Trial Outcomes & Findings for Idelalisib Post Allogeneic Hematopoietic Stem Cell Transplant (HSCT) in B Cell Derived Malignancies (NCT NCT03151057)
NCT ID: NCT03151057
Last Updated: 2024-03-12
Results Overview
The evaluation of the safety of Idelalisib as post-transplantation maintenance in patients with B cell hematologic malignancies
Recruitment status
TERMINATED
Study phase
PHASE1
Target enrollment
16 participants
Primary outcome timeframe
Day 90 - Day 270 post transplant
Results posted on
2024-03-12
Participant Flow
This was a double blinded randomized study with 2 participants randomized to idelalisib arm for every 1 randomized to placebo.
Participant milestones
| Measure |
Idelalisib 100mg
Idelalisib is an orally-administered, selective inhibitor of Phosphoinositide 3 kinase (PI3K)-delta which has been shown to be extremely effective in inducing partial to complete responses in many B-cell derived malignancies.
intervention: 100mg Idelalisib twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Idelalisib 100 MG: 100mg BID beginning on day 90 (+/- 10days) and continuing until day 270 post transplant.
|
Placebo Oral Tablet
Placebo to be taken twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Placebo Oral Tablet: placebo
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
7
|
|
Overall Study
COMPLETED
|
9
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Idelalisib Post Allogeneic Hematopoietic Stem Cell Transplant (HSCT) in B Cell Derived Malignancies
Baseline characteristics by cohort
| Measure |
Idelalisib 100mg
n=9 Participants
Idelalisib is an orally-administered, selective inhibitor of Phosphoinositide 3 kinase (PI3K)-delta which has been shown to be extremely effective in inducing partial to complete responses in many B-cell derived malignancies.
intervention: 100mg Idelalisib twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Idelalisib 100 MG: 100mg BID beginning on day 90 (+/- 10days) and continuing until day 270 post transplant.
|
Placebo Oral Tablet
n=7 Participants
Placebo to be taken twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Placebo Oral Tablet: placebo
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Age, Continuous
|
57.33 years
n=5 Participants
|
58.00 years
n=7 Participants
|
57.62 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 90 - Day 270 post transplantThe evaluation of the safety of Idelalisib as post-transplantation maintenance in patients with B cell hematologic malignancies
Outcome measures
| Measure |
Idelalisib 100mg
n=9 Participants
Idelalisib is an orally-administered, selective inhibitor of Phosphoinositide 3 kinase (PI3K)-delta which has been shown to be extremely effective in inducing partial to complete responses in many B-cell derived malignancies.
intervention: 100mg Idelalisib twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Idelalisib 100 MG: 100mg BID beginning on day 90 (+/- 10days) and continuing until day 270 post transplant.
|
Placebo Oral Tablet
n=7 Participants
Placebo to be taken twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Placebo Oral Tablet: placebo
|
|---|---|---|
|
Treatment-limiting Toxicities Will be Defined as Idelalisib Interruption for >14 Days, or Other >3 Adverse Events as Defined by CTCAE IV Not Captured in the Protocol for Dose De-escalation.
|
9 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Beginning Day 90 post transplant until Day 360Impact of Idelalisib on aGVHD, relapse, and non-relapse mortality
Outcome measures
| Measure |
Idelalisib 100mg
n=9 Participants
Idelalisib is an orally-administered, selective inhibitor of Phosphoinositide 3 kinase (PI3K)-delta which has been shown to be extremely effective in inducing partial to complete responses in many B-cell derived malignancies.
intervention: 100mg Idelalisib twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Idelalisib 100 MG: 100mg BID beginning on day 90 (+/- 10days) and continuing until day 270 post transplant.
|
Placebo Oral Tablet
n=7 Participants
Placebo to be taken twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Placebo Oral Tablet: placebo
|
|---|---|---|
|
Event Free Survival at One Year.
|
9 Participants
|
7 Participants
|
SECONDARY outcome
Timeframe: Beginning Day 90 post transplant until Day 270Population: Exploratory gene expression analysis of immune biomarkers in bone marrow aspirates and whole or targeted exome sequencing of lymphoma cells was not was not performed on any samples for this study. This study was terminated early due to safety concerns. Samples collected were not analyzed due to an initial lack of lab staffing during COVID then were destroyed after this sponsor/investigator left Johns Hopkins.
Search for Biomarkers which could better identify which patients would respond to treatment with Idelalisib in the post-transplant setting.
Outcome measures
Outcome data not reported
Adverse Events
Idelalisib 100mg
Serious events: 7 serious events
Other events: 9 other events
Deaths: 2 deaths
Placebo Oral Tablet
Serious events: 4 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Idelalisib 100mg
n=9 participants at risk
Idelalisib is an orally-administered, selective inhibitor of Phosphoinositide 3 kinase (PI3K)-delta which has been shown to be extremely effective in inducing partial to complete responses in many B-cell derived malignancies.
intervention: 100mg Idelalisib twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Idelalisib 100 MG: 100mg BID beginning on day 90 (+/- 10days) and continuing until day 270 post transplant.
|
Placebo Oral Tablet
n=7 participants at risk
Placebo to be taken twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Placebo Oral Tablet: placebo
|
|---|---|---|
|
Infections and infestations
Lung Infection
|
22.2%
2/9 • Number of events 2 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
Blood and lymphatic system disorders
Neutrophil Count Decreased
|
44.4%
4/9 • Number of events 4 • Up to 17 months
|
14.3%
1/7 • Number of events 1 • Up to 17 months
|
|
Gastrointestinal disorders
diarrhea
|
22.2%
2/9 • Number of events 2 • Up to 17 months
|
14.3%
1/7 • Number of events 1 • Up to 17 months
|
|
Investigations
increased Alanine transaminase (ALT)
|
22.2%
2/9 • Number of events 3 • Up to 17 months
|
28.6%
2/7 • Number of events 2 • Up to 17 months
|
|
Investigations
Increased Aspartate Transferase (AST)
|
22.2%
2/9 • Number of events 2 • Up to 17 months
|
28.6%
2/7 • Number of events 2 • Up to 17 months
|
|
Investigations
Increased bilirubin
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
Infections and infestations
sepsis
|
0.00%
0/9 • Up to 17 months
|
14.3%
1/7 • Number of events 1 • Up to 17 months
|
|
Metabolism and nutrition disorders
dehydration
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
General disorders
fatigue
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
Blood and lymphatic system disorders
febrile neutropenia
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
Renal and urinary disorders
Hemorrhagic cystitis
|
0.00%
0/9 • Up to 17 months
|
14.3%
1/7 • Number of events 1 • Up to 17 months
|
|
Gastrointestinal disorders
Nausea and vomiting
|
22.2%
2/9 • Number of events 2 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
Nervous system disorders
syncope
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
Blood and lymphatic system disorders
Decreased platelet count
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
14.3%
1/7 • Number of events 2 • Up to 17 months
|
Other adverse events
| Measure |
Idelalisib 100mg
n=9 participants at risk
Idelalisib is an orally-administered, selective inhibitor of Phosphoinositide 3 kinase (PI3K)-delta which has been shown to be extremely effective in inducing partial to complete responses in many B-cell derived malignancies.
intervention: 100mg Idelalisib twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Idelalisib 100 MG: 100mg BID beginning on day 90 (+/- 10days) and continuing until day 270 post transplant.
|
Placebo Oral Tablet
n=7 participants at risk
Placebo to be taken twice daily beginning +90(+/- 10) days after allo HSCT and continued through Day 270 post transplant
Placebo Oral Tablet: placebo
|
|---|---|---|
|
General disorders
idelalisib or placebo held for >14 days
|
55.6%
5/9 • Number of events 5 • Up to 17 months
|
42.9%
3/7 • Number of events 3 • Up to 17 months
|
|
Immune system disorders
Graft versus host disease (GVHD)
|
44.4%
4/9 • Number of events 4 • Up to 17 months
|
28.6%
2/7 • Number of events 2 • Up to 17 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
3/9 • Number of events 3 • Up to 17 months
|
14.3%
1/7 • Number of events 1 • Up to 17 months
|
|
Musculoskeletal and connective tissue disorders
back pain
|
0.00%
0/9 • Up to 17 months
|
14.3%
1/7 • Number of events 1 • Up to 17 months
|
|
Nervous system disorders
headache
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
Gastrointestinal disorders
constipation
|
11.1%
1/9 • Number of events 1 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
|
General disorders
fever
|
11.1%
1/9 • Number of events 2 • Up to 17 months
|
0.00%
0/7 • Up to 17 months
|
Additional Information
Dr. Douglas Gladstone
Northwell Health Cancer Institute
Phone: (516) 734-8900
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place