CompletedNot Applicable

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

United States25 participantsStarted 2010-02-15

Plain-language summary

The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.

Who can participate

Age range1 Year

SexALL

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

✓.Patient is greater than or equal to 1 year of age and developmental age greater than or equal to 1 year.

Exclusion criteria

✕. Patient has significant non-MPS IIIA-related CNS impairment or behavioral disturbances, which would confound the scientific integrity or interpretation of study assessments, as determined by the investigator.
✕. Patients who, for MPS IIIA behavioral-related reasons,in the opinion of the investigator, would preclude performance of study neurocognitive and developmental testing procedures.
✕. Patients who are pregnant, breast feeding, or female patients of childbearing potential, who will not or cannot comply with the use of an acceptable method of birth control such as condoms, barrier method, oral contraception, etc.
✕. Patient is blind and/or deaf.
✕. 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.
✕. Patient or patient family history of neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns.
✕. The Investigator may choose to exclude patients who have had complications resulting from prior lumbar punctures.
✕. Patient history of poorly controlled seizure disorder.

What they're measuring

Change From Baseline in Bayley Scales of Infant Development-III/Kaufman Assessment Battery for Children-II (BSID-III/KABC-II) Age-Equivalent Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)

Change From Baseline in BSID-III/KABC-II Developmental Quotient (DQ) Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)

Change From Baseline in Vineland Adaptive Behavior Scales-II (VABS-II) Age-equivalent Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)

Change From Baseline in VABS-II Overall DQ Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)

Trial details

NCT IDNCT01047306

SponsorShire

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2013-07-10

Results submitted2015-02-09

View on ClinicalTrials.gov More Sanfilippo Syndrome Type A trials

Plain-language summary

Who can participate

Age range1 Year

SexALL

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

✓.Patient is greater than or equal to 1 year of age and developmental age greater than or equal to 1 year.

Exclusion criteria

✕. Patient has significant non-MPS IIIA-related CNS impairment or behavioral disturbances, which would confound the scientific integrity or interpretation of study assessments, as determined by the investigator.
✕. Patients who, for MPS IIIA behavioral-related reasons,in the opinion of the investigator, would preclude performance of study neurocognitive and developmental testing procedures.
✕. Patients who are pregnant, breast feeding, or female patients of childbearing potential, who will not or cannot comply with the use of an acceptable method of birth control such as condoms, barrier method, oral contraception, etc.
✕. Patient is blind and/or deaf.
✕. 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.
✕. Patient or patient family history of neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns.
✕. The Investigator may choose to exclude patients who have had complications resulting from prior lumbar punctures.
✕. Patient history of poorly controlled seizure disorder.

What they're measuring

Change From Baseline in Bayley Scales of Infant Development-III/Kaufman Assessment Battery for Children-II (BSID-III/KABC-II) Age-Equivalent Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)

Change From Baseline in BSID-III/KABC-II Developmental Quotient (DQ) Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)

Change From Baseline in Vineland Adaptive Behavior Scales-II (VABS-II) Age-equivalent Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)

Change From Baseline in VABS-II Overall DQ Scores

Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)