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Experimental drug shows promise for rare immune disorder, but study cut short

NCT ID NCT02859727

Disease control Sponsor: Pharming Technologies B.V. 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
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the long-term safety and effectiveness of a drug called CDZ173 in people with APDS/PASLI, a rare genetic condition that causes a weak immune system and swollen lymph nodes. The 37 participants had already taken CDZ173 or a similar drug in a previous study. The trial was terminated early, so the full picture is unclear, but researchers were looking at side effects and quality of life improvements.

What this could mean

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

Active substance
CDZ173 (a drug that blocks a specific enzyme to help control immune system overactivity)
What this could lead to
If successful, this could provide a long-term treatment option to manage APDS/PASLI, reducing infections and improving quality of life.
What could go wrong
The study was terminated early, so results are limited. It is a small, open-label extension study, meaning no placebo group and potential bias. Long-term safety and efficacy are not fully established.

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.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

37 people

The number who actually took part.

Started

Sep 2016

Finished

Jan 2025

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.

Ages

12 to 75 years

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: * Written informed consent must be obtained before any assessment is performed. * Paients must have participated in the study CCDZ173X2201 or were treated previously with PI3Kδ inhibitors other than CDZ173. * Patients who are deemed by the Investigator to benefit from PI3Kδ inhibitor therapy. * Patients or their legal representatives (for patients under the age of 18 years) must be able to communicate well with the Investigator, to understand and comply with the requirements of the study. * Documented APDS/PASLI-associated genetic PI3K delta mutation. Exclusion Criteria: \- Any medically significant disease or condition that is unrelated to APDS/PASLI Other protocol-defined inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

activated PI3K-delta syndrome immunodeficiency 14 inborn error of immunity Lymphadenopathy

As listed by the trial registrant

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

Activated PI3KDELTA syndrome (APDS); pasli disease

Contacts and locations

Locations

  • Pharming Investigative Site

    Bethesda, Maryland, 20892, United States

  • Pharming Investigative Site

    Minsk, 223053, Belarus

  • Pharming Investigative Site

    Prague, CZE, 15006, Czechia

  • Pharming Investigative Site

    Dresden, 01307, Germany

  • Pharming Investigative Site

    Brescia, BS, 25123, Italy

  • Pharming Investigative Site

    Palermo, PA, 90127, Italy

  • Pharming Investigative Site

    Rotterdam, 3000 CA, Netherlands

  • Pharming Investigative Site

    Moscow, 117198, Russia

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