Trial Outcomes & Findings for A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia (NCT NCT04350606)

NCT ID: NCT04350606

Last Updated: 2022-04-27

Results Overview

Hematologic response was considered to be "effective" when 2 or more of the following criteria were met: absolute neutrophil count greater than or equal to (\>=) 500 per microliters, platelet count \>=20,000 per microliters and reticulocyte count \>=60,000 per microliters was observed. In this outcome measure, number of participants with hematologic response classified as effective and not effective were reported. Improvement in counts that were dependent upon exogenously administered growth factors or transfusion, was not considered as fulfilling response criteria.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 3 participants
• Primary outcome timeframe: Week 12 Follow-up Visit
• Results posted on: 2022-04-27

Participant Flow

Total 3 participants signed the inform consent form (ICF). Out of which 0 participants were screen failures, 3 actually enrolled into the study and assigned to a study treatment.

Participant milestones

Measure	PF-06462700 Participants aged 2 years or greater than (\>) 2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 milligram per kilogram per day (mg/kg/day), intravenously (IV) for 4 days. Participants after treatment were followed up for 24 weeks.
Overall Study STARTED	3
Overall Study Treated	3
Overall Study Follow-up	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia

Baseline characteristics by cohort

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Age, Continuous	29.67 Years STANDARD_DEVIATION 16.56 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 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	3 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	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 12 Follow-up Visit

Population: Full analysis set (FAS) included participants were assigned to investigational product and who took at least 1 dose of investigational product.

Hematologic response was considered to be "effective" when 2 or more of the following criteria were met: absolute neutrophil count greater than or equal to (>=) 500 per microliters, platelet count >=20,000 per microliters and reticulocyte count >=60,000 per microliters was observed. In this outcome measure, number of participants with hematologic response classified as effective and not effective were reported. Improvement in counts that were dependent upon exogenously administered growth factors or transfusion, was not considered as fulfilling response criteria.

Outcome measures

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Number of Participants With Hematologic Response at Week 12 Effective	2 Participants
Number of Participants With Hematologic Response at Week 12 Not Effective	1 Participants

SECONDARY outcome

Timeframe: Week 24 Follow-up Visit

Population: FAS included participants who were assigned to investigational product and who took at least 1 dose of investigational product.

Hematologic response was considered to be "effective" when 2 or more of the following criteria were met: absolute neutrophil count >=500 per microliters, platelet count >=20,000 per microliters and reticulocyte count >=60,000 per microliters was observed. In this outcome measure, number of participants with hematologic response classified as effective and not effective were reported. Improvement in counts that were dependent upon exogenously administered growth factors or transfusion, was not been considered as fulfilling response criteria.

Outcome measures

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Number of Participants With Hematologic Response at Week 24 Effective	2 Participants
Number of Participants With Hematologic Response at Week 24 Not Effective	1 Participants

SECONDARY outcome

Timeframe: Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24

Population: FAS included participants who were assigned to investigational product and who took at least 1 dose of investigational product.

Outcome measures

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Absolute Neutrophil Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24	NA Neutrophil cells per microliter It was planned data would not be summarized. Individual data cannot be reported for this outcome measure because they are directly personal information.

SECONDARY outcome

Timeframe: Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24

Population: FAS included participants who were assigned to investigational product and who took at least 1 dose of investigational product.

Outcome measures

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Platelet Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24	NA Platelet cells per microliter It was planned data would not be summarized. Individual data cannot be reported for this outcome measure because they are directly personal information.

SECONDARY outcome

Timeframe: Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24

Population: FAS included participants who were assigned to investigational product and who took at least 1 dose of investigational product.

Outcome measures

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Reticulocyte Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24	NA Reticulocyte cells per microliter It was planned data would not be summarized. Individual data cannot be reported for this outcome measure because they are directly personal information.

SECONDARY outcome

Timeframe: Screening (up to 28 days prior to Day 1 of treatment) up to 24 weeks of follow-up (approximately up to 28 weeks)

Population: FAS included participants who were assigned to investigational product and who took at least 1 dose of investigational product.

In this outcome measure, number of participants who survived during the study were observed.

Outcome measures

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Number of Participants Who Survived During the Study	3 Participants

SECONDARY outcome

Timeframe: Week 12 Transfusion Independence: Day 1 of Treatment up to Week 12 Follow-up Visit (approximately 12 weeks); Week 24 Transfusion Independence: Day after Week 12 Follow-up visit to Week 24 Follow-up Visit (approximately 12 weeks)

Population: FAS included participants who were assigned to investigational product and who took at least 1 dose of investigational product.

Transfusion independence at Week 12 was defined as when participants did not have any transfusion records from the time of the first dose of the investigational product at Day 1 to the day of Week 12 follow-up visit (inclusive). Transfusion independence at Week 24 was defined as when participants did not have any transfusion records from the day after Week 12 follow-up visit to the day of Week 24 follow-up visit (inclusive).

Outcome measures

Measure	PF-06462700 n=3 Participants Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Number of Participants With Transfusion Independence at Weeks 12 and 24 Week 12 Transfusion Independence	0 Participants
Number of Participants With Transfusion Independence at Weeks 12 and 24 Week 24 Transfusion Independence	2 Participants

Adverse Events

PF-06462700

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	PF-06462700 n=3 participants at risk Participants aged 2 years or \>2 years with moderate and above severity aplastic anemia received PF-06462700 at a dose of 40 mg/kg/day, IV for 4 days. Participants after treatment were followed up for 24 weeks.
Endocrine disorders Adrenal insufficiency	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Gastrointestinal disorders Abdominal pain	66.7% 2/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Gastrointestinal disorders Constipation	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Gastrointestinal disorders Dental caries	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Gastrointestinal disorders Gastrointestinal disorder	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Gastrointestinal disorders Gastrooesophageal reflux disease	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Gastrointestinal disorders Nausea	66.7% 2/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Gastrointestinal disorders Proctalgia	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
General disorders Feeling abnormal	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
General disorders Infusion site extravasation	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
General disorders Oedema	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Immune system disorders Hypogammaglobulinaemia	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Immune system disorders Serum sickness	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Infections and infestations Cytomegalovirus infection	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Infections and infestations Cytomegalovirus viraemia	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Infections and infestations Staphylococcal infection	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Alanine aminotransferase increased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Aspartate aminotransferase increased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Blood bilirubin increased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Blood creatinine increased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Blood lactate dehydrogenase increased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations C-reactive protein increased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Gamma-glutamyltransferase increased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Lymphocyte count decreased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations Oxygen saturation abnormal	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Investigations White blood cell count decreased	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Metabolism and nutrition disorders Hyperglycaemia	66.7% 2/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Metabolism and nutrition disorders Hypokalaemia	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Nervous system disorders Tremor	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Psychiatric disorders Insomnia	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Renal and urinary disorders Proteinuria	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Renal and urinary disorders Renal impairment	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Reproductive system and breast disorders Genital haemorrhage	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Respiratory, thoracic and mediastinal disorders Productive cough	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Skin and subcutaneous tissue disorders Acne	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Skin and subcutaneous tissue disorders Dry skin	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Skin and subcutaneous tissue disorders Nail bed inflammation	33.3% 1/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)
Vascular disorders Hypertension	66.7% 2/3 • Screening up to 24 weeks of follow-up (approximately up to 28 weeks)

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer Inc.

Phone: 1-800-718-1021

Email: ClinicalTrials.gov_Inquiries@pfizer.com

Results disclosure agreements

• Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from the study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
• Publication restrictions are in place

Restriction type: OTHER