NCT00010426

Brief Summary

OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation. II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Dec 1999

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 1999

Completed
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2001

Completed
1 day until next milestone

First Submitted

Initial submission to the registry

February 2, 2001

Completed
Same day until next milestone

First Posted

Study publicly available on registry

February 2, 2001

Completed
Last Updated

March 25, 2015

Status Verified

April 1, 2001

First QC Date

February 2, 2001

Last Update Submit

March 24, 2015

Conditions

Cystinosis

Keywords

cystinosisrare diseaserenal and genitourinary disorders

Interventions

cysteamine hydrochlorideDRUG

Eligibility Criteria

Age1 Year - 50 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Diagnosis of cystinosis with more than 2 nmole half-cystine/mg protein in leukocytes OR presence of corneal crystals consistent with cystinosis and distributed in corneal stroma observed by slit lamp biomicroscopy * Clinical history consistent with cystinosis * Safety study: History of adherence with current eye drop and follow-up schedule on protocol #86-El-0062 Any crystal density score, including zero, on photographs, that has been stable or improved over the past year * Efficacy study: Crystal density score at least 1.00 on photographs Concurrently on cysteamine for at least the past 6 months --Prior/Concurrent Therapy-- * No prior cysteamine drops (efficacy study) --Patient Characteristics-- * Age: 1 to 50 (safety study) 2 to 12 (efficacy study) * Other: Willingness and ability to tolerate corneal photographs

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

MeSH Terms

Conditions

CystinosisRare DiseasesUrogenital Diseases

Interventions

Cysteamine

Condition Hierarchy (Ancestors)

Lysosomal Storage DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

MercaptoethylaminesEthylaminesAminesOrganic ChemicalsSulfhydryl CompoundsSulfur Compounds

Study Officials

  • Edward F. Lemanowicz

    Leadiant Biosciences, Inc.

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
not applicable
Purpose
TREATMENT
Sponsor Type
FED

Study Record Dates

First Submitted

February 2, 2001

First Posted

February 2, 2001

Study Start

December 1, 1999

Study Completion

February 1, 2001

Last Updated

March 25, 2015

Record last verified: 2001-04