Trial Outcomes & Findings for Safety and Tolerability Of Allogeneic Mesenchymal Stromal Cells in Pediatric Inflammatory Bowel Disease (NCT NCT02150551)

Last Updated: 2024-05-06

Results Overview

Safety and tolerability of the administration of human allogeneic bone marrow-derived stromal cells to children and young adults with IBD, measured by the frequency of any SAEs, AEs and/or early treatment discontinuations. Weekly infusions for 8 weeks, post-treatment assessment 45 days after last infusion, three additional follow-up visits over 2 years.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

1 participants

Primary outcome timeframe

45 days after the last infusion

Results posted on

2024-05-06

Participant Flow

STOMP001 was enrolled in the study on 6/7/2018 at CNH outpatient clinic. Only one patient was enrolled in this study.

Participant milestones

Participant milestones
Measure	Mesenchymal Stromal Cells (MSCs) A fixed dose of Mesenchymal Stromal Cells (MSCs) will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator. Allogeneic bone marrow-derived mesenchymal stromal cells: This study is a pilot phase 1 study of patients with moderately to severely active Crohn Disease (CD) and ulcerative colitis (UC) (≥ 18 years, Mayo score: ≥6 or CDAI: ˃ 220; \<18 years, Pediatric Crohn's Disease Activity Index (PCDAI) :\> 30) or Pediatric Ulcerative Colitis Activity Index (PUCAI) : \>34). A fixed dose will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator.
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Tolerability Of Allogeneic Mesenchymal Stromal Cells in Pediatric Inflammatory Bowel Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Mesenchymal Stromal Cells (MSCs) n=1 Participants A fixed dose of Mesenchymal Stromal Cells (MSCs) will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator. Allogeneic bone marrow-derived mesenchymal stromal cells: This study is a pilot phase 1 study of patients with moderately to severely active Crohn Disease (CD) and ulcerative colitis (UC) (≥ 18 years, Mayo score: ≥6 or CDAI: ˃ 220; \<18 years, Pediatric Crohn's Disease Activity Index (PCDAI) :\> 30) or Pediatric Ulcerative Colitis Activity Index (PUCAI) : \>34). A fixed dose will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Customized 1	1 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 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	0 Participants n=5 Participants
Race (NIH/OMB) White	1 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
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 45 days after the last infusion

Population: STOMP001 was enrolled in the study on 6/7/2018 and was withdrawn from the study 09/05/2018 due to refusal to continue the follow-up visits for the study. Due to patient's refusal to continue the study, there were no relative data to be analyzed.

Outcome measures

Outcome measures
Measure	Mesenchymal Stromal Cells (MSCs) n=1 Participants A fixed dose of Mesenchymal Stromal Cells (MSCs) will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator. Allogeneic bone marrow-derived mesenchymal stromal cells: This study is a pilot phase 1 study of patients with moderately to severely active Crohn Disease (CD) and ulcerative colitis (UC) (≥ 18 years, Mayo score: ≥6 or CDAI: ˃ 220; \<18 years, Pediatric Crohn's Disease Activity Index (PCDAI) :\> 30) or Pediatric Ulcerative Colitis Activity Index (PUCAI) : \>34). A fixed dose will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator.
Number of Subjects Who Experience Serious Adverse Events, Adverse Events, and/or Early Treatment Discontinuations.	1 Participants

SECONDARY outcome

Timeframe: 45 days after last infusion

Proportion of subjects that achieve a clinical response by 45 days after last infusion, as defined by a decrease in PCDAI from baseline by greater than or equal to 12.5 points (for CD) or a decrease in PUCAI of greater than or equal to 20 points (for UC).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 45 days after last infusion

Improvement in laboratory parameters (i.e. C-reactive protein, fecal calprotectin, anti-HLA antibodies, and viral specific T-cell activity)

Outcome measures

Outcome data not reported

Adverse Events

Mesenchymal Stromal Cells (MSCs)

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Mesenchymal Stromal Cells (MSCs) n=1 participants at risk A fixed dose of Mesenchymal Stromal Cells (MSCs) will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator. Allogeneic bone marrow-derived mesenchymal stromal cells: This study is a pilot phase 1 study of patients with moderately to severely active Crohn Disease (CD) and ulcerative colitis (UC) (≥ 18 years, Mayo score: ≥6 or CDAI: ˃ 220; \<18 years, Pediatric Crohn's Disease Activity Index (PCDAI) :\> 30) or Pediatric Ulcerative Colitis Activity Index (PUCAI) : \>34). A fixed dose will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator.
Gastrointestinal disorders Worsening Crohn's disease	100.0% 1/1 • Number of events 1 • 3 months Participant was withdrawn from the study after 3 months due to refusal to continue all study related follow-up visits.

Other adverse events

Other adverse events
Measure	Mesenchymal Stromal Cells (MSCs) n=1 participants at risk A fixed dose of Mesenchymal Stromal Cells (MSCs) will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator. Allogeneic bone marrow-derived mesenchymal stromal cells: This study is a pilot phase 1 study of patients with moderately to severely active Crohn Disease (CD) and ulcerative colitis (UC) (≥ 18 years, Mayo score: ≥6 or CDAI: ˃ 220; \<18 years, Pediatric Crohn's Disease Activity Index (PCDAI) :\> 30) or Pediatric Ulcerative Colitis Activity Index (PUCAI) : \>34). A fixed dose will be studied: 1 x 106 cells/kg administered intravenously (IV) weekly for 4 consecutive weeks, with the option of an additional 4 weeks of treatment, at the discretion of the principal investigator.
Cardiac disorders Fainting	100.0% 1/1 • Number of events 1 • 3 months Participant was withdrawn from the study after 3 months due to refusal to continue all study related follow-up visits.
Gastrointestinal disorders Superior mesenteric artery syndrome (Partial Obstruction of Duodenum)	100.0% 1/1 • Number of events 1 • 3 months Participant was withdrawn from the study after 3 months due to refusal to continue all study related follow-up visits.

Additional Information

Catherine Bollard, MD

Children's National Hospital

Phone: 202-476-4776

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place