Ignite Creation Date:
2025-12-24 @ 5:47 PM
Ignite Modification Date:
2025-12-29 @ 11:45 PM
Study NCT ID:
NCT03058068
Status:
WITHDRAWN
Last Update Posted:
2020-05-13
First Post:
2017-02-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Human MesenchymAl Stem Cells Infusion in Patients With Cystic Fibrosis
Sponsor:
Joshua M Hare
Organization:
Study Overview
Official Title:
A Phase I, Randomized and Placebo-controlled Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human MesenchymAl Stem Cells Infusion in Patients With Cystic Fibrosis - HAPI
Status:
WITHDRAWN
Status Verified Date:
2020-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
PI left the institution
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HAPI
Brief Summary:
A Safety Run-In will be followed by a Double-Blinded Randomized Phase. All subjects shall meet the inclusion/exclusion criteria, and will be evaluated prior to the scheduled infusion to establish baseline.
There will be 3 subjects in the safety run-in phase and 15 subjects in the double-blinded phase.
There will be 3 subjects in the safety run-in phase and 15 subjects in the double-blinded phase.
Detailed Description:
Primary objective is to demonstrate the safety of Mesenchymal Stem Cells (MSCs) intravenously administered to subjects with cystic fibrosis.
Secondary Objective is to explore if MSCs can improve the symptoms of cystic fibrosis, including lung function, the rate of pulmonary exacerbation, systemic and local inflammation and symptom-related quality of life.
The Safety Run-In will be performed to evaluate the safety of MSC infusion into subjects with cystic fibrosis. 3 subjects will participate and they will receive a single administration of allogeneic MSCs given through intravenous infusion.
In the randomized phase the subjects will be randomized at a ratio of 1:1:1 into 3 cohorts to receive infusions. There will be a total of 15 subjects in 3 cohorts.
The total duration for each subject after infusion is 12 months, plus up to an additional 2 months for the Screening and Baseline Visits. Approximately 9 visits in total.
Secondary Objective is to explore if MSCs can improve the symptoms of cystic fibrosis, including lung function, the rate of pulmonary exacerbation, systemic and local inflammation and symptom-related quality of life.
The Safety Run-In will be performed to evaluate the safety of MSC infusion into subjects with cystic fibrosis. 3 subjects will participate and they will receive a single administration of allogeneic MSCs given through intravenous infusion.
In the randomized phase the subjects will be randomized at a ratio of 1:1:1 into 3 cohorts to receive infusions. There will be a total of 15 subjects in 3 cohorts.
The total duration for each subject after infusion is 12 months, plus up to an additional 2 months for the Screening and Baseline Visits. Approximately 9 visits in total.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: