The purpose of this study is to determine the safety of ascending doses of HGT-1110 administered by intrathecal (IT) injection for 38 weeks (20 injections) in children with metachromatic leukodystrophy (MLD).
Who can participate
Age range12 Years
SexALL
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Inclusion criteria
✓. Confirmed diagnosis of metachromatic leukodystrophy by both:
✓. Appearance of the first symptoms of disease at or before 30 months of age.
✓. Ambulatory at the time of screening. The minimum level of function required to meet this criterion is defined as the ability to walk forward 10 steps with one hand held.
✓. The patient is less than 12 years of age at the time of screening.
✓.1 Minimum motor function requirements:
✓. A total GMFM-88 (percent) score ≥40 at the screening examination and a total GMFM-88 (percent) score ≥35 at the baseline examination, AND
✓. GMFM-88 Dimension E: Walking, Running \& Jumping, item 68 ("walk forward 10 steps with one hand held") score of at least 1 "initiates" at the screening and baseline examinations (if applicable).
✓.1 The patient is less than 8 years of age at the time of screening.
Exclusion criteria
✕. History of hematopoietic stem cell transplantation (HSCT).
✕. The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions.
✕. Any other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial.
What they're measuring
1
Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity
Timeframe: From start of study treatment up to Week 42
2
Number of Participants With Clinical Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)
Timeframe: From start of study treatment up to Week 40
3
Number of Participants With Vital Sign Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)
Timeframe: From start of study treatment up to Week 40
4
Number of Participants With Electrocardiogram (ECG) Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)
Timeframe: From start of study treatment up to Week 40
5
Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Treatment Emergent Adverse Events (TEAE)
Timeframe: From start of study treatment up to Week 40
6
Number of Participants With Cerebrospinal Fluid (CSF) Chemistry Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)
Timeframe: From start of study treatment up to Week 40
7
Number of Participants With Positive Anti-SHP611 Antibodies in Cerebrospinal Fluid (CSF) and or Serum
✕. The patient is enrolled in another clinical study that involves the use of any investigational product (drug or device) other than HGT-1110 or the IDDD used in this study within 30 days prior to study enrollment or at any time during the study.
✕. The patient is pregnant or breastfeeding.
✕. The patient has a condition that is contraindicated as described in the SOPH-A-PORT
✕. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device.
✕. 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.