2024-513119-29-00招募中2 期
A Phase 2, Randomized, Open-Label, 24-Week Study to Assess the Pharmacodynamics, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of GLM101 Administered Intravenously to Adult, Adolescent and Pediatric Participants with PMM2-CDG
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Change in ICARS
研究概览
简要总结
To characterize the changes from baseline in ataxia after 12 and 24 weeks of dosing with GLM101 in participants with PMM2-CDG
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Is male or female, 18 to 65 years of age, inclusive, at Screening (Cohorts 1-3, 7), 12-17 years of age, inclusive, at Screening (Cohort 4) or 2-11 years of age, inclusive, at Screening (Cohorts 5 and 6)
- •Molecularly confirmed diagnosis of PMM2-CDG. Diagnosis is defined as biallelic pathogenic and/or likely pathogenic variants, or, in the case of variants of uncertain pathogenicity, demonstration of bi-allelic variants AND phosphomannomutase-2 (PMM2) enzyme activity consistent with a diagnosis of PMM2-CDG. Historical diagnosis with lab report(s) on file is permitted
- •If the participant is a female of childbearing potential (i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile (permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy)) she must not be pregnant (confirmed by a negative serum pregnancy test), is using a medically accepted method of contraception (abstinence, a hormonal contraceptive associated twith inhibition of ovulation in conjunction with a barrier method, or use of an intrauterine device), and must agree to continue using this method for 50 days after the last infusion of GLM101
- •If the participant is a female of non-childbearing potential, she must be pre-pubertal, surgically sterile, or must have an ovarian dysfunction confirmed by a follicle stimulating hormone (FSH) >40 IU/L and absence of menses for 12 months without an alternative medical cause
- •If the participant is a sexually active male with female partners, the sexually mature, nonsterile male participant agrees to use a medically acceptable method of contraception (abstinence, the partner taking a hormonal contraceptive in conjunction with a male condom, or use by the partner of an intrauterine device with a male condom) and agrees to continue using this method for 50 days after the last infusion of GLM
- •Males are considered surgically sterile if they have undergone bilateral orchiectomy or vasectomy at least 3 months prior to Screening
- •If the participant is male, he must agree to refrain from donating sperm during the study and 60 days after the last infusion of GLM101
- •Is willing and able to provide informed consent/assent, directly or through his/her legally authorized representative
排除标准
- •Diagnosis of congenital disorder of glycosylation (CDG) other than PMM2; Diagnosis is defined as biallelic pathogenic and/or likely pathogenic variants, or, in the case of variants of uncertain pathogenicity, demonstration of bi-allelic variants AND the defined CDG enzyme activity consistent with a diagnosis of the CDG other than PMM2 CDG.
- •Has Screening or eligibility confirmation laboratory value(s) outside the laboratory reference range considered clinically significant and not related to PMM2-CDG
- •If female, has a positive serum pregnancy test during Screening
- •If female, must not be breastfeeding
- •Has serology positive for hepatitis B surface antigen or hepatitis C antibody during Screening;
- •Has history or presence, upon clinical evaluation, of any illness that might impact the safety of GLM101 infusion or evaluability of drug effect based on the Investigator’s and Medical Monitor’s discretion
- •Has a QTc ≥ 450 ms, or other clinically significant ECG abnormalities
- •Has uncontrolled cardiovascular, hepatic, pulmonary, gastro-intestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric or other significant disease
- •Is currently participating in another interventional clinical study or has completed another clinical study with an investigational drug or device within 30 days or 5 half-lives before GLM101 infusion
- •Weight exceeds 75 kg
- •Has an active infection requiring parenteral antibiotics, antivirals, or antifungals or treatment with systemic steroids within 7 days prior to Screening
- •Has confirmed active coronavirus disease-2019 (COVID-19) or tests positive for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at Screening or check in to the clinical site
- •ALT or AST >3× ULN OR total bilirubin >2× ULN or INR >1.5
- •Has a history of a severe allergic reaction to any drug or excipients of GLM101 (as listed in the GLM101 Investigator’s Brochure)
- •Has a known history of poor venous access
- •Has a history of liver transplant
- •Has a history of drug or alcohol use disorder within 12 months prior to Screening
- •Has had a major surgical procedure within 30 days prior to Screening
结局指标
主要结局
Change in ICARS
Change in ICARS
次要结局
- Evaluation of safety through the collection of safety parameters: AEs, AESIs (including Infusion-Associated Reactions), SAEs, deaths, and discontinuations due to AEs, clinical laboratory tests (hematology, chemistry, and urinalysis), ECG, vital signs, and PE findings
- Concentrations of total M1P to estimate PK parameters including Cmax, Clast, tmax, tlast, t1/2, AUC0-last, AUC0-∞, AUC0-tau, %AUCex, CL, Vz, Vss, and λz
研究者
Rose Marino
Scientific
Glycomine Inc.
研究点 (1)
Loading locations...
相似试验
已完成
1 期
Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic AtrophyGeographic AtrophyNCT03777332Apellis Pharmaceuticals, Inc.19
招募中
3 期
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman SyndromeAngelman Syndrome2024-510917-14-00Ultragenyx Pharmaceutical Inc.15
已完成
3 期
Relapse-prevention Study With Lu AA21004 (Vortioxetine) in Patients With Generalized Anxiety DisorderGeneralized Anxiety DisorderNCT00788034H. Lundbeck A/S459
已完成
1 期
A Phase 1 Study of AMG 208 in Subjects With Advanced Solid TumorsOncologyAdvanced MalignancyAdvanced Solid TumorsTumorsOncology PatientsCancerNCT00813384Amgen54
已完成
2 期
A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic AtrophyGeographic AtrophyNCT04656561Annexon, Inc.270