CompletedPhase 1/2

Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)

Australia, Denmark, France24 participantsStarted 2012-02-02

Plain-language summary

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 range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity

Timeframe: From start of study treatment up to Week 42

Number of Participants With Clinical Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)

Timeframe: From start of study treatment up to Week 40

Number of Participants With Vital Sign Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)

Timeframe: From start of study treatment up to Week 40

Number of Participants With Electrocardiogram (ECG) Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)

Timeframe: From start of study treatment up to Week 40

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

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

Trial details

NCT IDNCT01510028

SponsorShire

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-01-20

Results submitted2018-01-19

View on ClinicalTrials.gov More Metachromatic Leukodystrophy (MLD) trials

Who can participate

Age range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity

Timeframe: From start of study treatment up to Week 42

Number of Participants With Clinical Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)

Timeframe: From start of study treatment up to Week 40

Number of Participants With Vital Sign Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)

Timeframe: From start of study treatment up to Week 40

Number of Participants With Electrocardiogram (ECG) Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)

Timeframe: From start of study treatment up to Week 40

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

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