Trial Outcomes & Findings for Ibrutinib in Preventing Acute Leukemia in Patients After Reduced-Intensity Conditioning and Stem Cell Transplant (NCT NCT03267186)

Last Updated: 2023-08-01

Results Overview

Relapsed leukemia is defined as \> 5% leukemic blasts detected in bone marrow or peripheral blood. Participants were also be considered to have relapsed leukemia if they receive any active treatment for progressive leukemia after allogeneic HCT, even if they have \< 5% leukemic blasts. Withdrawal of immunosuppression alone is not considered an active treatment for progressive disease.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

Up to 18 months

Results posted on

2023-08-01

Participant Flow

Participant milestones

Participant milestones
Measure	Prevention (Ibrutinib) Beginning 60-90 days after allogeneic hematopoietic cell transplantation (HCT), patients receive ibrutinib orally (PO) once daily (QD) for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ibrutinib in Preventing Acute Leukemia in Patients After Reduced-Intensity Conditioning and Stem Cell Transplant

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Prevention (Ibrutinib) n=8 Participants Beginning 60-90 days after allogeneic HCT, patients receive ibrutinib PO QD for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Age, Customized 50-59	2 Participants n=5 Participants
Age, Customized 60-69	6 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	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	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Region of Enrollment United States	8 Participants n=5 Participants
Diagnosis Acute leukemia	4 Participants n=5 Participants
Diagnosis Acute myeloid leukemia	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 18 months

Outcome measures

Outcome measures
Measure	Prevention (Ibrutinib) n=8 Participants Beginning 60-90 days after allogeneic HCT, patients receive ibrutinib PO QD for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Number of Participants With Relapsed Leukemia Relapsed	3 Participants
Number of Participants With Relapsed Leukemia Not relapsed	5 Participants

SECONDARY outcome

Timeframe: At 180 days

Acute GVHD grades II-IV and III-IV was evaluated according to the following criteria. Stage of Acute GvHD was assessed as follows. * Stage 1: Skin: rash \< 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea \>500 mL/day or upper-gut symptoms with positive histology * Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea \>1000 mL/day. * Stage 3: Skin: rash \> 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea \> 1500 mL/day. * Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin \> 15 mg/dL. Gut: diarrhea \> 2500 mL/day or severe abdominal pain with or without ileus Grade of Acute GvHD was determined as follows. * Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage * Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut * Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut * Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage

Outcome measures

Outcome measures
Measure	Prevention (Ibrutinib) n=8 Participants Beginning 60-90 days after allogeneic HCT, patients receive ibrutinib PO QD for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Number of Participants With Acute Graft-versus-host Disease (GVHD) (Any Grade)	4 Participants

SECONDARY outcome

Timeframe: At 18 months

Assessment of chronic GvHD was performed using the 2015 NIH consensus criteria.

Outcome measures

Outcome measures
Measure	Prevention (Ibrutinib) n=8 Participants Beginning 60-90 days after allogeneic HCT, patients receive ibrutinib PO QD for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Number of Participants With Chronic GVHD	3 Participants

SECONDARY outcome

Timeframe: day +90, day +180, 1 year, 1.5 years

Population: Participants who had minimal residual disease testing at the respective time point.

Detectable minimal residual disease was assessed using high-throughput sequencing (ClonoSEQ) and high-sensitivity flow cytometry. Minimal residual disease was considered present if \> 1 x 10\^-6 leukemic clones are detected by ClonoSEQ, or if any aberrant blasts matching the original leukemic immunophenotype are detected by high-sensitivity flow cytometry.

Outcome measures

Outcome measures
Measure	Prevention (Ibrutinib) n=8 Participants Beginning 60-90 days after allogeneic HCT, patients receive ibrutinib PO QD for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Number of Participants Negative for Detectable Minimal Residual Disease day +90	8 Participants
Number of Participants Negative for Detectable Minimal Residual Disease day +180	7 Participants
Number of Participants Negative for Detectable Minimal Residual Disease 1 year	5 Participants
Number of Participants Negative for Detectable Minimal Residual Disease 1.5 years	5 Participants

Adverse Events

Prevention (Ibrutinib)

Serious events: 3 serious events

Other events: 7 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Prevention (Ibrutinib) n=8 participants at risk Beginning 60-90 days after allogeneic HCT, patients receive ibrutinib PO QD for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Blood and lymphatic system disorders Pancytopenia	12.5% 1/8 • up to 18 months
Gastrointestinal disorders Diarrhea	25.0% 2/8 • up to 18 months
General disorders Fever	12.5% 1/8 • up to 18 months

Other adverse events

Other adverse events
Measure	Prevention (Ibrutinib) n=8 participants at risk Beginning 60-90 days after allogeneic HCT, patients receive ibrutinib PO QD for up to 18 months post-transplant in the absence of disease progression or unacceptable toxicity.
Metabolism and nutrition disorders Anorexia	12.5% 1/8 • up to 18 months
Metabolism and nutrition disorders Hypokalemia	12.5% 1/8 • up to 18 months
Metabolism and nutrition disorders Hyperkalemia	12.5% 1/8 • up to 18 months
Metabolism and nutrition disorders Hyperglycemia	12.5% 1/8 • up to 18 months
Nervous system disorders Peripheral Neuropathy	12.5% 1/8 • up to 18 months
Musculoskeletal and connective tissue disorders Leg Pain	12.5% 1/8 • up to 18 months
Musculoskeletal and connective tissue disorders Bilateral Hand Pain	12.5% 1/8 • up to 18 months
Musculoskeletal and connective tissue disorders Arthritis	12.5% 1/8 • up to 18 months
Respiratory, thoracic and mediastinal disorders Shortness of breath (dyspnea)	25.0% 2/8 • up to 18 months
Gastrointestinal disorders Nausea	12.5% 1/8 • up to 18 months
Gastrointestinal disorders Diarrhea	25.0% 2/8 • up to 18 months
Gastrointestinal disorders Abdominal Pain	12.5% 1/8 • up to 18 months
Gastrointestinal disorders Oral Ulcer	12.5% 1/8 • up to 18 months
Skin and subcutaneous tissue disorders Facial hyperpigmentation	12.5% 1/8 • up to 18 months
Skin and subcutaneous tissue disorders Dry Skin	12.5% 1/8 • up to 18 months
Skin and subcutaneous tissue disorders Itchy Skin	12.5% 1/8 • up to 18 months
Vascular disorders Hypertension	12.5% 1/8 • up to 18 months
Eye disorders Blepharitis	12.5% 1/8 • up to 18 months
General disorders Imbalance	12.5% 1/8 • up to 18 months
General disorders Edema, bilateral	12.5% 1/8 • up to 18 months

Additional Information

Andrew R. Rezvani, MD

Stanford University

Phone: 650-725-4077

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place