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Aplastic AnemiaPhase 3NCT04350606

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

A Phase 3 study testing PF-06462700 in people with Aplastic Anemia, run by Pfizer at 3 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Aplastic Anemia
What is being tested?
PF-06462700
Type of treatment
Vaccines
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
3 sitesJapan
Study dates
25 Jul 2020to 19 Apr 2021
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male or female participants between the ages of 2 years and more, inclusive, at Visit
  • 1 (Screening).
  • Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
  • Have a clinical diagnosis of aplastic anemia by bone marrow aspiration/biopsy findings and/or magnetic resonance imaging (MRI) etc.
  • Must meet the following criteria of moderate and above aplastic anemia
  • Capable of giving signed informed consent/assent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the informed consent document (ICD)/assent document and in this protocol.

Who cannot join

  • Eligible and willing to have a sibling allogeneic stem cell transplantation.
  • Evidence of a myelodysplastic syndrome (except for refractory cytopenia in children), as well as other primitive marrow disease.
  • History or clinical suspicion of congenital aplastic anemia (Fanconi anemia, Congenital keratosis, etc).
  • History of malignant tumors with active disease within 5 years from study participation.
  • Participants who are clearly infected with hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV), and human T-cell leukemia virus type
  • 1 (HTLV-1).
Show all 17Show fewer
  • Pregnant or breast-feeding participants.
  • Participants with severe hepatic, renal or cardiac failure, or any other life-threatening concurrent [aspartate aminotransferase (AST) or alanine aminotransferase (ALT) or total bilirubin values >5 × upper limit of normal (ULN), and/or creatinine value >2 × ULN].
  • Participants with hypersensitivity such as shock after skin test of this study drug.
  • Participants with uncontrolled severe infection (pneumonia, sepsis, etc).
  • Participants who received live vaccine or live attenuated vaccine within 6 weeks prior to the first dose of study drug.
  • Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
  • Prior immunosuppressive therapy with lymphocyte-depleting agents/therapies, including both non-B-cell selective and B-cell-depleting agents (e.g., alefacept, alemtuzumab, rituximab). However, participants previously treated with rATG may enroll.
  • Previous history of stem cell transplantation.
  • Previous administration with an investigational drug within 30 days (or as determined by the local requirement) or 5 half-lives preceding the first dose of investigational product used in this study (whichever is longer).
  • Baseline 12-lead electrocardiogram (ECG) that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results (e.g., baseline corrected QT [QTc] interval >450 msec, complete left bundle branch block [LBBB], signs of an acute or indeterminate-age myocardial infarction, ST-T interval changes suggestive of myocardial ischemia, second- or third-degree atrioventricular [AV] block, or serious bradyarrhythmias or tachyarrhythmias). If the baseline uncorrected QT interval is >450 msec, this interval should be rate-corrected using the Fridericia method and the resulting QTcF should be used for decision making and reporting. If QTc exceeds 450 msec, or QRS exceeds 120 msec, the ECG should be repeated 2 more times and the average of the 3 QTc or QRS values should be used to determine the participant's eligibility. Computer-interpreted ECGs should be overread by a physician experienced in reading ECGs before excluding participants.
  • Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or Pfizer employees, including their family members, directly involved in the conduct of the study.

Trial locations and contacts

3 sites in 1 country.

Japan3 sites

  • Nagoya University Hospital

    Nagoya, Aichi-ken, 466-8560

  • Jichi Medical University Hospital

    Shimotsuke, Tochigi, 329 0498

  • Keio University Hospital

    Shinjuku-ku, Tokyo, 160-8582

Other trials in Japan

About this study

Official title: A MULTICENTER, OPEN-LABEL, SINGLE-ARM STUDY 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

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.

Conditions
Aplastic Anemia
Being tested
PF-06462700
Keywords
Aplastic Anemia, globulin, ATG, Anti-human Thymocyte Immunogloblin
Medical subject headings
Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PF-06462700Biological

    Also called Brand name in the US: ATGAM

    PF-06462700 is classified as an immunosuppressant/ immunosuppressive agent. It is the purified, concentrated, and sterile gamma globulin, primarily monomeric immunoglobulin G (IgG), from hyperimmune serum of horses that are immunized with human thymus lymphocytes.

Study groups in NCT04350606
Study groupRoleWhat they receive
PF-006462700 groupExperimentalAll enrolled participants will be administrated PF-006462700.
Receives: Biological: PF-06462700

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Hematologic Response at Week 12

    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.

    Measured: Week 12 Follow-up Visit

Secondary outcomes (6)

  1. Number of Participants With Hematologic Response at Week 24

    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.

    Measured: Week 24 Follow-up Visit

  2. Absolute Neutrophil Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24

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

  3. Platelet Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24

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

  4. Reticulocyte Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24

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

  5. Number of Participants Who Survived During the Study

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

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

  6. Number of Participants With Transfusion Independence at Weeks 12 and 24

    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).

    Measured: 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)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Apr 2020
  2. Study start25 Jul 2020
  3. Primary completion22 Jan 2021
  4. Study completion19 Apr 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04350606
Sponsor's protocol ID
B5411003
Other IDs
ATGAM Japan local ph3 study
Status last verified
Mar 2022
First submitted
1 Apr 2020
Last updated
27 Apr 2022
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Publications

  • Kanda Y, Mori T, Narita A, Wolter KD, Yoshimatsu H, Nishimura K. Efficacy and safety of equine anti-thymocyte immunoglobulin (eATG) in three Japanese patients with moderate to very severe aplastic anemia: a case series. Int J Hematol. 2023 Jan;117(1):37-43. doi: 10.1007/s12185-022-03496-5. Epub 2022 Nov 28.

    PubMed 36441357

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing PF-06462700 in people with Aplastic Anemia, run by Pfizer at 3 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 27 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 Apr 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Hematologic Response at Week 12 (time frame: Week 12 Follow-up Visit). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04350606, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2022; AzTrials last refreshed its copy on 8 Nov 2026.

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