Trial · NCT02437253

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

NCT02437253, Phase 1, Phase 2, Completed, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center; results first posted 2017-09-15

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ConductScience Library. Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI. Trials, record NCT02437253. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02437253

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
NCT number: NCT02437253
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 2 participants, actual
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Conditions: Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI
Interventions: Adalimumab, Placebo
First posted: 2015-05-07
Last updated: 2017-09-15
Results first posted: 2017-09-15
Limitations and caveats: Small pilot study of 2 participants.

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score	day 0 to week 16 of treatment with adalimumab versus placebo	Children's Health Questionnaire - Parent Form 50 bodily pain (BP) standardized score for participants \< 18 years of age. Range is from 0 to 100 (no units) and standardization if determined by comparison to healthy age matched children. Lower values mean increased pain. The difference of change in BP standardized score from day 0 to week 16 during adalimumab treatment minus change in BP standardized score from day 0 to week 16 during placebo treatment is reported.
Secondary	Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score	Day 0 to week 16 of treatment with adalimumab versus placebo	Children's Health Questionnaire - Parent Form 50 physical function (PF) standardized score for participants \< 18 years of age. Range is from 0 to 100 (no units) and standardization if determined by comparison to healthy age matched children. Lower values mean decreased physical function. The difference of change in PF standardized score from day 0 to week 16 during adalimumab treatment minus change in PF standardized score from day 0 to week 16 during placebo treatment is reported.
Secondary	Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)	Day 0 to week 16 of treatment with adalimumab versus placebo	Percent of participants with a \>10 mm improvement in either "how you feel now" or "worst pain you had this week" on the VA in the PPQ during adalimumab versus during placebo by either parental or subject report. Range is 0-100 mm; lower number means less pain.
Secondary	Range of Motion - Bilateral Shoulder, Elbow, Hip, Knee	Day 0 to week 16 of treatment with adalimumab versus placebo	Number of joints with a \>5 degree more positive change during 16 weeks of adalimumab versus 16 weeks of placebo. A total of eight joints were measured.
Secondary	Anti-ERT Antibodies	Day 0 to week 16 of treatment with adalimumab versus placebo	Anti-laronidase antibodies for subjects with MPS I. Anti-idursulfase antibodies for subjects with MPS II.

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score	day 0 to week 16 of treatment with adalimumab versus placebo	This was a cross-over study so within-individual changes are reported.	units on a scale	MEAN
SECONDARY	Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score	Day 0 to week 16 of treatment with adalimumab versus placebo		units on a scale	MEAN
SECONDARY	Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)	Day 0 to week 16 of treatment with adalimumab versus placebo		Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Range of Motion - Bilateral Shoulder, Elbow, Hip, Knee	Day 0 to week 16 of treatment with adalimumab versus placebo		joints	NUMBER
SECONDARY	Anti-ERT Antibodies	Day 0 to week 16 of treatment with adalimumab versus placebo	No participants had anti-ERT antibodies	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group10

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score	Adalimumab First, Then Placebo	1.4	Full Range 1.4 to 1.4	units on a scale
Children's Health Questionnaire - Parent Form 50 Bodily Pain Standardized Score	Placebo First, Then Adalimumab	1.0	Full Range 1.0 to 1.0	units on a scale
Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score	Adalimumab First, Then Placebo	0	Full Range 0 to 0	units on a scale
Children's Health Questionnaire - Parent Form 50 Physical Function (PF) Standardized Score	Placebo First, Then Adalimumab	0.4	Full Range 0.4 to 0.4	units on a scale
Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)	Adalimumab First, Then Placebo	0		Participants
Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)	Placebo First, Then Adalimumab	1		Participants
Range of Motion - Bilateral Shoulder, Elbow, Hip, Knee	Adalimumab First, Then Placebo	7		joints
Range of Motion - Bilateral Shoulder, Elbow, Hip, Knee	Placebo First, Then Adalimumab	5		joints
Anti-ERT Antibodies	Adalimumab First, Then Placebo	0		Participants
Anti-ERT Antibodies	Placebo First, Then Adalimumab	0		Participants

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Adalimumab First, Then Placebo	1
Overall Study	STARTED	Placebo First, Then Adalimumab	1
Overall Study	COMPLETED	Adalimumab First, Then Placebo	1
Overall Study	COMPLETED	Placebo First, Then Adalimumab	1
Overall Study	NOT COMPLETED	Adalimumab First, Then Placebo	0
Overall Study	NOT COMPLETED	Placebo First, Then Adalimumab	0

Source ClinicalTrials.gov

Adverse events14

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	Diarrhea	Gastrointestinal disorders	Adalimumab	1	2	1
other	Diarrhea	Gastrointestinal disorders	Placebo	0	2	0
other	Thrombocytopenia	Blood and lymphatic system disorders	Adalimumab	1	2	1
other	Thrombocytopenia	Blood and lymphatic system disorders	Placebo	0	2	0
other	Mood lability	Psychiatric disorders	Adalimumab	0	2	0
other	Mood lability	Psychiatric disorders	Placebo	1	2	1
other	Upper Respiratory Infection	Infections and infestations	Adalimumab	0	2	0
other	Upper Respiratory Infection	Infections and infestations	Placebo	2	2	2
other	Headache	Nervous system disorders	Adalimumab	0	2	0
other	Headache	Nervous system disorders	Placebo	1	2	1

1 to 10 of 14Source ClinicalTrials.gov

See all 14 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Adalimumab	0	2
Total, all-cause mortality	Placebo	0	2
Total, other adverse events	Adalimumab	2	2
Total, other adverse events	Placebo	2	2
Total, serious adverse events	Adalimumab	0	2
Total, serious adverse events	Placebo	0	2

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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 comparisons4

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Adalimumab First, Then Placebo vs Placebo First, Then Adalimumab: Anti-ERT Antibodies	Trial comparisons
Adalimumab First, Then Placebo vs Placebo First, Then Adalimumab: Pain Measured by the Visual Analog Scale (VAS) in the Pediatric Pain Questionnaire (PPQ)	Trial comparisons
Adalimumab vs Placebo: deaths from any cause	Trial comparisons
Adalimumab vs Placebo: serious adverse events	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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

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The record and each of its lists are JSON, twenty rows a page. No key.

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