EUCTR2019-001153-10-IT进行中（未招募）1 期
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension - A treatment study in patients with WHIM Syndrome.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 28
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Inclusion criteria for Randomized Period:
- •1. Be at least 12 years of age.
- •2. Have signed the current approved informed consent form. Patients under 18 years of age (in the Netherlands and other applicable regions, patients 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.
- •5. Be willing and able to comply with this protocol.
- •6. Have a confirmed ANC =400 cells/µL during screening, obtained while patient has no clinical evidence of infection. For ANC count >400 cells/µL due to an ongoing infection, patients may be allowed to rescreen after the infection is determined by the treating physician to be cleared. The Medical Monitor should be notified and approve each rescreen occurrence.
- •Inclusion criteria for Open-Label Period:
- •1. Completed the Randomized Period.
- •2. Granted Early Release from the Randomized Period.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 23
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •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 inadequately controlled diabetes (hemoglobin A1C >8.5%).
- •4. Has a known history of a positive serology or viral load for human immunodeficiency virus or a known history of acquired immune deficiency syndrome.
- •5. Has, at screening, laboratory tests meeting one or more of the
- •following criteria:
- •- A positive hepatitis C virus antibody (HCVAb) 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 patient tests negative for HBsAg, but positive for HBcAb, the
- •patient would be considered eligible if the patient tests positive for hepatitis B surface antibody (also referred to as anti-HBsAg) on reflex testing.
- •6. Has, at screening, safety laboratory tests meeting one 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 >2.5x the upper limit of normal (ULN)
- •- Serum alanine aminotransferase >2.5x ULN
- •- Total bilirubin >1.5x ULN (unless due to Gilbert's syndrome, in which case total bilirubin =3.0x ULN and direct bilirubin >1.5x ULN)
- •7. Had surgery requiring general anesthesia within the 4 weeks prior to
- •8. Received any of the following treatments:
- •- Plerixafor within 18 months prior to Day 1.
- •- Chronic or prophylactic use of antibiotics (systemic or inhaled) within 4 weeks prior to Day 1.
- •- G-CSF or granulocyte macrophage-colony stimulating factor within 2 weeks of Day 1.
- •- Ig (including intravenous or subcutaneous) within 5 months of Day 1, unless deemed necessary by the treating physician and after agreement from the Sponsor. Provision is made for a stratum for patients having received any Ig treatment within 5 months of Day 1.
- •- 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.
- •9. Is currently taking or has, within 2 weeks prior to Day 1, received any medication that is a
- •strong inhibitor or inducer of cytochrome P450 (CYP) 3A4 and/or Pglycoprotein. (P-gp) (ie, amiodarone, carvedilol, dronedarone, quercetin, quinidine, ranolazine, verapamil).
- •10. Has, at the planned initiation of study drug, a clinically diagnosed active infection (excluding
- •warts) that has the potential to raise the ANC counts.
- •11. Has had a total splenectomy witin 3 years.
- •12. Has a current diagnosis of myelofibrosis.
- •13. Has a medical history of hematological malignancies.
- •14. Has any other medical or personal condition, which in the opinion of the Investigator may potentially compromise the safety or compliance of the patient or may preclude the patient's successful completion of the clinical study.
- •15. Has Grade >1 QTc prolongation calculated per Fridericia's formula.
- •Exclusion criteria for Open-Label Period:
- •1.Patients who experience any treatment-limiting toxicity (TLT) during the first 4 weeks of treatment or any critical TLT at any time.
研究者
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