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Cystinosis clinical trials

The registry lists 47 studies for Cystinosis across more than 10 countries. 15 are recruiting or about to start. 39 of them are mainly about Cystinosis (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-03.

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A study of drug A after surgery for early breast cancer

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Status

Completed
28
Recruiting
10
Not yet recruiting
3
Enrolling by invitation
2
Status unknown
1
Active, not recruiting
1

Phase

Phase 2
10
Phase 1
7
Phase 3
5
Phase 4
1
Early Phase 1
1

Where

United States
22
France
11
Germany
5
Belgium
5
Italy
4
Netherlands
3

Who runs them

Amgen (5) · Cystinose Stiftung (3) · Hospices Civils de Lyon (3) · National Human Genome Research Institute (NHGRI) (2) · Novartis Pharmaceuticals (2) · Institut National de la Santé Et de la Recherche Médicale, France (2) · National Center for Research Resources (NCRR) (2) · National Eye Institute (NEI) (2)

What is being tested here

Drugs under test in Cystinosis studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Cysteamine-pantetheine disulfide101
Cyclophosphamide101
Melphalan101
Cysteamine0511
N-Acetylcysteinamide001
Deferasirox001
Acetylcysteine001
ELX-02001

Coming up

0phase 3 studies under way
3studies finish main data collection within a year

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

All 3

Who these studies take

Of the 10 Cystinosis studies recruiting now, 4 (40%) set no upper age limit and 4 (40%) stop at 75 or younger. 10 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.

Recruiting studies that take each age, of 10
AgeWomenMen
108 (80%)8 (80%)
188 (80%)8 (80%)
307 (70%)7 (70%)
406 (60%)6 (60%)
506 (60%)6 (60%)
656 (60%)6 (60%)
756 (60%)6 (60%)
856 (60%)6 (60%)

Across all 47 studies, recruiting or not: 38% stop at 75 or younger and 36% at 65; 89% take children; 0% are for women only and 0% for men only; 13% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 47 of 47. search them.