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NCT04350606

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

Completed Phase 3 Results posted Last updated 27 April 2022
What this trial tests

Phase 3 trial testing PF-06462700 in Aplastic Anemia in 3 participants. Completed in 19 April 2021.

Timeline
25 July 2020
Primary endpoint
22 January 2021
19 April 2021

Quick facts

Lead sponsorPfizer
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment3
Start date25 July 2020
Primary completion22 January 2021
Estimated completion19 April 2021
Sites3 locations across Japan

Drugs / interventions tested

Conditions studied

Sponsor

Pfizer — full company profile →

Who can join

2 and older, any sex, with Aplastic Anemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Hematologic Response at Week 12 Primary · Week 12 Follow-up Visit

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.

Effective
GroupValue95% CI
PF-064627002
Not Effective
GroupValue95% CI
PF-064627001
Number of Participants With Hematologic Response at Week 24 Secondary · Week 24 Follow-up Visit

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.

Effective
GroupValue95% CI
PF-064627002
Not Effective
GroupValue95% CI
PF-064627001
Absolute Neutrophil Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24 Secondary · Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24
GroupValue95% CI
PF-06462700NA
Platelet Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24 Secondary · Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24
GroupValue95% CI
PF-06462700NA
Reticulocyte Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24 Secondary · Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24
GroupValue95% CI
PF-06462700NA
Number of Participants Who Survived During the Study Secondary · Screening (up to 28 days prior to Day 1 of treatment) up to 24 weeks of follow-up (approximately up to 28 weeks)

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

GroupValue95% CI
PF-064627003
Number of Participants With Transfusion Independence at Weeks 12 and 24 Secondary · 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)

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).

Week 12 Transfusion Independence
GroupValue95% CI
PF-064627000
Week 24 Transfusion Independence
GroupValue95% CI
PF-064627002

Adverse events — posted to ClinicalTrials.gov

Time frame: Screening up to 24 weeks of follow-up (approximately up to 28 weeks). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

PF-06462700
Serious: 0/3 (0%)
Deaths: 0/3
Other adverse events (38 terms — click to expand)

ReactionSystemPF-06462700
Abdominal painGastrointestinal disorders
NauseaGastrointestinal disorders
HyperglycaemiaMetabolism and nutrition disorders
HypertensionVascular disorders
Adrenal insufficiencyEndocrine disorders
ConstipationGastrointestinal disorders
Dental cariesGastrointestinal disorders
Gastrointestinal disorderGastrointestinal disorders
Gastrooesophageal reflux diseaseGastrointestinal disorders
ProctalgiaGastrointestinal disorders
Feeling abnormalGeneral disorders
Infusion site extravasationGeneral disorders
OedemaGeneral disorders
HypogammaglobulinaemiaImmune system disorders
Serum sicknessImmune system disorders
Cytomegalovirus infectionInfections and infestations
Cytomegalovirus viraemiaInfections and infestations
Staphylococcal infectionInfections and infestations
Alanine aminotransferase increasedInvestigations
Aspartate aminotransferase increasedInvestigations
Blood bilirubin increasedInvestigations
Blood creatinine increasedInvestigations
Blood lactate dehydrogenase increasedInvestigations
C-reactive protein increasedInvestigations
Gamma-glutamyltransferase increasedInvestigations
Lymphocyte count decreasedInvestigations
Oxygen saturation abnormalInvestigations
White blood cell count decreasedInvestigations
HypokalaemiaMetabolism and nutrition disorders
TremorNervous system disorders
InsomniaPsychiatric disorders
ProteinuriaRenal and urinary disorders
Renal impairmentRenal and urinary disorders
Genital haemorrhageReproductive system and breast disorders
Productive coughRespiratory, thoracic and mediastinal disorders
AcneSkin and subcutaneous tissue disorders
Dry skinSkin and subcutaneous tissue disorders
Nail bed inflammationSkin and subcutaneous tissue disorders

Data from ClinicalTrials.gov NCT04350606 adverse events section.

Sponsor's own description

The purpose of the study is to assess the efficacy and safety of PF-06462700 administered intravenously at 40 mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia for making an approval application in Japan.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

Verify or expand the search:

Other recruiting trials for Aplastic Anemia

Currently open trials in the same condition.

Other Pfizer trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04350606.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing