Trial Outcomes & Findings for Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis (NCT NCT00001213)

Last Updated: 2014-07-22

Results Overview

Since efficacy of ophthalmic cysteamine was established and a New Drug Application (NDA) filed, the post-hoc primary outcome measure is the evaluation of safety information. There was no specified time frame for this outcome measure, as safety data was being collected until the drug became available for commercial purchase in May 2013.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

328 participants

Primary outcome timeframe

Any Time Point up to 27 Years

Results posted on

2014-07-22

Participant Flow

Participant milestones

Participant milestones
Measure	Cysteamine Topical Solution Cysteamine topical solution administered hourly while awake in both eyes Cysteamine
Overall Study STARTED	328
Overall Study COMPLETED	310
Overall Study NOT COMPLETED	18

Reasons for withdrawal

Reasons for withdrawal
Measure	Cysteamine Topical Solution Cysteamine topical solution administered hourly while awake in both eyes Cysteamine
Overall Study Death	18

Baseline Characteristics

Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cysteamine Topical Solution n=328 Participants Cysteamine topical solution administered hourly while awake in both eyes Cysteamine
Age, Continuous	13.1 years STANDARD_DEVIATION 10.04 • n=5 Participants
Gender Female	153 participants n=5 Participants
Gender Male	173 participants n=5 Participants

PRIMARY outcome

Timeframe: Any Time Point up to 27 Years

Outcome measures

Outcome measures
Measure	Cysteamine Topical Solution n=328 Participants Cysteamine topical solution administered hourly while awake in both eyes Cysteamine
Number of Participants With Serious and Non-Serious Adverse Events	257 participants

PRIMARY outcome

Timeframe: Any Time Point Up to 19 Years

Population: One hundred sixty-one (161) participants were analyzed in the pre-specified intent-to-treat population \[defined as patients who received study medication (between 1986 and 2005), and had a baseline and a post-baseline CCCS value\]. After 2005, all participants enrolled received open-label treatment and only safety data was obtained.

Response is defined as a decrease from baseline of at least 1 in Corneal Cystine Crystal Score (CCCS) at any time on study when baseline CCCS is greater than or equal to 1, or CCCS does not increase at least 1 at any time on study when baseline CCCS is less than 1. The CCCS is based on a library of slit-lamp photographs of corneas with increasing crystal densities (0-3). Slit-lamp photos were to be taken to assess the extent of the corneal crystal accumulation. To minimize bias when assessing the extent of corneal crystal accumulation, photos were centrally graded at the National Eye Institute (NEI) where each photo was graded independently by masked graders. If more than one CCCS was recorded in a given study year, the highest (worst) CCCS value was used for that year. The results were obtained from a combined analyses of the NIH cysteamine studies evaluating various cysteamine ophthalmic solution formulations from 1986 through 2005.

Outcome measures

Outcome measures
Measure	Cysteamine Topical Solution n=321 eyes Cysteamine topical solution administered hourly while awake in both eyes Cysteamine
Number of Eyes With a Corneal Cystine Crystal Score (CCCS) Response	98 eyes

Adverse Events

Cysteamine Topical Solution

Serious events: 11 serious events

Other events: 257 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cysteamine Topical Solution n=328 participants at risk Cysteamine topical solution administered hourly while awake in both eyes Cysteamine
Eye disorders Optic disc disorder	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Renal and urinary disorders Renal failure	0.61% 2/328 • Number of events 2 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Blindness	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Nervous system disorders Benign intracranial hypertension	2.4% 8/328 • Number of events 8 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Infections and infestations Gastrointestinal infection	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Gastrointestinal disorders Intestinal obstruction	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).

Other adverse events

Other adverse events
Measure	Cysteamine Topical Solution n=328 participants at risk Cysteamine topical solution administered hourly while awake in both eyes Cysteamine
Eye disorders Photophobia	72.9% 239/328 • Number of events 239 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Conjunctival hyperaemia	21.0% 69/328 • Number of events 69 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eye pain	20.1% 66/328 • Number of events 66 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Ocular hyperaemia	15.9% 52/328 • Number of events 52 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eye irritation	14.9% 49/328 • Number of events 49 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Lacrimation increased	7.0% 23/328 • Number of events 23 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Optic disc disorder	5.8% 19/328 • Number of events 19 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Vision blurred	6.1% 20/328 • Number of events 20 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Keratitis	5.8% 19/328 • Number of events 19 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Dry eye	4.3% 14/328 • Number of events 14 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eye pruritis	4.0% 13/328 • Number of events 13 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Conjunctivitis	3.7% 12/328 • Number of events 12 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eyelid oedema	3.7% 12/328 • Number of events 12 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Retinal disorder	3.4% 11/328 • Number of events 11 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Blindness	2.7% 9/328 • Number of events 9 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Corneal epithelium disorder	1.8% 6/328 • Number of events 6 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Blepharitis	1.5% 5/328 • Number of events 5 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Erythema of eyelid	1.5% 5/328 • Number of events 5 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eye swelling	0.91% 3/328 • Number of events 3 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Conjunctival oedema	0.61% 2/328 • Number of events 2 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eye oedema	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eyelid cyst	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Eyelid irritation	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Ulcerative keratitis	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Nervous system disorders Headache	28.7% 94/328 • Number of events 94 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Nervous system disorders Visual field defect	10.7% 35/328 • Number of events 35 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Nervous system disorders Benign intracranial hypertension	1.2% 4/328 • Number of events 4 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
General disorders Instillation site irritation	10.1% 33/328 • Number of events 33 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
General disorders Instillation site pain	6.7% 22/328 • Number of events 22 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
General disorders Adverse drug reaction	2.1% 7/328 • Number of events 7 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
General disorders Instillation site erythema	1.5% 5/328 • Number of events 5 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
General disorders Swelling	0.61% 2/328 • Number of events 2 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Eye disorders Drug ineffective	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
General disorders Instillation site lacrimation	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
General disorders Instillation site reaction	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Infections and infestations Eye infection	1.8% 6/328 • Number of events 6 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Infections and infestations Conjunctivitis ineffective	0.91% 3/328 • Number of events 3 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Infections and infestations Hordeolum	0.91% 3/328 • Number of events 3 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Gastrointestinal disorders Vomiting	2.4% 8/328 • Number of events 8 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Gastrointestinal disorders Abdominal pain	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Immune system disorders Drug hypersensitivity	0.61% 2/328 • Number of events 2 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).
Investigations Visual evoked potentials abnormal	0.30% 1/328 • Number of events 1 • Adverse events (AEs) were collected throughout the study. No specified time period is noted, as participants were followed until the drug became available commercially. Cystinosis patients typically have multiple medical problems, including kidney failure. The AEs reported do not reflect these systemic problems, focusing instead on ocular AEs (e.g., renal failure is noted in only 2 of 328 patients, though the majority have been managed for kidney disease and/or failure and have received a kidney transplant).

Additional Information

Rachel J. Bishop, MD, Principal Investigator, National Eye Institute

National Institutes of Health

Phone: 301-402-3771

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place