An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 13
- 主要终点
- Safety will be measured by the following endpoints: Reporting of treatment-emergent adverse events.
研究概览
简要总结
The primary objective of this study is to collect long-term safety data in patients with MLD who are receiving HGT-1110 and have participated in Study HGT-MLD-070 through Week 40.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Patient has participated in Study HGT-MLD-070 through Week
- •Patient must have no safety or medical issues that contraindicate participation.
- •The patient, patient's parent or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities.
排除标准
- •The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.
- •6d. The patient is at risk of abnormal bleeding due to a medical condition or therapy.
- •6e. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation.
- •6f. The patient has a functioning CSF shunt device.
- •6g. The patient has shown intolerance to an implanted device.
- •Undergoes bone marrow transplantation, hematopoietic stem cell transplantation, or gene therapy at any point during the study.
- •The patient has any known or suspected hypersensitivity to agents used for anesthesiaor is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions.
- •The patient is pregnant or breastfeeding.
- •The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or drug delivery device) other than those used in HGT-MLD-070 within 6 months prior to study enrollment or at any time during the study.
- •The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use, including:
- •6a. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device.
- •6b. The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the investigator.
- •6c. The patient has a known or suspected local or general infection.
结局指标
主要结局
Safety will be measured by the following endpoints: Reporting of treatment-emergent adverse events.
Safety will be measured by the following endpoints: Reporting of treatment-emergent adverse events.
Change from baseline in clinical laboratory testing (serum chemistry including liver function tests, hematology, and urinalysis).
Change from baseline in clinical laboratory testing (serum chemistry including liver function tests, hematology, and urinalysis).
Change from baseline in vital signs, physical examinations, and CSF chemistries (including cell counts, glucose, albumin, and protein); as of 15 March 2022, CSF albumin will not be collected.
Change from baseline in vital signs, physical examinations, and CSF chemistries (including cell counts, glucose, albumin, and protein); as of 15 March 2022, CSF albumin will not be collected.
Determination of the presence of anti-HGT-1110 antibodies in CSF and/or serum.
Determination of the presence of anti-HGT-1110 antibodies in CSF and/or serum.
次要结局
- 2. Change from baseline at end of study in the adaptive behavior composite standard score as measured by the VABS-II; as of 12 October 2021, VABS-II will not be collected
- 3. Change from baseline at end of study in the domain-specific Caregiver Observed MLD Functioning and Outcomes Reporting Tool (COMFORT) scores
- 4. Repeated-dose PK parameter estimates for HGT-1110 in serum
- 5. Concentrations of HGT-1110 in CSF at selected time points after repeated investigational drug product administration
- 1a. Change from baseline at end of study in motor function using the Gross Motor Function Measure-88 (GMFM-88) total score.
- 1b. The motor function assessments (GMFM-88, global impression of motor function-change [GIMF-C], and global impression of motor function-severity [GIMF-S]) will no longer be collected when patients reach Gross Motor Function Classification System (GMFCS) level 5
研究者
CJ Malanga
Scientific
Shire Human Genetic Therapies Inc.