EUCTR2018-002969-19-GB进行中（未招募）1 期
An open-label study to evaluate the safety and tolerability of 12 weeks treatment with oral REN001 in patients with primary mitochondrial myopathy (PMM), with an optional extension of treatment - A safety study of REN001 in mitochondrial myopathy
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
- 性别
- All
入选标准
- •Subjects must meet all of the following inclusion criteria to be eligible for inclusion in the study:
- •1. Males or females aged 16 years or older.
- •2. Established diagnosis of PMM according to the 2016 Rome Consensus recommendations (Mancuso, McFarland, Klopstock, & Hirano, 2017).
- •3. A score of 2-4 on Question 5 Section III of NMDAS from subject's medical history or at screening whichever is most relevant.
- •4. Either a confirmed m.3243A>G mutation or other mtDNA defects, with myopathy.
- •5. Have no changes to their exercise regimen within 30 days prior to Day 1 and be willing to remain on the same exercise regimen for the duration of the study.
- •6. Subjects must be ambulatory and able to perform the study exercise tests independently.
- •7.Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes in medication for diabetes.
- •8. Have adequate kidney function defined as an estimated glomerular filtration rate (eGFR) = 60 mL/min/1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation (Levey, et al., 2009) at Screening and Baseline.
- •9. Be able to swallow capsules.
- •10. Females should be either of non-child-bearing potential or must agree to use a highly effective method of contraception from Screening. Males with partners who are WOCBP must also use contraception from Baseline.
- •11. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 3
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 21
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects presenting with any of the following will not be included in the study:
- •1. Participation in a prior REN001 (previously known as HPP-593) study.
- •2. Employee of the study site or of the Sponsor’s company or delegates.
- •3. Documented evidence of ongoing rhabdomyolysis.
- •4. Currently taking or anticipated to need a PPAR agonist during the study.
- •5. Subjects with motor abnormalities other than related to the mitochondrial disease that may interfere with the outcome measures.
- •6. Treatment with an investigational drug within 3 months prior to Day 1.
- •7. Currently taking or anticipated to need a prescription and/or non-prescription drug that might interfere with the study endpoints.
- •8. Evidence of significant concomitant clinical disease that may need a change in management during the study or could interfere with the conduct or safety of this study (Stable well-controlled chronic conditions such hypercholesterolemia, gastroesophageal reflux, or depression under control with medication (other than tricyclic antidepressants), are acceptable provided the symptoms and medications would not be predicted to compromise safety or interfere with the tests and interpretations of this study).
- •9. Subjects with diabetes who require treatment with insulin who are unwilling to undertake glucose monitoring for at least the first 12 weeks of dosing or in the Investigators opinion will not be able to adequately maintain glucose homeostasis.
- •10. Subjects with a history of cancer. A history of in situ basal cell carcinoma in the skin is allowed.
- •11. Have been hospitalised within the 3 months prior to Screening for any major medical condition (as deemed by the Principal Investigator).
- •12. Clinically significant cardiac disease or ECG abnormalities that in the opinion of the Principal Investigator should exclude the subject from enrolment into the study.
- •13. Any condition possibly reducing drug absorption (e.g., gastrectomy or serious GI dysmotility).
- •14. Subjects who have poor nutritional status as determined by the Investigator.
- •15. History of clinically significant liver disease and/or liver function tests (AST, ALT, total Bilirubin) >1.5 x ULN. Subjects with LFTs >1.5 x ULN and <2 x ULN at Screening may be retested once. Subjects with an isolated elevated bilirubin (e.g.< 2 x ULN) may be included after discussion with the Medical Monitor if the cause is due to a benign hereditary disorder of metabolism such as Gilbert’s syndrome.
- •16. Evidence of clinically significant muscle damage tests (CPK > 3 x ULN). Subjects with CPK > 3 x ULN and < 5 x ULN at Screening may be retested once.
- •17. Subjects with positive hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb) or hepatitis C or human immunodeficiency virus (HIV) at Screening.
- •18. Pregnant or nursing females.
- •19. History of sensitivity to PPAR agonists.
- •20. Subjects who are taking anticoagulants that would preclude muscle biopsy.
- •21. Donation or intent to donate blood, or blood components during the study or within one month after completion of the study.
- •22. Subjects with a history of drug abuse or with a positive urine drug screen at Screening. Positive results for opiates and/or benzodiazepines will only be allowed if supported by a corresponding prescription from a medically qualified person.
- •23. History of regular alcohol consumption exceeding 14 units/week (1 unit = 75 mL of wine or 250 mL of beer or 25 mL of spirits) within 6 months of Screening.
- •24. Inabilit
研究者
相似试验
已完成
不适用
Safety of turmeric-boswellia product in healthy peopleCTRI/2022/02/040107Arjuna Natural Private Limited
已完成
不适用
To study the safety of Ashwagandha extract in healthy peopleCTRI/2022/08/044636Arjuna Natural Private Limited62
已完成
1 期
Admiral: a study of the safety of multiple increasing doses of STK-001 in children and adolescents with Dravet syndromeDravet syndromeNervous System DiseasesEpilepsyISRCTN99651026Stoke Therapeutics, Inc19
招募中
3 期
A safety and efficacy study of GWP42003-P oral solution as adjunctive treatment for Japanese children and adults with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complexennox-Gastaut syndrome, Dravet syndrome, tuberous sclerosis complexJPRN-jRCT2031220041Yamamoto Tomoyuki84
进行中（未招募）
1 期
An open-label study to evaluate the safety and efficacy of IMATINIB with chemotherapy in pediatric patients with Ph+/BCR-ABL+ acute lymphoblastic leukemia (Ph+ALL)Children and adolescents aged 1-17 years at diagnosis, with Ph+ALLMedDRA version: 14.1Level: LLTClassification code 10000844Term: Acute lymphoblastic leukaemiaSystem Organ Class: 100000004864EUCTR2004-001647-30-ITA.I.E.O.P. - ASSOCIAZIONE ITALIANA EMATOLOGIA ONCOLOGIA PEDIATRICA550