EUCTR2022-002301-24-DK招募中1 期
A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability ofNMD670 in ambulatory adults with Type 3 spinal muscular atrophy
MD Pharma A/S0 个研究点目标入组 54 人开始时间: 2023年1月24日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 54
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
- 性别
- All
入选标准
- •1. Participant must be 18 to 75 years of age inclusive, at the time of signing the informed consent.
- •2. Participants who are with a clinical diagnosis of Type 3 SMA.
- •3. Participants who are ambulatory, defined as being able to walk at least 50 metres without walking aids at screening during the 6-minute walk test.
- •4. Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1]).
- •5. Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
- •6. Participants with an MFM-32 dimension 1 (D1) score <80% at screening.
- •7. Participants with =7% CMAP amplitude decrement at screening [RNS].
- •8. Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
- •9. Participant is male or female.
- •10. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Male Participants:
- •A male participant must agree to use a highly effective contraception as detailed in Appendix 4 of this protocol during the intervention period and until the follow-up visit (corresponding to time needed to eliminate study intervention) and refrain from donating sperm during this period.
- •Female Participants:
- •A female participant is eligible to participate if she is not pregnant (see Appendix 4), not breastfeeding, and at least one of the following conditions applies:
- •o Not a woman of childbearing potential (WOCBP) as defined in Appendix 4.
- •o A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 during the intervention period and until the follow-up visit (corresponding to time needed to eliminate study intervention).
- •11. Participant is capable of giving signed informed consent as described in Appendix 1, Section 10.1.3 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- •Are the trial subjects under 18? no
- •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 49
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1. Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
- •2. Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
- •3. Participant with a clinical diagnosis of gout, or with serum uric acid > ULN at screening.
- •4. Participant with clinically significant electrocardiogram (ECG) abnormalities at screening including PR interval =220 msec, irregular rhythms (other than sinus arrhythmia or occasional, rare supraventricular or rare ventricular ectopic beats) in the judgement of the Investigator, or T-wave configurations are not of sufficient quality for assessing QT interval duration.
- •5. Participant with any of the following abnormalities in QT interval corrected for heart rate using Fridericia’s correction (QTcF) at screening:
- •a. QTcF >450 msec for male participants without bundle branch block.
- •b. QTcF >470 msec for female participants without bundle branch block.
- •c. QTcF >480 msec in participants with bundle branch block.
- •6. Participant with any of the following:
- •a. Abnormal liver function test defined as total bilirubin >1.5×ULN (participants with Gilbert's syndrome can be included with total bilirubin >1.5×ULN if direct bilirubin is =1.5×ULN and =35% of total bilirubin).
- •b. Current or chronic history of liver disease including (but not limited to) hepatitis virus infections, drug- or alcohol-related liver disease, non-alcoholic steatohepatitis, autoimmune hepatitis, hemochromatosis, Wilson's disease, a-1 antitrypsin deficiency, primary biliary cholangitis, primary sclerosing cholangitis, or any other liver disease
- •considered clinically significant by the Investigator.
- •c. Known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones).
- •7. Participant with clinically significant laboratory test abnormalities at screening.
- •8. Participant with breast cancer within the past 10 years or lymphoma, leukaemia, or any malignancy within the past 5 years. An exception of this 5-year requirement is basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease.
- •9. Participants with ongoing significant psychiatric disorder (e.g., uncontrolled depression, anxiety).
- •10. Participants with positive drug screen (cocaine, heroin, opiates, and ketamine) at screening. Participants will not be excluded from the study if they showed a positive drug screen due to medically prescribed, opiates or ketamine.
- •11. Participants with positive human immunodeficiency virus (HIV) antibody test.
- •12. Participants with presence of hepatitis B surface antigen (HBsAg) [or hepatitis B core antibody (HBcAb)] at screening or within 3 months prior to first dose of investigational intervention.
- •13. Participants with positive hepatitis C antibody or ribonucleic acid (RNA) test result at screening or within 3 months prior to Day 1.
- •NOTE: Participants with hepatitis C with positive hepatitis C antibody could be enrolled, if a negative hepatitis C RNA test is obtained.
- •14. Participants with a clinically significant history of allergic conditions (including drug allergies and anaphylactic reactions) or hypersensitivity to any component of the study intervention.
- •15. Participants with evidence of a lens opacity or cataract, or an inability to undergo the ophthalmologic evaluation, at screening.
- •16. Participants w
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