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

NCT ID NCT06039020

Ongoing Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

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.

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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.