CystinosisNCT00010426
Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
A study testing cysteamine hydrochloride in people with Cystinosis, run by FDA Office of Orphan Products Development.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 50 years
- Conditions the study is for
- Cystinosis
- What is being tested?
- cysteamine hydrochloride
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- Dec 1999to Feb 2001
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
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
About this study
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.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, multicenter study Patients are randomized to receive the current formulation of cysteamine hydrochloride as drops in one eye and the new formulation of cysteamine hydrochloride as drops in the other eye.
Patients receive the two formulations of cysteamine hydrochloride in their assigned eyes every hour during waking hours daily for 6 months (safety study) or for 1 year (efficacy study).
- Conditions
- Cystinosis
- Being tested
- cysteamine hydrochloride
- Keywords
- cystinosis, rare disease, renal and genitourinary disorders
- Medical subject headings
- Cystinosis, Rare Diseases, Urogenital Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cysteamine hydrochlorideDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2001 (estimated)
- Study startDec 1999
- Study completionFeb 2001
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CystinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Enrolment
- 30 participants
Sponsor and investigators
- Lead sponsor
- FDA Office of Orphan Products Development
- Sponsor type
- US federal agency
- Collaborators
- Leadiant Biosciences, Inc.
- Investigators
- Edward F. Lemanowicz, Study chair, Leadiant Biosciences, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00010426
- Sponsor's protocol ID
- 199/15704
- Other IDs
- SIGMATAU-FDR001769
- Status last verified
- Apr 2001
- First submitted
- 2 Feb 2001
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
- ClinicalTrials.gov ID
- NCT00010426
- Acronym / sponsor ID
- 199/15704
- Conditions
- Cystinosis
- Keywords
- cystinosis, rare disease, renal and genitourinary disorders
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants
- Lead sponsor
- FDA Office of Orphan Products DevelopmentUS federal agency
- Collaborators
- Leadiant Biosciences, Inc.
- Study start
- Dec 1999
- Study completion
- Feb 2001
- First posted
- 2 Feb 2001 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
PROTOCOL OUTLINE: This is a randomized, multicenter study Patients are randomized to receive the current formulation of cysteamine hydrochloride as drops in one eye and the new formulation of cysteamine hydrochloride as drops in the other eye.
Patients receive the two formulations of cysteamine hydrochloride in their assigned eyes every hour during waking hours daily for 6 months (safety study) or for 1 year (efficacy study).
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 1 year to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| cysteamine hydrochloride | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Edward F. Lemanowicz study chair
Leadiant Biosciences, Inc.
Questions about this trial
What is this clinical trial studying?
A study testing cysteamine hydrochloride in people with Cystinosis, run by FDA Office of Orphan Products Development.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2015.