EUCTR2017-002158-35-FR进行中（未招募）1 期
A phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI. - iMProveS
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 24
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
入选标准
- •1. Male or female gender.
- •2. Age =16 years.
- •3. Diagnosis of MPS VI.
- •4. Urine GAG above upper limit of normal (ULN) at screening visit.
- •5. Willing and able to provide written, dated, signed informed consent, or in the case of subjects age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures or study assessment.
- •6. Able to comply with all study procedures.
- •7. Women with childbearing potential must agree to use a highly effective method of birth control during the study and at least 10 weeks after last administration. Highly effective forms of birth control include oral contraceptives (which must be stable for at least 1 full month prior to screening visit (V-1) and should remain stable during the study) associated with double-barrier methods or single-barrier methods of diaphragm with adjunct spermacide or condom with adjunct spermicide.
- •Inclusion criteria for Enzyme Replacement Therapy (ERT) receiving group
- •1. Patients with MPS Type VI receiving enzyme replacement therapy (Naglazyme) for at least 6 months on the licensed dosage or as per local guidelines.
- •Inclusion criteria for not ERT receiving group:
- •Patients with MPS Type VI not receiving enzyme replacement therapy for the following reasons:
- •1. Patients previously treated with ERT but have discontinued for more than 3 months either due to medical decision or personal choice
- •2. Patients allergic to ERT therapy
- •3. Patients that have had a previous hematopoietic stem cell transplant (HSCT)
- •4. Patients not treated with ERT i.e. treatment naïve
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 14
- •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 the entire cohort
- •1. Use of any investigational product or investigational medical device within 30 days prior to screening. This will include product bought over the counter specifically compounds like genistein and pentosane polysulphate which may not be considered as investigational products by patients and some health care professionals.
- •2. Concurrent disease or condition that would interfere with study participation or pose a safety concern for example patient with: severe cardiac insufficiency as define NYHA class > II, and severe restrictive chronic respiratory insufficiency as reflected by serum [HCO3-] =28 mEq/L.
- •3. Subjects who had surgery within 3 months before study starts, or for whom surgery is planned during study period.
- •4. Patient with spinal cord compression requiring surgical intervention.
- •5. Subjects with the following liver test abnomalies: any ALT, AST > 3 x ULN or bilirubin >1.5 x ULN (except if Gilbert syndrome) at screening visit.
- •6. Evidence of an immunosuppressive state, including known HIV infection, agammaglubilinemias, T-Cell deficiencies.
- •7. Subjects with history of chronic infections, including but not limited to subjects with history of viral hepatitis C, or B, with recent history of serious or life-threatening infection or any current signs or symptoms that may indicate infection at visit V-1 of study as per investigators clinical judgement.
- •8. History of malignant cancer except of cervical carcinoma in situ, basal cell carcinoma, dermatological squamous cell carcinoma.
- •9. Subjects with significant haematologic abnormalities, such as hemoglobin <8 g/dL, or WBC<2000 /mm3 or absolute neutrophil count <1300 /mm3, or platelet <30.000 /mm3.
- •10. International Normalized Ratio (INR), activated partial thromboplastin time (aPTT) or thrombin time (TT) values above the laboratory reference range at screening. For patients on anti-coagulants, they should be within their target effect on INR and be stable.
- •11. Any history of bleeding diathesis.
- •12. Patient with coexistence of corneal pathologies other than corneal clouding (e.g. exposure keratopathy)
- •13. An unwillingness on the part of male patients to abstain from sexual intercourse with pregnant or lactating women; or an unwillingness to use highly effective form of birth control if engaging in sexual intercourse with a woman who could become pregnant from the time of the first dose of study medication until completion of follow-up procedures.
- •14. An unwillingness on the part of female patients to use highly effective form of birth control if engaging in sexual intercourse and to have a monthly pregnancy test during treatment and until completion of follow-up procedures.
- •15. Pregnant or lactating women.
- •16. Have a known hypersensitivity to any of the ingredients or excipients of the IMP including: Microcrystalline Cellulose, Povidone, Sodium starch glycolate (type A), Magnesium stearate, Opadry™ II 85F18422
- •Exclusion criteria for ERT treated group:
- •1. Previous hematopoietic stem cell transplant (HSCT)
研究者
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