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Single-Patient trial tests safety of ATGAM for rare blood disorder

NCT ID NCT06039020

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study is testing the safety of a drug called ATGAM in one patient with moderate to severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The patient receives ATGAM by IV for 4 days and is monitored for 24 weeks for side effects. The goal is to confirm the drug's safety in Japanese patients.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Anti-human thymocyte immunoglobulin (equine)
What this could lead to
If successful, this could confirm that ATGAM is safe for use in Japanese patients with aplastic anemia, supporting its continued use as a treatment.
What could go wrong
This is a very small study with only one participant, so results may not apply to others. It focuses on safety, not effectiveness, and the drug may cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

1 person

The number who actually took part.

Started

Feb 2024

Expected to finish

Jan 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients with moderate to severe aplastic anemia who receive ATGAM

Ages

0 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients with moderate to severe aplastic anemia who receive ATGAM Exclusion Criteria: * No exclusion criteria is set out in this study.

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Conditions

The condition(s) this trial relates to.

aplastic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Aplastic anemia

Contacts and locations

Locations

  • Pfizer

    Tokyo, Japan

More trials for these conditions

Other studies related to the condition(s) this trial covers.