NL-OMON54582招募中3 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in patients with WHIM Syndrome with Open-Label Extension - Mavorixafor (X4P-001) in patients with WHIM Syndrome
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 12 至 64（—）
入选标准
- •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. Local laboratory may be used
- •if central laboratory is not available.
- •a. If the ANC is below the lower limit of detection for the laboratory and the
- •total WBC count is <= 400 cells/µL, then the participant is considered eligible
- •for the study.
- •b. If the ANC is > 400 cells/µL in the context of a recent infection or
- •inflammation prior to screening, it is acceptable to redraw a blood sample and
- •confirm that the ANC meets inclusion criteria (<= 400 cells/µL) once the
- •infection or inflammatory episode is resolved.
- •c. If the participant experiences an infection or inflammatory episode between
- •screening and baseline that may impact the ANC, or receives G-CSF between
- •screening and baseline, the baseline visit may be postponed for up to 4 weeks
- •until the ANC has been confirmed to be <= 400 cells/µL.
排除标准
- •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
- •* 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
- •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
- •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 × the upper limit of normal (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.
- •Note: Central results should be used for determination of screening laboratory
- •values when possible. However, local laboratory analysis may be used if central
- •laboratories are not available, or for unscheduled visits, repeated samples, or
- 另有 4 项未显示
研究者
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