Rare leptin disorder patients gain access to experimental drug

NCT ID NCT04710056

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This program provides expanded access to REGN4461 (mibavademab) for people with rare diseases caused by deficient leptin signaling, such as generalized lipodystrophy and monogenic obesity. The drug aims to help control metabolic issues linked to these conditions. Participants receive the medication outside of a formal clinical trial.

What this could mean

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

Active substance

REGN4461 (mibavademab)

What this could lead to

If it works, this could help control metabolic complications in people with rare leptin-signaling disorders.

What could go wrong

This is an expanded access program, not a formal trial, so evidence on safety and effectiveness is limited. Benefits may vary and risks are not fully known.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

generalized lipodystrophy inherited obesity lipodystrophy Lipodystrophy, Congenital Generalized

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••