NL-OMON56436尚未招募不适用
A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients with Adrenomyeloneuropathy - PROPE
SwanBio Therapeutics, Ltd.0 个研究点目标入组 5 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 5
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
- 年龄范围
- 18 至 64（—）
入选标准
- •1. Male adults aged >=18 and <= 65 years
- •2. Diagnosed with X-linked adrenoleukodystrophy (ALD)), including
- •proven mutation in the ABCD1 gene through confirmatory genetic
- •testing, and supported by historically elevated VLCFA.
- •3. Clinical evidence of spinal cord involvement with EDSS score between
- •1 and 4.5 and pyramidal function score of >=1 in the Functional System
- •Score of the EDSS. Signs of pyramidal tract dysfunction don't include
- •hyperreflexia.
- •4. Must agree to use reliable double-barrier contraception methods (e.g.,
- •XML File Identifier: kPpg2r7PH4BP7A3Dw3ckGhWrK7g=
- •condom and 1 other form of contraception for female sexual partners
- •that are of childbearing potential, such as diaphragm, intrauterine
- •device, spermicidal jelly, and/or hormonal contraceptive) and to not
- •donate sperm for at least 6 months following the IMP procedure.
- •5. For patients who are receiving any other treatment for ALD, including
- •off-label medications and/or supplements (e.g., antioxidants, Lorenzo's
- •oil, statins, etc.) or physical rehabilitation support, such treatments must
- •have been at a stable dose and/or frequency for >=4 weeks prior to
- •Screening and patients must agree to continue at the same dose and/or
- •frequency through Part 1 of the study.
- •6. The patient provided written informed consent prior to any study
- •procedures being performed
排除标准
- •1. Presence of inflammatory cerebral disease, established by
- •radiographic review of brain MRI demonstrating a Loes score >=0.5 on
- •the 34-point scale, except for the abnormalities that can be observed in
- •patients with AMN without inflammatory cerebral demyelination with
- •Loes score <=4.
- •2. Pathological changes identified on brain MRI including all lesions from
- •previous diagnosis of inflammatory cerebral disease with Loes score >=0.5 on the
- •34-point scale, except for the abnormalities that can be observed in patients
- •with AMN without inflammatory cerebral demyelination with Loes score <=4
- •3. 15 years or more have elapsed since the initial onset of
- •myeloneuropathy manifestations, such as walking or running difficulties,
- •bladder dysfunction, increased muscular tone, spasticity, weakness,
- •balance problems, etc.
- •4. Contraindications for MRI procedure and/or contrast materials.
- •5. History of a brain or spinal cord disease that would interfere with
- •lumbar puncture procedures, CSF circulation, and/or safety
- •assessments.
- •6. Contraindication to steroids, sirolimus, tacrolimus, and/or anesthetic
- •medications.
- •7. Contraindication to SBT101 and/or any of its ingredients.
- •8. Unstable adrenal function (e.g., untreated or inappropriately treated
- •adrenal insufficiency).
- •a. Adrenal function will be evaluated through laboratory assessments of
- •serum cortisol, plasma adrenocorticotropic hormone (ACTH), plasma
- •renin, aldosterone, sodium, and potassium levels at Screening.
- •Monitoring of replacement therapy will be mainly clinical. Determination
- •of inappropriately treated adrenal insufficiency will be made by the
- •Investigator in consultation with the Medical Monitor.
- •b. Patients who meet the following criteria will be considered to have
- •adrenal insufficiency: (i) basal cortisol concentration <275 nmol (10
- •µg/dL) in the morning (i.e., 6 AM to 10 AM) and (ii) plasma ACTH >= 2 ×
- •upper limit of normal (ULN).
- •9. Positive for human immunodeficiency virus (HIV) type 1 or 2 (HIV-1,
- •HIV-2), hepatitis B virus (HBV), or hepatitis C virus (HCV).
- •a. Patients who have been vaccinated against HBV (e.g., HBV surface
- •antibody-positive) who are negative for other markers of prior HBV
- •infection (e.g., HBV core antibody-negative) are eligible.
- •b. Patients who are positive for anti-HCV antibodies are eligible, as long
- •as they have a negative HCV load as measured by quantitative
- •polymerase chain reaction (qPCR).
- •10. Presence of a clinically significant active bacterial, viral, fungal,
- •parasitic, or prion-associated infection.
- •11. History of diabetes or abnormal fasting plasma glucose (>=126
- •mg/dL) or hemoglobin A1C >=6.5%.
- •12. Patients who have received a gene therapy.
- •13. Current use of medications that could potentially lead to changes in
- •intracranial pressure (e.g., levothyroxine, vitamin A supplementation,
- •oral contraceptives, tetracycline, acetazolamide [Diamox]).
- •14. Patients who are currently using strong CYP3A4 inducers or strong
- •CYP3A4 inhibitors, and are unwilling or unable to stop prior to and
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研究者
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