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Condition (MeSH)

Cystinosis

ClinicalTrials.gov lists 47 trials for Cystinosis or a narrower condition beneath it. Amgen has the most among industry and government lead sponsors, with 5.

MeSH D003554 Congenital / Hereditary / Neonatal C16 C18 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 2 Phase 1/2 Phase 1/2 6 Phase 2 Phase 2 4 Phase 3 Phase 3 5 Phase 4 Phase 4 1 Not applicable Not applicable 10 Observational Observational 19 Phase 1 Phase 1 2 Phase 1/2 Phase 1/2 6 Phase 2 Phase 2 4 Phase 3 Phase 3 5 Phase 4 Phase 4 1 Not applicable Not applicable 10 Observational Observational 19

Trials per phase

47 of the 47 trials are tagged with Cystinosis itself; the rest are tagged with a narrower condition beneath it.

Get the Cystinosis report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Cystinosis trials start? (20)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 5

1998: 1 trials (Phase 1 1) 1999: 3 trials (Observational 2, Not applicable 1) 2003: 3 trials (Observational 2, Phase 2 1) 2004: 2 trials (Observational 1, Not applicable 1) 2008: 1 trials (Not applicable 1) 2009: 2 trials (Observational 1, Phase 1/2 1) 2010: 4 trials (Observational 2, Phase 3 2) 2011: 3 trials (Not applicable 2, Phase 4 1) 2012: 1 trials (Phase 3 1) 2013: 1 trials (Phase 3 1) 2014: 2 trials (Phase 1/2 1, Phase 1 1) 2017: 1 trials (Observational 1) 2018: 2 trials (Observational 1, Not applicable 1) 2019: 3 trials (Observational 1, Phase 2 1, Phase 1/2 1) 2020: 2 trials (Observational 1, Phase 3 1) 2021: 1 trials (Observational 1) 2022: 5 trials (Observational 4, Not applicable 1) 2023: 3 trials (Not applicable 1, Phase 1/2 2) 2025: 2 trials (Not applicable 1, Phase 1/2 1)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 3
2025 2
2023 3
2022 5
2021 1
2020 2
2019 3
2018 2
2017 1
2014 2

Showing 10 of 20 earlier years.

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No forecast is made for this condition: it averages 2.6 trial starts a year over the five years to September, under the 5 a year the method needs.

Recruitment duration, a model prediction: 4 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Cystinosis trials in? (7)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 2
Phase 1/2 6
Phase 2 4
Phase 3 5
Phase 4 1
Not applicable 10
Observational 19

How many Cystinosis trials are recruiting? (8)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 3
Recruiting 10
Enrolling by invitation 1
Active, not recruiting 2
Completed 26

Showing 5 of 8 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Cystinosis trials? (6)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
Amgen 5
Institut National de la Santé Et de la Recherche Médicale, France 2
National Center for Research Resources (NCRR) 2
National Eye Institute (NEI) 2
National Human Genome Research Institute (NHGRI) 2

Showing 5 of 6 sponsors.

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Which drugs are being tested for Cystinosis? (4)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
(Cystagon) Cysteamine Bitartrate 13
RITUXIMAB 1
N-Acetylcysteine Amide 1
Cytoxan 1

Interventions as named in the registry (11)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
RP103 3
Cysteamine Bitartrate Delayed-release Capsules 2
TTI-0102 1
ATG 1
N Acetyl Cysteine 1

Showing 5 of 11 interventions.

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These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Cystinosis trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Gastroenteritis 15 4
Dehydration 13 3
Vomiting 11 4
Benign intracranial hypertension 8 1
Appendicitis 7 1

Showing 5 of 10 adverse events.

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Which publications cover Cystinosis? (38)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Newborn Screening: Review of its Impact for Cystinosis. Cells 2022 Yes
Relationship between age at initiation of cysteamine treatment, adherence with therapy, and glomerular kidney function in infantile nephropathic cystinosis. Molecular genetics and metabolism 2022 Yes
Beneficial effects of starting oral cysteamine treatment in the first 2 months of life on glomerular and tubular kidney function in infantile nephropathic cystinosis. Molecular genetics and metabolism 2022 Yes
Adherence to cysteamine in nephropathic cystinosis: A unique electronic monitoring experience for a better understanding. A prospective cohort study: CrYSTobs. Pediatric nephrology (Berlin, Germany) 2021 Yes
Next generation sequencing as second-tier test in high-throughput newborn screening for nephropathic cystinosis. European journal of human genetics : EJHG 2020 Yes

Showing 5 of 38 publications.

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Prevalence (30)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Cystinosis Prevalence at birth 1-9 / 100 000 United Kingdom GB Unspecified
Cystinosis Prevalence at birth 1-9 / 100 000 United Kingdom GB Exact
Cystinosis Prevalence at birth 1-9 / 100 000 United Kingdom GB Exact
Cystinosis Prevalence at birth 1-9 / 1 000 000 Germany Not stated Exact
Cystinosis Prevalence at birth 1-9 / 1 000 000 France Not stated Exact

Showing 5 of 30 prevalence observations.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Cystinosis 213 Exact Orphadata Science knowledge files, Product 1
Cystinosis 213 Exact Orphanet Rare Disease Ontology (ORDO)

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: sub-conditions in mesh.

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