Clinical Trial Information

COVID-19 Patient Survey Results

In April 2020, many participated in surveys regarding how you felt participating in clinical trials during a world-wide health pandemic. Click on the links to see results.

Severe Congenital Neutropenia

WHIM Syndrome

Waldenstroms Macroglobulinemia

Thanks to Xr Pharmaceuticals and SavvyCoop for sharing the surveys and the results with us.

 

X4 Pharmaceutical’s (Cambridge, MA) Phase 3 WHIM trial is open now for enrollment, entitled “A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial of mavorixafor (X4P-001) in Patients with WHIM Syndrome with Open-Label Extension

WHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) is a rare autosomal dominant primary immunodeficiency disease caused by gain-of-function mutations in the chemokine receptor CXCR4.  CXCR4 mutations induce increased leukocyte homing and retention in the bone marrow resulting in severe chronic neutropenia and lymphopenia.

X4 Pharmaceuticals is planning to sponsor a 52-week global Phase 3 study to evaluate the safety and efficacy of mavorixafor, a once-daily oral CXCR4 antagonist, in genetically confirmed WHIM patients who are aged 12 years or older.  Mavorixafor is a first-in-class, oral, small molecule antagonist of the CXCR4 chemokine receptor that has demonstrated a favorable safety profile and dose-dependent increases in ANC and ALC in a Phase 2 study. Trial subjects will participate in a one-year trial with an opportunity to continue in the subsequent open label extension.  Travel support to study sites will be provided.

To support confirming the diagnosis of WHIM, X4 Pharmaceuticals will offer genetic testing at no charge to patients meeting eligibility criteria suggestive for the disease.  For information and support for genetic confirmation of your patients with suspected WHIM, please contact patientinfo@x4pharma.com

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Free genetic testing is available for people living with congenital, chronic, and/or idiopathic neutropenia to help them learn more about their condition.There are 407 genes known to cause primary immune deficiencies (PIDs), some of which may cause neutropenia. This genetic testing is only looking at the 407 genes known to cause PIDs and won’t tell you if  you’re at risk for other conditions.

You can take the test at your doctor’s office or at home. And there is no cost to you for this testing.
Click flyer for additional information and how to enroll.

 

 

 

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