Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Cystinosis: later-stage trial registry
2 of 5 indexed Cystinosis trials are currently recruiting, with more weight in Phase 3-4.
Corpus placement: #3,391 of 28,707 conditions by registered trial count.
5 US clinical trials · 2 currently recruiting · #3,391 of 28,707 conditions by registered trial count
Cystinosis: 5 US clinical trials tracked, 2 recruiting.
Cystinosis is the subject of 5 registered US clinical trials on ClinicalTrials.gov, 2 of them currently open to new participants. 2 are in Phase 3-4 (later-stage) and 1 in Phase 1-2 (earlier-stage). The most active sponsor is Amgen, running 2 of these trials.
Enrollment posture brief
Registry condition cystinosis
OPEN 2 | RECR 40% | LATE 2 | EARLY 1 | RANK #3391 | SPON 2
Volume-matched condition peers
Nearest open-share peer: Achondroplasia (40% recruiting · 10 trials)
Cystinosis lists 2 open slots (40% of 5 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Cystinosis on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
-
Cystinosis is recruiting below its size-band peers.
2 of 5 indexed trials (40%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
-
One sponsor concentrates Cystinosis research.
Amgen accounts for 2 of 5 trials (40%). 4 distinct sponsors appear on at least one study for this condition label.
-
Most Cystinosis trials plan to enrol fewer than 100 people.
Median target enrolment is 60 among the 5 trials that report a genuine target (sentinel values excluded). 3 of those (60%) plan for fewer than 100 participants.
-
Cystinosis splits between interventional and observational designs.
3 interventional and 2 observational studies make up this index (60% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
5 total, page 1 of 1
Use of Cysteamine in the Treatment of Cystinosis
National Human Genome Research Institute (NHGRI)
NCT00359684
Early Trial of Allogeneic Hematopoietic Stem Cell Transplantation for Patients Who Will Receive a Kidney Transplant From the Same Donor
Alice Bertaina
NCT05508009
Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis
National Center for Research Resources (NCRR)
NCT00004312
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Amgen
NCT01197378
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
Amgen
NCT01744782
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 3 | 2 |
Top Sponsors
Named interventions
Most-linked intervention names on Cystinosis records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Cystinosis trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Cystinosis page
5 registered trials, 2 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 2 Cystinosis trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Amgen sponsors the most Cystinosis trials on record, review their full trial history before evaluating a specific study. See Amgen's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Cystinosis condition card totals 5 registered trials; most-linked intervention RP103; top US site state California. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.