kathleen.quigley@nm.org

Results disclosure agreements

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