Trial Outcomes & Findings for Acalabrutinib for the Treatment of Relapsed or Refractory Autoimmune Hemolytic Anemia in Patients With Chronic Lymphocytic Leukemia (NCT NCT04657094)
NCT ID: NCT04657094
Last Updated: 2025-04-27
Results Overview
ORR is defined as proportion of patients who achieve complete response (CR) and partial response (PR). The probability of having AIHA-ORR at 6 cycles were measured and reported with 95% exact confidence interval (CI). An exact binomial test against a null hypothesis of 30% rate was performed at the 1-sided alpha of 0.05 to determine whether the AIHA-ORR rate at 6 cycles is disappointing or promising.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
Participants were assessed at the end of the 6-week therapy.
Results posted on
2025-04-27
Participant Flow
Participant milestones
| Measure |
Treatment (Acalabrutinib)
Patients receive acalabrutinib PO BID on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Treatment with acalabrutinib may be continued beyond 12 cycles for a maximum of 36 cycles if, in the opinion of the treating physician, the patient might benefit from ongoing therapy.
Acalabrutinib: Given PO
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Acalabrutinib for the Treatment of Relapsed or Refractory Autoimmune Hemolytic Anemia in Patients With Chronic Lymphocytic Leukemia
Baseline characteristics by cohort
| Measure |
Treatment (Acalabrutinib)
n=4 Participants
Patients receive acalabrutinib PO BID on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Treatment with acalabrutinib may be continued beyond 12 cycles for a maximum of 36 cycles if, in the opinion of the treating physician, the patient might benefit from ongoing therapy.
Acalabrutinib: Given PO
|
|---|---|
|
Age, Continuous
|
59 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 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
|
1 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
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Participants were assessed at the end of the 6-week therapy.Population: Among the 4 enrolled participants, one was in-evaluable for not finishing 6 cycles of the study treatment due to toxicity.
ORR is defined as proportion of patients who achieve complete response (CR) and partial response (PR). The probability of having AIHA-ORR at 6 cycles were measured and reported with 95% exact confidence interval (CI). An exact binomial test against a null hypothesis of 30% rate was performed at the 1-sided alpha of 0.05 to determine whether the AIHA-ORR rate at 6 cycles is disappointing or promising.
Outcome measures
| Measure |
Treatment (Acalabrutinib)
n=3 Participants
Patients receive acalabrutinib PO BID on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Treatment with acalabrutinib may be continued beyond 12 cycles for a maximum of 36 cycles if, in the opinion of the treating physician, the patient might benefit from ongoing therapy.
Acalabrutinib: Given PO
|
|---|---|
|
Autoimmune Hemolytic Anemia (AIHA) - Overall Response Rate (ORR)
|
67 percentage of participants
Interval 9.0 to 99.0
|
Adverse Events
Treatment (Acalabrutinib)
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Acalabrutinib)
n=4 participants at risk
Patients receive acalabrutinib PO BID on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Treatment with acalabrutinib may be continued beyond 12 cycles for a maximum of 36 cycles if, in the opinion of the treating physician, the patient might benefit from ongoing therapy.
Acalabrutinib: Given PO
|
|---|---|
|
Immune system disorders
DRESS Syndrome
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Infections and infestations
COVID-19
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
Other adverse events
| Measure |
Treatment (Acalabrutinib)
n=4 participants at risk
Patients receive acalabrutinib PO BID on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Treatment with acalabrutinib may be continued beyond 12 cycles for a maximum of 36 cycles if, in the opinion of the treating physician, the patient might benefit from ongoing therapy.
Acalabrutinib: Given PO
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Gastrointestinal disorders
Dental Fractures
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Gastrointestinal disorders
Mucositis oral
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
General disorders
Edema limbs
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
General disorders
Fatigue
|
75.0%
3/4 • Number of events 3 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
General disorders
Neck Numbness
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
General disorders
Non-cardiac chest pain
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Immune system disorders
DRESS Syndrome
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Infections and infestations
Skin infection
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Injury, poisoning and procedural complications
Bruising
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Injury, poisoning and procedural complications
Fall
|
50.0%
2/4 • Number of events 2 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
Alanine aminotransferase increased
|
50.0%
2/4 • Number of events 3 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
Aspartate aminotransferase increased
|
50.0%
2/4 • Number of events 4 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
Blood lactate dehydrogenase increased
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
INR increased
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
Lymphocyte count decreased
|
50.0%
2/4 • Number of events 3 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
Lymphocyte count increased
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
Neutrophil count decreased
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
Platelet count decreased
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Investigations
White blood cell decreased
|
50.0%
2/4 • Number of events 6 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Metabolism and nutrition disorders
Anorexia
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
50.0%
2/4 • Number of events 4 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
50.0%
2/4 • Number of events 3 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
50.0%
2/4 • Number of events 2 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
50.0%
2/4 • Number of events 5 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
50.0%
2/4 • Number of events 3 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Psychiatric disorders
Delirium
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
50.0%
2/4 • Number of events 2 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
25.0%
1/4 • Number of events 1 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
50.0%
2/4 • Number of events 5 • From the initial study treatment up to 12 months after the end of the treatment, approximately two years.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place