Trial Outcomes & Findings for Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy (NCT NCT00258180)

NCT ID: NCT00258180

Last Updated: 2019-04-16

Results Overview

Number of participants off therapy 1 year after study completion without relapse.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 3 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2019-04-16

Participant Flow

1 subject was not assigned intervention due to PI decision.

Participant milestones

Measure	Severe Autoimmune Enteropathy Young patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover.
Overall Study STARTED	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy

Baseline characteristics by cohort

Measure	Severe Autoimmune Enteropathy n=2 Participants Young patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover.
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	2 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	2 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	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Number of participants off therapy 1 year after study completion without relapse.

Outcome measures

Measure	Severe Autoimmune Enteropathy n=2 Participants Young patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover.
Number of Participants With Treatment-free Remission at 1 Year After Study Completion	2 Participants

PRIMARY outcome

Timeframe: 1 month

Outcome measures

Measure	Severe Autoimmune Enteropathy n=2 Participants Young patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover.
Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month	0 Participants

Adverse Events

Severe Autoimmune Enteropathy

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

Maria Oliva-Hemker MD

Johns Hopkins School of Medicine

Phone: 410-955-8769

Email: moliva@jhmi.edu

Results disclosure agreements

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