Trial Outcomes & Findings for Pilot Study of Pembrolizumab Treatment for Disease Relapse After Allogeneic Stem Cell Transplantation (NCT NCT02981914)
NCT ID: NCT02981914
Last Updated: 2024-04-22
Results Overview
A DLT is defined as the development of grade 3 or 4 acute GVHD (defined by the Gluckenberg scale), graft rejection, the development of any unexpected grade \> 2 toxicity felt to be related to pembrolizumab, or the development of \> grade 2 dysfunction of a vital organ felt to be secondary to an immune-related adverse event within 90 days following the initiation of pembrolizumab treatment.
Recruitment status
COMPLETED
Study phase
EARLY_PHASE1
Target enrollment
12 participants
Primary outcome timeframe
90 days
Results posted on
2024-04-22
Participant Flow
Participant milestones
| Measure |
Pembrolizumab
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pilot Study of Pembrolizumab Treatment for Disease Relapse After Allogeneic Stem Cell Transplantation
Baseline characteristics by cohort
| Measure |
Pembrolizumab
n=12 Participants
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Age, Continuous
|
50.7 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 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
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 90 daysA DLT is defined as the development of grade 3 or 4 acute GVHD (defined by the Gluckenberg scale), graft rejection, the development of any unexpected grade \> 2 toxicity felt to be related to pembrolizumab, or the development of \> grade 2 dysfunction of a vital organ felt to be secondary to an immune-related adverse event within 90 days following the initiation of pembrolizumab treatment.
Outcome measures
| Measure |
Pembrolizumab
n=12 Participants
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Number of Patients With Dose-limiting Toxicities (DLTs)
|
3 Participants
|
SECONDARY outcome
Timeframe: From date of response until the date of first documented progression or relapse, whichever came first, assessed up to 12 monthsPopulation: Note: one patient censored at 54 days, second patient had disease progression at 364 days. Per the area under the Kaplan-Meier curve, the mean would be estimated as 364 days. However this estimate is unreliable given that it is based on only one censored observation and one event. The censored observation has been omitted in the full range below, as "54+" is rejected as a valid entry.
To determine the duration of response (DOR) of patients treated with pembrolizumab in the setting of relapsed myeloid malignancies and mature B cell lymphomas following alloSCT.
Outcome measures
| Measure |
Pembrolizumab
n=2 Participants
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Time Between Initial Response and Subsequent Disease Progression or Relapse
|
364 days
Interval 364.0 to 364.0
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: The trial enrolled 8 AML patients, 3 lymphoma patients, and 1 MDS patient. Both responders were lymphoma patients.
For B cell lymphoma patients, partial and complete responses will be defined according to the International Working Group criteria. For MDS patients, partial and complete responses (including cytogenetic responses) will be defined according to the International Working Group criteria. Lastly, for AML patients, complete (including CR, CRp, CRi), and partial responses will be defined according to the European Leukemia Net response criteria.
Outcome measures
| Measure |
Pembrolizumab
n=12 Participants
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Objective Response Rate
|
2 Participants
|
SECONDARY outcome
Timeframe: From start date of therapy to the date of death from any cause, whichever may come first, assessed up to 24 monthsSurvival time in days, patients alive as of last follow-up were censored
Outcome measures
| Measure |
Pembrolizumab
n=12 Participants
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Time Between the Start of Therapy to Death From Any Cause.
|
285 days
Interval 21.0 to
NA = Upper confidence limit cannot be estimated due to insufficient number of participants with events
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 monthsPopulation: Data were not collected.
To determine the effect of pembrolizumab on restoring donor chimerism in patients with relapsed myeloid malignancies and mature B cell lymphomas following alloSCT.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 monthsPopulation: Data were not collected.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 monthsPopulation: Data were not collected.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: 24 monthsPopulation: Data were not collected.
Outcome measures
Outcome data not reported
Adverse Events
Pembrolizumab
Serious events: 7 serious events
Other events: 11 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Pembrolizumab
n=12 participants at risk
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Secondary cancer
|
8.3%
1/12 • 24 months
|
|
Investigations
Platelet count decreased
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
16.7%
2/12 • 24 months
|
|
Infections and infestations
Pneumonia
|
8.3%
1/12 • 24 months
|
|
Blood and lymphatic system disorders
Hemolytic anemia
|
8.3%
1/12 • 24 months
|
|
General disorders
Fatigue
|
8.3%
1/12 • 24 months
|
|
General disorders
Fever
|
16.7%
2/12 • 24 months
|
|
Endocrine disorders
Hyperthyroidism
|
8.3%
1/12 • 24 months
|
Other adverse events
| Measure |
Pembrolizumab
n=12 participants at risk
Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks. Treatment will be administered for up to 24 months, provided that neither disease progression, nor development of a dose-limiting toxicity (DLT), has occurred.
Pembrolizumab: Pembrolizumab will be administered at a fixed dose of 200 mg IV every 3 weeks for a max of 24 months, so long as there is no disease progression.
|
|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
8.3%
1/12 • 24 months
|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
3/12 • 24 months
|
|
Renal and urinary disorders
Acute kidney injury
|
8.3%
1/12 • 24 months
|
|
Investigations
Alanine aminotransferase increased
|
8.3%
1/12 • 24 months
|
|
Investigations
Alkaline phosphatase increased
|
8.3%
1/12 • 24 months
|
|
Gastrointestinal disorders
Anal ulcer
|
8.3%
1/12 • 24 months
|
|
Blood and lymphatic system disorders
Anemia
|
50.0%
6/12 • 24 months
|
|
Metabolism and nutrition disorders
Anorexia
|
8.3%
1/12 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
41.7%
5/12 • 24 months
|
|
Investigations
Aspartate aminotransferase increased
|
8.3%
1/12 • 24 months
|
|
Metabolism and nutrition disorders
Acidosis
|
8.3%
1/12 • 24 months
|
|
Cardiac disorders
Atrial fibrillation
|
8.3%
1/12 • 24 months
|
|
Immune system disorders
Autoimmune hemolytic anemia
|
8.3%
1/12 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
1/12 • 24 months
|
|
Investigations
Blood bilirubin increased
|
8.3%
1/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Bullous dermatitis
|
8.3%
1/12 • 24 months
|
|
General disorders
Chills
|
8.3%
1/12 • 24 months
|
|
Gastrointestinal disorders
Constipation
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Coronavirus PNA
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
2/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Crackles (bilateral)
|
8.3%
1/12 • 24 months
|
|
Investigations
Creatinine increased
|
8.3%
1/12 • 24 months
|
|
General disorders
Diaphoresis
|
8.3%
1/12 • 24 months
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
4/12 • 24 months
|
|
Gastrointestinal disorders
Diverticulitis
|
8.3%
1/12 • 24 months
|
|
Nervous system disorders
Dizziness
|
16.7%
2/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.3%
1/12 • 24 months
|
|
Gastrointestinal disorders
Dysphagia
|
8.3%
1/12 • 24 months
|
|
Renal and urinary disorders
Dysuria
|
8.3%
1/12 • 24 months
|
|
Injury, poisoning and procedural complications
Ecchymoses
|
8.3%
1/12 • 24 months
|
|
General disorders
Edema
|
33.3%
4/12 • 24 months
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
4/12 • 24 months
|
|
Infections and infestations
Enterocolitis infectious
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
16.7%
2/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
8.3%
1/12 • 24 months
|
|
General disorders
Fatigue
|
50.0%
6/12 • 24 months
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
8.3%
1/12 • 24 months
|
|
General disorders
Fever
|
58.3%
7/12 • 24 months
|
|
General disorders
Gum bleeding
|
8.3%
1/12 • 24 months
|
|
Nervous system disorders
Headache
|
25.0%
3/12 • 24 months
|
|
Renal and urinary disorders
Hematuria
|
8.3%
1/12 • 24 months
|
|
Gastrointestinal disorders
Hemorrhoids
|
8.3%
1/12 • 24 months
|
|
Investigations
High FT4
|
8.3%
1/12 • 24 months
|
|
Vascular disorders
Hypertension
|
8.3%
1/12 • 24 months
|
|
Endocrine disorders
Hyperthyroidism
|
8.3%
1/12 • 24 months
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
8.3%
1/12 • 24 months
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
8.3%
1/12 • 24 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
8.3%
1/12 • 24 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
8.3%
1/12 • 24 months
|
|
Vascular disorders
Hypotension
|
33.3%
4/12 • 24 months
|
|
Endocrine disorders
Hypothyroidism
|
16.7%
2/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
8.3%
1/12 • 24 months
|
|
Psychiatric disorders
Insomnia
|
8.3%
1/12 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
8.3%
1/12 • 24 months
|
|
Blood and lymphatic system disorders
Leukocytosis
|
8.3%
1/12 • 24 months
|
|
Investigations
Low TSH
|
8.3%
1/12 • 24 months
|
|
Hepatobiliary disorders
Mild scleral icterus
|
8.3%
1/12 • 24 months
|
|
Infections and infestations
Molluscum contagiosum
|
8.3%
1/12 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
16.7%
2/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
8.3%
1/12 • 24 months
|
|
Gastrointestinal disorders
Nausea
|
66.7%
8/12 • 24 months
|
|
Investigations
Neutropenic fever
|
8.3%
1/12 • 24 months
|
|
Investigations
Neutrophil count decreased
|
33.3%
4/12 • 24 months
|
|
General disorders
Non-cardiac chest pain
|
8.3%
1/12 • 24 months
|
|
General disorders
Oral ulcer
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Organizing pneumonia
|
8.3%
1/12 • 24 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.3%
1/12 • 24 months
|
|
Cardiac disorders
Palpitations
|
8.3%
1/12 • 24 months
|
|
Investigations
Pancytopenia
|
8.3%
1/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Periorbital edema
|
8.3%
1/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
8.3%
1/12 • 24 months
|
|
General disorders
Pharynx erythematous
|
8.3%
1/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Photosensitivity
|
8.3%
1/12 • 24 months
|
|
Investigations
Platelet count decreased
|
41.7%
5/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary infiltrates
|
16.7%
2/12 • 24 months
|
|
General disorders
Right side flank pain
|
8.3%
1/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
6/12 • 24 months
|
|
Renal and urinary disorders
Renal calculi
|
8.3%
1/12 • 24 months
|
|
Renal and urinary disorders
Renal colic
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
8.3%
1/12 • 24 months
|
|
Infections and infestations
Rhinovirus (pos)
|
8.3%
1/12 • 24 months
|
|
General disorders
Shortness of breath
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
8.3%
1/12 • 24 months
|
|
Nervous system disorders
Sinus pain
|
8.3%
1/12 • 24 months
|
|
Cardiac disorders
Sinus tachycardia
|
25.0%
3/12 • 24 months
|
|
Skin and subcutaneous tissue disorders
Skin rash
|
8.3%
1/12 • 24 months
|
|
Infections and infestations
Sore throat
|
16.7%
2/12 • 24 months
|
|
Endocrine disorders
Subacute thyroiditis
|
8.3%
1/12 • 24 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
T-AML
|
8.3%
1/12 • 24 months
|
|
Cardiac disorders
Tachycardia
|
8.3%
1/12 • 24 months
|
|
General disorders
Tender abdomen
|
8.3%
1/12 • 24 months
|
|
Vascular disorders
Thromboembolic event
|
8.3%
1/12 • 24 months
|
|
Ear and labyrinth disorders
Tinnitus
|
8.3%
1/12 • 24 months
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory infection
|
8.3%
1/12 • 24 months
|
|
Renal and urinary disorders
Urinary retention
|
8.3%
1/12 • 24 months
|
|
Reproductive system and breast disorders
Vaginal hemorrhage
|
8.3%
1/12 • 24 months
|
|
Infections and infestations
Urinary tract infection
|
8.3%
1/12 • 24 months
|
|
General disorders
Weakness
|
8.3%
1/12 • 24 months
|
|
Investigations
Weight loss
|
16.7%
2/12 • 24 months
|
|
Investigations
White blood cell decreased
|
16.7%
2/12 • 24 months
|
Additional Information
Theodore Karrison (Research Professor)
University of Chicago
Phone: 773-702-9326
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place