EUCTR2015-002548-15-IT进行中（未招募）1 期
A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease. - VTS301
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 51
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
- •1. Male or female subjects, 4 to 21 years of age at time of screening with onset of neurological symptoms prior to 15 years of age.
- •2. Diagnosis of NPC1
- •3. Subject or parent/guardian must provide written informed consent to participate in the study. In addition to parental consent, assent to participate must also be sought from minor children.
- •4. Ability to undergo a LP and IT drug administration under monitored anesthesia care (conscious sedation) or if medically necessary, general anesthesia.
- •5. An NPC SS score of 0 through 4, inclusive, on the cognition component, and 1 through 4 in two or more of the following components: ambulation, fine motor skills, or swallowing.
- •6. Total NPC SS score of 10 or greater.
- •7. Subjects with adequately controlled seizures.
- •8. If taking miglustat, must have been on a stable dose for the past 6 to 8 weeks and be willing to remain on a stable dose for the duration of participation in Parts A and B of the study. Alternatively, subjects may elect to discontinue miglustat use and be eligible for trial entry after undergoing a minimum 6 week washout period prior to Study Day 0.
- •9. Agree to discontinue all nonprescription supplements.
- •10. Agree to discontinue any other investigational treatments for NPC including, for example, vorinostat or arimoclomol, acetylleucine, or curcumin at least 3 months prior to first dose (Study Day 0).
- •11. Females of childbearing potential (not surgically sterile) must use a medically acceptable method of contraception.
- •12. Subject or caregiver must possess the ability, per the Investigator, to understand and comply with protocol requirements including clinical outcome measurements and instructions for the entire duration of the study.
- •13. Caregiver, parent, guardian or responsible adult must be able and willing to accompany the subject to study visits.
- •Criteri inclusione Part C :
- •1. Subject has completed Part B, meets the criteria for dose reduction for a second time (see Protocol Section 9.2), or meets the criteria for the rescue option (see Protocol Section 9.4) OPPURE
- •2. Subject is a current participant in the NIH phase 1/2a open-label study and
- •a. Subject agrees to convert from the dose of VTS-270 currently receiving as a subject in the NIH phase 1/2a protocol to the dose chosen for Parts B and C of this study 900 mg;
- •b. Subject agrees to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 week dosing regimen.
- •c. In instances where NIH phase 1/2a subjects eligible to enroll into Part C are unable to convert from their current NIH phase 1/2a dose or monthly regimen, the investigator must receive prior written authorization from the sponsor for the subject to enter Part C of the study on an amended dose and/or regimen OPPURE
- •3. Subject has received prior written authorization from Vtesse to enroll directly into Part C.
- •4. Females of childbearing potential (not surgically sterile) must use a medically acceptable method of contraception.
- •5. Subject or parent/guardian must provide written informed consent to participate in the study. In addition to parental consent, assent to participate must also be sought from minor children.
- •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 16
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Exclusion criteria as assessed by NPC SS:
- •a. Unable to walk, wheelchair dependent (ambulation score=5)
- •b. Needs a nasogastric tube or gastric tube for all feedings (swallowing=5). Note: Nasogastric or gastric tube use
- •for supplemental feeding or medication administration is permitted and will not exclude a subject from this trial.
- •c. Severe dysmetria (fine motor score=5)
- •d. Minimal cognitive function (cognition score=5)
- •2. Body weight < 15 kg.
- •3. Prior treatment with IV HP-ß-CD for NPC1 disease, unless the subject has undergone a minimum 3-month washout period prior to Study Day 0. Note: Any prior IT administration of HP-ß-CD for the treatment of NPC1 disease will exclude a subject from enrollment.
- •Treatment of other medical conditions with drug preparations containing HP-ß-CD as an excipient (inactive ingredient) is acceptable and will not exclude a subject from immediate entry into this trial (no washout period required).
- •4. Status epilepticus occurring within 3 months of screening and/or seizure frequency that cannot be quantified.
- •5. Subjects on typical or atypical antipsychotics for treatment of psychosis. Note: Use of antipsychotic medication for treatment of other disorders (e.g., attention deficit hyperactivity disorder) will not exclude a subject from this trial.
- •6. History of hypersensitivity reactions to any product containing HP-ß-CD.
- •7. Prior treatment with any other investigational product within 3 months prior to first dose (Study Day 0).
- •8. Female subjects who are pregnant or nursing.
- •9. Subjects with suspected infection of the central nervous system (CNS) or any systemic infection.
- •10. Spinal deformity that could impact the ability to perform a LP.
- •11. Skin infection in the lumbar region within 2 months of study entry.
- •12. Neutropenia, defined as an absolute neutrophil count (ANC) of less than 1.5 x 109/L.
- •13. Thrombocytopenia (platelet count less than 75 x 109/L).
- •14. Activated partial thromboplastin time (aPTT) or prothrombin time (PT) prolonged by greater than 1.5 times the upper limit of normal (ULN) or known history of a bleeding disorder.
- •15. Evidence of obstructive hydrocephalus or normal pressure hydrocephalus.
- •16. Recent use of anticoagulants (in past 2 weeks prior to first dose [Study Day 0].
- •17. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) greater than 4 times the ULN.
- •18. Anemia: Hemoglobin more than 2 standard deviations below normal for age and gender.
- •19. Estimated glomerular filtration rate (eGFR) < 60 mL/minute/1.73m2 calculated using the modified Schwartz formula (2009) for subjects aged
- •4 through 17 years old or using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for subjects aged 18 years or older.
- •20. Active pulmonary disease, oxygen requirement, or clinically significant history of decreased blood oxygen saturation, pulmonary therapy, or requiring active suction.
- •21. Subjects who, in the opinion of the investigator, are unable to comply with the protocol or have medical conditions that would potentially increase the risk of participation.
- •22. Life expectancy less than one year.
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