Ended Therapeutic confirmatory (Phase III) WHIM Syndrome

A treatment study in patients with WHIM Syndrome.

EU CTIS ID: 2024-518461-10-00

What this study is testing

-Randomized Placebo-Controlled Period: To demonstrate the efficacy of mavorixafor in participants with Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome as assessed by increasing levels of circulating neutrophils compared with placebo and relative to a clinically meaningful threshold. -Open label period: To evaluate the long-term safety and tolerability of mavorixafor in participants with WHIM syndrome

  • Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • Inclusion criteria for Randomized Period: 1. Be at least 12 years of age. 2. Have signed the current approved Informed Consent Form. Participants under 18 years of age (in the Netherlands and other applicable regions, participants under 16 years of age) will sign an approved informed assent form and must also have a signed parental/legal guardian consent. 3. Have a genotype-confirmed mutation of CXCR4 consistent with WHIM phenotype. 4. Agree to use a highly effective form of contraception, as detailed in Section 5.3.1. 5. Be willing and able to comply with this protocol. 6. Have a confirmed ANC ≤ 400 cells/μL during screening, obtained while participant has no clinical evidence of infection.
  • Inclusion criteria for Open-Label Period: 1. Completed the Randomized Placebo-Controlled Period. 2. Granted Early Release from the Randomized Placebo-Controlled Period. 3. Blind broken.

You likely can't join if

  • Exclusion criteria for Randomized Period: 1. Has known systemic hypersensitivity to the mavorixafor drug substance, its inactive ingredients, or the placebo. 2. Is pregnant or breastfeeding. 3. Has a known history of a positive serology or viral load for HIV or a known history of AIDS. 4. Has, at screening, laboratory tests meeting 1 or more of the following criteria: − A positive hepatitis C virus antibody with confirmation by hepatitis C virus ribonucleic acid polymerase chain reaction reflex testing. − A positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb). − NOTE: If a participant tests negative for HBsAg, but positive for HBcAb, the participant would be considered eligible if the participant tests positive for hepatitis B surface antibody (also referred to as anti-HBsAg) on reflex testing. 5. Has, at screening, safety laboratory tests meeting 1 or more of the following criteria: − Hemoglobin < 8.0 g/dL − Platelets < 75,000 cells/μL − Estimated glomerular filtration rate based on the Modification of Diet in Renal Disease of ≤ 29 mL/min/1.73 m2 (Stage 4 or 5 chronic kidney disease) − Serum aspartate aminotransferase > 2.5 × ULN − Serum alanine aminotransferase > 2.5 × ULN − Total bilirubin > 1.5 × ULN (unless due to Gilbert’s syndrome, in which case total bilirubin ≥ 3.0 × ULN and direct bilirubin > 1.5 × ULN) 6. Had surgery requiring general anesthesia within the 4 weeks prior to Day 1. 7. Received any of the following treatments: − Plerixafor within 6 months prior to Day 1. − Chronic or prophylactic use of antibiotics (systemic or inhaled) within 4 weeks prior to Day 1. − Chronic or prophylactic use of G-CSF or granulocyte macrophage-colony stimulating factor within 2 weeks of Day 1. − Chronic or prophylactic use of systemic glucocorticoid use (> 5 mg prednisone equivalent per day) within 2 weeks prior to Day 1. − Any investigational therapy within 5 half-lives or 2 weeks prior to Day 1, whichever is longer. Prior use of any investigational therapies must be discussed with the Medical Monitor. 8. Is currently taking or has, within 2 weeks prior to Day 1, received any medication that is prohibited (see Section 6.4.1), based on potential for drug-drug interactions. 9. Has, at the planned initiation of study drug, a clinically diagnosed active infection (excluding warts) that has the potential to raise the ANC counts. 10. Has had a total splenectomy within 1 year. 11. Has a current diagnosis of myelofibrosis. 12. Has a medical history of hematological malignancies. 13. Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the participant or may preclude the participant’s successful completion of the clinical study. 14. Has corrected QT interval using Fridericia’s formula of > 450 ms.

The study team makes the final eligibility decision.

Where it's taking place

  • Russian Federation
  • United States
  • Australia
  • United Kingdom
  • Korea, Republic of

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Russian Federation; United States; Australia; United Kingdom; Korea, Republic of. Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.