Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00048620
Other study ID # ASB-00-01
Secondary ID
Status Completed
Phase Phase 1
First received November 4, 2002
Last updated November 2, 2006
Start date September 2000
Est. completion date November 2005

Study information

Verified date November 2006
Source BioMarin Pharmaceutical
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial Summary

The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.


Recruitment information / eligibility

Status Completed
Enrollment 0
Est. completion date November 2005
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group N/A and older
Eligibility Inclusion Criteria:

- Patient consent

- Patient must be five years of age or older

- Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI

- Leukocyte ASB enzyme activity level less than 20% of the normal range

- Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.

- Ability to perform all protocol tests

- Ability to stand independently for six minutes

- Sexually active subjects must agree to use an adequate form of contraception

Exclusion Criteria:

- History of bone marrow transplantation

- Pregnant or lactating patient

- Use of an investigational drug or device within 30 days prior to study participation.

- A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up

- Known hypersensitivity to rhASB or to components of the study drug

- History of cancer (except low grade and fully resolved skin malignancy)

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
N-acetylgalactosamine 4-sulfatase


Locations

Country Name City State
United States BioMarin Pharmaceutical Inc. Novato California

Sponsors (1)

Lead Sponsor Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Lysosomal storage disease
See also
  Status Clinical Trial Phase
Terminated NCT01675674 - Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics N/A
Completed NCT00067470 - Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI Phase 3
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT01707433 - Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease N/A
Active, not recruiting NCT03632213 - Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI Phase 2
Terminated NCT00748969 - Clinical Trial of Growth Hormone in MPS I, II, and VI Phase 2/Phase 3
Recruiting NCT05619900 - Registry of Patients Diagnosed With Lysosomal Storage Diseases
Completed NCT00299000 - A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI Phase 4
Completed NCT00176917 - Stem Cell Transplantation for Hurler Phase 2
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed NCT00048711 - Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI Phase 2
Active, not recruiting NCT03153319 - Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI Phase 1/Phase 2
Completed NCT00104234 - Study of rhASB in Patients With Mucopolysaccharidosis VI Phase 3
Completed NCT03370653 - A Study in MPS VI to Assess Safety and Efficacy of Odiparcil Phase 2
Recruiting NCT06036693 - MPS (RaDiCo Cohort) (RaDiCo-MPS)
Active, not recruiting NCT00005900 - Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation N/A
Completed NCT01586455 - Human Placental-Derived Stem Cell Transplantation Phase 1

External Links