2023-506795-27-00招募中3 期
Effect of dimethyl fumarate administered to patients with adrenomyeloneuropathy: a multicenter, placebo controlled, phase IIb/III trial
Fundacio Institut D Investigacio Biomedica De Bellvitge IDIBELL3 个研究点 分布在 1 个国家目标入组 40 人开始时间: 2024年1月31日最近更新:
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 3
- 主要终点
- The primary endpoint is the mean change in 2 Minute Walk Test (2MWT) between M0 and M24. This criterion will also be evaluated at M6, M12 and at the end of the extension phase (M36). Details about the 2MWT are given in chapter 3.7.
研究概览
简要总结
The primary objective of the trial is to demonstrate the superiority of DMF at 480 mg/day over placebo in the clinical improvement of patients with AMN.
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Men and women of 18 to 65 years old at the time of the inclusion, suffering from AMN with: - elevated plasma VLCFA - ABCD1 gene mutation identified
- •Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk (EDSS score ≥ 2.0 and ≤ 6.5). EDSS score will also be re-evaluated at M12, M24 and M
- •Normal brain MRI or brain MRI showing: - abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4 - and/or stable (≥ 6 months) cerebral demyelination without gadolinium enhancement with a Loes score ≤ 12
- •Appropriate steroid replacement if adrenal insufficiency is present
- •Potential childbearing women should use an adequate method of contraception to avoid pregnancy throughout the study to minimize the risk of pregnancy. If oral contraceptives are used, the use of an alternative barrier method is recommended.
- •Likely to be able to participate in all scheduled evaluations and complete all required study procedures
- •Signed and dated written informed consent to participate in the study in accordance with local regulations
排除标准
- •Any progressive neurological disease other than AMN
- •Not easily contactable by the investigator in case of emergency or not able to call the investigator
- •Leukopenia below 3.0x109 /L, lymphopenia below 0.5x109 /L or other pathological results in the complete blood count
- •Suspected or confirmed progressive multifocal leukoencephalopathy (PML)
- •Severe gastrointestinal disease
- •Uncontrolled hepatic, renal or cardiovascular disease, or any evolutive malignancy
- •Pregnancy and breast-feeding in woman and potential childbearing woman unable or unwilling to use an acceptable contraceptive method during the study
- •Any new medication for AMN initiated less than three months prior to inclusion
- •Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body as aneurysm clips, pacemakers, intraocular metal or cochlear implants
- •Inclusion in another therapeutic clinical trial for ALD
结局指标
主要结局
The primary endpoint is the mean change in 2 Minute Walk Test (2MWT) between M0 and M24. This criterion will also be evaluated at M6, M12 and at the end of the extension phase (M36). Details about the 2MWT are given in chapter 3.7.
The primary endpoint is the mean change in 2 Minute Walk Test (2MWT) between M0 and M24. This criterion will also be evaluated at M6, M12 and at the end of the extension phase (M36). Details about the 2MWT are given in chapter 3.7.
次要结局
- Secondary efficacy endpoints will be evaluated by the 6 Minute Walk Test (6MWT), the Time to walk 25 feet (TW25), the Postural Sway, the stair-climbing test, the strength, one questionnaire about urinary tract function (SF-Qualiveen), one questionnaire to assess fecal incontinence (RFIS), neuroimaging and biological markers. Details are given in chapter 3.7.
- Mean changes in 6MWT, TW25 between M0 and M24. Evaluation will also be done at M6, M12 and M36.
- Mean changes in postural sway, stair-climbing test, and strength between M-1 and M24. Evaluation will also be done at M12 and M36.
- Mean changes in the SF-Qualiveen and RFIS between M0 and M24. Evaluation will also be done at M12 and M36.
- Mean changes in Loes score, diffusion tensor imaging (DTI), and magnetic resonance spectroscopy (MRS) parameters in the cerebral white matter on M-1 and M24. Evaluation will also be done at M12 and M36.
- Mean changes in markers of target-engagement (oxidative damage and inflammation) between M-1 and M24. Evaluation will also be done at M3 and M12.
- Markers of neuroaxonal damage and astrocyte activation: • Mean changes in NfL and GFAP in plasma at M-1, M3, M12, and M24, and in CSF at M0 and M24.
- Very long-chain fatty acid (VLCFA) levels • Mean changes in C26:0 and C24:0 in plasma and CSF at M-1, and M24.
- Single cell RNA sequencing (scRNAseq) of PBMC • Comparison of scRNAseq data from n=5 AMN patients (group 1: placebo), and n=10 from AMN (group 2: DMF), plus 10 samples from of age and sex-matched control adults at M-1 and M3.
- Lipidomics • Analysis of glycerolipids, glycerophospholipids, and sphingolipids at M-1, M3, M12 and M24 in plasma.
- Metabolomics • Analysis of metabolomics in stools at M-1, and M6.
- Metagenomics • Identification in stools of the different taxa, and enzymes affected by the treatment with DMF at M-1 and M6.
研究者
Montserrat Ruiz Sales
Scientific
Fundacio Institut D Investigacio Biomedica De Bellvitge IDIBELL
研究点 (3)
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