Trial Outcomes & Findings for Combined PEX, Rituximab and Steroids in Acute Idiopathic Pulmonary Fibrosis Exacerbations (NCT NCT01266317)
NCT ID: NCT01266317
Last Updated: 2018-03-14
Results Overview
To assess the feasibility and safety of combined PEX, rituximab, and conventional corticosteroid administrations for the treatment of hospitalized patients with acute IPF exacerbations by monitoring indices of respiratory (PaO2) and cardiovascular function during the treatment interval. Respiratory deterioration was defined by a compilations of respiratory deteriorations (deteriorating gas exchange) and hemodynamic deteriorations (defined as a need for medical intervention).
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
9 participants
Primary outcome timeframe
28 days
Results posted on
2018-03-14
Participant Flow
Participant milestones
| Measure |
Combined PEX, Rituximab and Steroids
Standard Steroid Treatment: One gm of methylprednisolone I.V., on day 0, followed by 40 mg/day I.V. on days 1-4, and days 6-12 (or the P.O. prednisone equivalent). Methylprednisolone 100 mg I.V. will be administered on days 5 and 13. Steroid doses will then be 20 mg methylprednisolone I.V. (or P.O. prednisone equivalent) from days 14-28, and then reduced thereafter at the discretion of the principle investigator.
Plasma exchange (PEX) will consist of 1.5x estimated plasma volume exchanges for 3 successive days (0, 1,2) and then, after a one day interval to enable equilibration of autoantibodies sequestered in tissues, two more daily treatments on days 4 and 5.
Rituximab: One gm I.V. will be administered on day 5 (after completion of the last PEX) and day 13.
Combined Plasma Exchange (PEX), Rituximab, and Corticosteroids: Standard Steroid Treatment, Plasma exchange will consist of 1.5x estimated plasma volume exchanges, Rituximab
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Combined PEX, Rituximab and Steroids
Standard Steroid Treatment: One gm of methylprednisolone I.V., on day 0, followed by 40 mg/day I.V. on days 1-4, and days 6-12 (or the P.O. prednisone equivalent). Methylprednisolone 100 mg I.V. will be administered on days 5 and 13. Steroid doses will then be 20 mg methylprednisolone I.V. (or P.O. prednisone equivalent) from days 14-28, and then reduced thereafter at the discretion of the principle investigator.
Plasma exchange (PEX) will consist of 1.5x estimated plasma volume exchanges for 3 successive days (0, 1,2) and then, after a one day interval to enable equilibration of autoantibodies sequestered in tissues, two more daily treatments on days 4 and 5.
Rituximab: One gm I.V. will be administered on day 5 (after completion of the last PEX) and day 13.
Combined Plasma Exchange (PEX), Rituximab, and Corticosteroids: Standard Steroid Treatment, Plasma exchange will consist of 1.5x estimated plasma volume exchanges, Rituximab
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Combined PEX, Rituximab and Steroids in Acute Idiopathic Pulmonary Fibrosis Exacerbations
Baseline characteristics by cohort
| Measure |
Combined PEX, Rituximab and Steroids
n=9 Participants
Standard Steroid Treatment: One gm of methylprednisolone I.V., on day 0, followed by 40 mg/day I.V. on days 1-4, and days 6-12 (or the P.O. prednisone equivalent). Methylprednisolone 100 mg I.V. will be administered on days 5 and 13. Steroid doses will then be 20 mg methylprednisolone I.V. (or P.O. prednisone equivalent) from days 14-28, and then reduced thereafter at the discretion of the principle investigator.
Plasma exchange (PEX) will consist of 1.5x estimated plasma volume exchanges for 3 successive days (0, 1,2) and then, after a one day interval to enable equilibration of autoantibodies sequestered in tissues, two more daily treatments on days 4 and 5.
Rituximab: One gm I.V. will be administered on day 5 (after completion of the last PEX) and day 13.
Combined Plasma Exchange (PEX), Rituximab, and Corticosteroids: Standard Steroid Treatment, Plasma exchange will consist of 1.5x estimated plasma volume exchanges, Rituximab
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
6 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 28 daysTo assess the feasibility and safety of combined PEX, rituximab, and conventional corticosteroid administrations for the treatment of hospitalized patients with acute IPF exacerbations by monitoring indices of respiratory (PaO2) and cardiovascular function during the treatment interval. Respiratory deterioration was defined by a compilations of respiratory deteriorations (deteriorating gas exchange) and hemodynamic deteriorations (defined as a need for medical intervention).
Outcome measures
| Measure |
Combined PEX, Rituximab and Steroids
n=7 Participants
Standard Steroid Treatment: One gm of methylprednisolone I.V., on day 0, followed by 40 mg/day I.V. on days 1-4, and days 6-12 (or the P.O. prednisone equivalent). Methylprednisolone 100 mg I.V. will be administered on days 5 and 13. Steroid doses will then be 20 mg methylprednisolone I.V. (or P.O. prednisone equivalent) from days 14-28, and then reduced thereafter at the discretion of the principle investigator.
Plasma exchange (PEX) will consist of 1.5x estimated plasma volume exchanges for 3 successive days (0, 1,2) and then, after a one day interval to enable equilibration of autoantibodies sequestered in tissues, two more daily treatments on days 4 and 5.
Rituximab: One gm I.V. will be administered on day 5 (after completion of the last PEX) and day 13.
Combined Plasma Exchange (PEX), Rituximab, and Corticosteroids: Standard Steroid Treatment, Plasma exchange will consist of 1.5x estimated plasma volume exchanges, Rituximab
|
|---|---|
|
Number of Participants With Respiratory and/or Hemodynamic Deteriorations
Respiratory Deterioration
|
1 Participants
|
|
Number of Participants With Respiratory and/or Hemodynamic Deteriorations
Hemodynamic Deterioration
|
3 Participants
|
SECONDARY outcome
Timeframe: 60 daysThe secondary outcome measures a composite outcome defined as survival to 60 days or survival to transplantation at any time post therapy.
Outcome measures
| Measure |
Combined PEX, Rituximab and Steroids
n=7 Participants
Standard Steroid Treatment: One gm of methylprednisolone I.V., on day 0, followed by 40 mg/day I.V. on days 1-4, and days 6-12 (or the P.O. prednisone equivalent). Methylprednisolone 100 mg I.V. will be administered on days 5 and 13. Steroid doses will then be 20 mg methylprednisolone I.V. (or P.O. prednisone equivalent) from days 14-28, and then reduced thereafter at the discretion of the principle investigator.
Plasma exchange (PEX) will consist of 1.5x estimated plasma volume exchanges for 3 successive days (0, 1,2) and then, after a one day interval to enable equilibration of autoantibodies sequestered in tissues, two more daily treatments on days 4 and 5.
Rituximab: One gm I.V. will be administered on day 5 (after completion of the last PEX) and day 13.
Combined Plasma Exchange (PEX), Rituximab, and Corticosteroids: Standard Steroid Treatment, Plasma exchange will consist of 1.5x estimated plasma volume exchanges, Rituximab
|
|---|---|
|
Number of Participants Survived to 60 Days or to Transplantation
|
3 Participants
|
Adverse Events
Combined PEX, Rituximab and Steroids
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Combined PEX, Rituximab and Steroids
n=10 participants at risk
Standard Steroid Treatment: One gm of methylprednisolone I.V., on day 0, followed by 40 mg/day I.V. on days 1-4, and days 6-12 (or the P.O. prednisone equivalent). Methylprednisolone 100 mg I.V. will be administered on days 5 and 13. Steroid doses will then be 20 mg methylprednisolone I.V. (or P.O. prednisone equivalent) from days 14-28, and then reduced thereafter at the discretion of the principle investigator.
Plasma exchange (PEX) will consist of 1.5x estimated plasma volume exchanges for 3 successive days (0, 1,2) and then, after a one day interval to enable equilibration of autoantibodies sequestered in tissues, two more daily treatments on days 4 and 5.
Rituximab: One gm I.V. will be administered on day 5 (after completion of the last PEX) and day 13.
Combined Plasma Exchange (PEX), Rituximab, and Corticosteroids: Standard Steroid Treatment, Plasma exchange will consist of 1.5x estimated plasma volume exchanges, Rituximab
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Hypoxemia
|
10.0%
1/10 • Number of events 1 • 60 days
|
Other adverse events
| Measure |
Combined PEX, Rituximab and Steroids
n=10 participants at risk
Standard Steroid Treatment: One gm of methylprednisolone I.V., on day 0, followed by 40 mg/day I.V. on days 1-4, and days 6-12 (or the P.O. prednisone equivalent). Methylprednisolone 100 mg I.V. will be administered on days 5 and 13. Steroid doses will then be 20 mg methylprednisolone I.V. (or P.O. prednisone equivalent) from days 14-28, and then reduced thereafter at the discretion of the principle investigator.
Plasma exchange (PEX) will consist of 1.5x estimated plasma volume exchanges for 3 successive days (0, 1,2) and then, after a one day interval to enable equilibration of autoantibodies sequestered in tissues, two more daily treatments on days 4 and 5.
Rituximab: One gm I.V. will be administered on day 5 (after completion of the last PEX) and day 13.
Combined Plasma Exchange (PEX), Rituximab, and Corticosteroids: Standard Steroid Treatment, Plasma exchange will consist of 1.5x estimated plasma volume exchanges, Rituximab
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Hypoxemia
|
40.0%
4/10 • Number of events 4 • 60 days
|
|
Vascular disorders
Hypotension
|
10.0%
1/10 • Number of events 1 • 60 days
|
|
Endocrine disorders
Hyperglycemia
|
10.0%
1/10 • Number of events 1 • 60 days
|
|
Skin and subcutaneous tissue disorders
Rash
|
10.0%
1/10 • Number of events 1 • 60 days
|
|
Skin and subcutaneous tissue disorders
Infection
|
10.0%
1/10 • Number of events 1 • 60 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place