Trial · NCT01510028
Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
NCT01510028, Phase 1, Phase 2, Completed, Shire; results first posted 2018-10-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD). Trials, record NCT01510028. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01510028
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
- NCT number
- NCT01510028
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 24 participants, actual
- Lead sponsor
- Shire
- Conditions
- Metachromatic Leukodystrophy (MLD)
- Interventions
- Recombinant human arylsulfatase A
- First posted
- 2012-01-13
- Last updated
- 2021-06-14
- Other registries' IDs
- eudract:2011-002044-28
- Results first posted
- 2018-10-15
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | From start of study treatment up to Week 42 | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were defined as all AEs that occurred at or after the first dose of the investigational product or device implant surgery (whichever occurred earlier) and through the last follow-up date. Drug-related and device-related types of TEAEs were analyzed and reported. The severity of AEs was assessed by the investigator using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 4.0 grading scale. Severity of all AEs or SAEs was recorded as grade 1, 2, 3, 4, or 5 corresponding, respectively, to a severity of mild, moderate, severe, life-threatening, or fatal. Here SDI refers to surgical device implantation. |
| Primary | Number of Participants With Clinical Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | Clinical laboratory test included serum chemistry, hematology and urinalysis. Clinical laboratory abnormalities were recorded and reported as TEAE. An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were defined as all AEs that occurred at or after the first dose of the investigational product or device implant surgery (whichever occurred earlier) and through the last follow-up date. |
| Primary | Number of Participants With Vital Sign Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | Vital sign assessments included blood pressure, heart rate, respiratory rate and body temperature. Vital sign abnormalities were recorded and reported as TEAE. An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were defined as all AEs that occurred at or after the first dose of the investigational product or device implant surgery (whichever occurred earlier) and through the last follow-up date. Vital sign abnormalities included pyrexia which was considered as TEAE and was reported. |
| Primary | Number of Participants With Electrocardiogram (ECG) Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | 12-lead ECG was recorded and measured with the participant in rested supine position for at least 10 minutes. ECG abnormalities were recorded and reported as TEAE. An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were defined as all AEs that occurred at or after the first dose of the investigational product or device implant surgery (whichever occurred earlier) and through the last follow-up date. |
| Primary | Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Treatment Emergent Adverse Events (TEAE) | From start of study treatment up to Week 40 | Complete physical examination included evaluation of the port and catheter track. Height or length and weight were recorded and used to calculate growth. Body weight and height measurements were used to calculate the body mass index (BMI). Head circumference was measured in uniform manner for all participants. Clinical significance was defined as any variation in physical findings that had medical relevance resulting in an alteration in medical care. Clinically significant abnormalities related to physical examination were recorded and reported as TEAE. An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were defined as all AEs that occurred at or after the first dose of the investigational product or device implant surgery (whichever occurred earlier) and through the last follow-up date. |
| Primary | Number of Participants With Cerebrospinal Fluid (CSF) Chemistry Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | CSF chemistry assessments (including cell counts, glucose and protein) was measured. CSF chemistry abnormalities were recorded and reported as TEAE. An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were defined as all AEs that occurred at or after the first dose of the investigational product or device implant surgery (whichever occurred earlier) and through the last follow-up date. |
| Primary | Number of Participants With Positive Anti-SHP611 Antibodies in Cerebrospinal Fluid (CSF) and or Serum | Baseline up to Week 40 | Number of participants with positive anti-SHP611 antibody results in serum and in CSF were reported. A participant was considered positive if they had at least 1 positive result during the study. |
| Secondary | Change From Baseline in Motor Function Using Gross Motor Function Measure 88 (GMFM-88) Total Score at Week 40 | Baseline, Week 40 | The GMFM-88 was used to measure motor function. The GMFM-88 item scores were used to calculate domain-specific percent score for each of the 5 GMFM-88 dimensions (lying and rolling; sitting; crawling and kneeling; standing; walking, running, and jumping), and a total GMFM-88 (percent) score was calculated based on each dimension score. Each of the 88 items was rated on a 4-point scale: 0=does not initiate; 1=initiates; 2=partially completes; and 3=completes. The GMFM-88 total scores ranged from 0% (no mobility) to a score of 100%, that is (i.e,) the score that can be obtained by an average 5-year-old or older child with normal motor abilities. |
| Secondary | Number of Participants With Shift in Functional Endoscopic Evaluation of Swallowing (FEES) for Texture Utilized at Week 40 | Week 40 | The FEES assessment was performed to evaluate the structure and function of the upper throat during swallowing and for an assessment of aspiration risk. Each participant had this assessment performed at the clinical site using transnasal flexible laryngoscopy. FEES for texture utilized was evaluated. Data was presented only for the shifts observed. |
| Secondary | Number of Participants With Shift in Functional Endoscopic Evaluation of Swallowing for Feeding Assessment (Laryngeal Penetration) at Week 40 | Week 40 | The FEES assessment was performed to evaluate the structure and function of the upper throat during swallowing and for an assessment of aspiration risk. Each participant had this assessment performed at the clinical site using transnasal flexible laryngoscopy. Feeding assessment for laryngeal penetration was assessed. Data was presented only for the shifts observed. Here TL refers to thin liquids, THL refers to thickened liquids, PT refers to puree texture, WCC refers to with cough and clearance and WCNC refers to with cough and no clearance. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | From start of study treatment up to Week 42 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinical Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Vital Sign Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Electrocardiogram (ECG) Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Treatment Emergent Adverse Events (TEAE) | From start of study treatment up to Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Cerebrospinal Fluid (CSF) Chemistry Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) | From start of study treatment up to Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Positive Anti-SHP611 Antibodies in Cerebrospinal Fluid (CSF) and or Serum | Baseline up to Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Motor Function Using Gross Motor Function Measure 88 (GMFM-88) Total Score at Week 40 | Baseline, Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Score on a Scale | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Shift in Functional Endoscopic Evaluation of Swallowing (FEES) for Texture Utilized at Week 40 | Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Shift in Functional Endoscopic Evaluation of Swallowing for Feeding Assessment (Laryngeal Penetration) at Week 40 | Week 40 | Safety set consisted of participants who received at least 1 dose of investigational product or underwent device implant surgery. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group763
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 10 mg (Process A) | TEAE | 6 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 30 mg (Process A) | TEAE | 6 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 100 mg (Process A) | TEAE | 6 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 100 mg (Process B) | TEAE | 6 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 10 mg (Process A) | SHP611-related TEAE | 3 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 30 mg (Process A) | SHP611-related TEAE | 4 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 100 mg (Process A) | SHP611-related TEAE | 4 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 100 mg (Process B) | SHP611-related TEAE | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 10 mg (Process A) | SDI-related TEAE | 5 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Type and Severity | SHP611 30 mg (Process A) | SDI-related TEAE | 3 | Participants |
1 to 10 of 763Source ClinicalTrials.gov
See all 763 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | SHP611 10 mg (Process A) | 6 | |
| Overall Study | STARTED | SHP611 30 mg (Process A) | 6 | |
| Overall Study | STARTED | SHP611 100 mg (Process A) | 6 | |
| Overall Study | STARTED | SHP611 100 mg (Process B) | 6 | |
| Overall Study | COMPLETED | SHP611 10 mg (Process A) | 5 | |
| Overall Study | COMPLETED | SHP611 30 mg (Process A) | 6 | |
| Overall Study | COMPLETED | SHP611 100 mg (Process A) | 6 | |
| Overall Study | COMPLETED | SHP611 100 mg (Process B) | 6 | |
| Overall Study | NOT COMPLETED | SHP611 10 mg (Process A) | 1 | |
| Overall Study | NOT COMPLETED | SHP611 30 mg (Process A) | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events732
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Eosinophilia | Blood and lymphatic system disorders | SHP611 10 mg (Process A) | 0 | 6 | 0 |
| other | Eosinophilia | Blood and lymphatic system disorders | SHP611 30 mg (Process A) | 2 | 6 | 2 |
| other | Eosinophilia | Blood and lymphatic system disorders | SHP611 100 mg (Process A) | 0 | 6 | 0 |
| other | Eosinophilia | Blood and lymphatic system disorders | SHP611 100 mg (Process B) | 0 | 6 | 0 |
| other | Leukocytosis | Blood and lymphatic system disorders | SHP611 10 mg (Process A) | 0 | 6 | 0 |
| other | Leukocytosis | Blood and lymphatic system disorders | SHP611 30 mg (Process A) | 1 | 6 | 1 |
| other | Leukocytosis | Blood and lymphatic system disorders | SHP611 100 mg (Process A) | 0 | 6 | 0 |
| other | Leukocytosis | Blood and lymphatic system disorders | SHP611 100 mg (Process B) | 0 | 6 | 0 |
| other | Lymphopenia | Blood and lymphatic system disorders | SHP611 10 mg (Process A) | 0 | 6 | 0 |
| other | Lymphopenia | Blood and lymphatic system disorders | SHP611 30 mg (Process A) | 1 | 6 | 1 |
1 to 10 of 732Source ClinicalTrials.gov
See all 732 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | SHP611 10 mg (Process A) | 0 | 6 |
| Total, all-cause mortality | SHP611 30 mg (Process A) | 0 | 6 |
| Total, all-cause mortality | SHP611 100 mg (Process A) | 0 | 6 |
| Total, all-cause mortality | SHP611 100 mg (Process B) | 0 | 6 |
| Total, other adverse events | SHP611 10 mg (Process A) | 6 | 6 |
| Total, other adverse events | SHP611 30 mg (Process A) | 6 | 6 |
| Total, other adverse events | SHP611 100 mg (Process A) | 6 | 6 |
| Total, other adverse events | SHP611 100 mg (Process B) | 6 | 6 |
| Total, serious adverse events | SHP611 10 mg (Process A) | 5 | 6 |
| Total, serious adverse events | SHP611 30 mg (Process A) | 4 | 6 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 32792226 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01510028
GET https://conductscience.org/api/trials/v1/NCT01510028/registered?page=2