Pharming Clinical Trials

Pharming, in coordination with Novartis Pharmaceuticals, is recruiting patients for the NCT02435173 clinical trial evaluating an experimental treatment for APDS, a primary immune deficiency.

This study is designed to explore CDZ173 (leniolisib), an investigational selective APDS inhibitor, in patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI (APDS/p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency).

Pharming is looking for patients and healthcare professionals who are interested in learning more about APDS and leniolisib, the current study protocol (inclusion/exclusion criteria) and potentially participating in the current or future clinical trials.

By completing this form, you are agreeing to receive communications from Pharming Healthcare Inc, its affiliates, and their agents and representatives regarding clinical trials, APDS and Leniolisib. You may opt out at any time.

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Are you a Health Care Provider?^*

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This registration form is for Health Care Providers only.

First Name^*

Last Name^*

Email Address^*

Phone Number^*

Title

Hospital Affiliation

Specialty^*

Specialty: Other

Practice Name^*

Practice Street Address

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Number of APDS patients^*

Did you receive the link to this site from your Genetic Testing lab partner?^*

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If Yes:

Invitae Other

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If yes, please provide additional information on the genetic testing results:

Patient Gender

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Patient Current Age

Patient Age at Diagnosis

Patient Genetic Mutation Identified

PIK3CD PIK3R1 Other/Unknown

Patient Current Medications (check all that apply)

subcutaneous immunoglobulin (SCIG) intravenous immunoglobulin (IVIG) mTOR inhibitor (e.g. Rapamune/sirolimus) Immunosuppressants (e.g. steroids, rituximab, azathioprine, methotrexate, cyclosporin) Other None

Patient Current Medications: Other

I hereby give consent to Pharming Healthcare, Inc., its affiliates, and their agents and representatives, to send me future communications and information related to APDS, APDS products, and other information via the contact information I have provided above.

Please complete the following registration form to receive information about Activated Phosphoinositide 3-kinase Delta Syndrome (APDS)

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