Cystinosis Clinical Trial
Official title:
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce white blood cell (WBC) cystine will be assessed and compared with cysteamine. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT05545774 -
Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
|
||
| Completed |
NCT01432561 -
Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™
|
N/A | |
| Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
| Completed |
NCT04125927 -
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
|
Phase 3 | |
| Completed |
NCT00071903 -
The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study
|
N/A | |
| Completed |
NCT00004312 -
Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis
|
N/A | |
| Completed |
NCT00004350 -
Evaluation of Fanconi Syndrome and Cystinosis
|
N/A | |
| Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
| Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
|
||
| Completed |
NCT02533076 -
The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients
|
Phase 0 | |
| Enrolling by invitation |
NCT05146830 -
A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
|
||
| Completed |
NCT00872729 -
Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
|
Phase 1/Phase 2 | |
| Completed |
NCT00001736 -
New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis
|
Phase 1 | |
| Recruiting |
NCT05959668 -
Development of Health-related Quality of Life Instrument for Patients With Cystinosis
|
||
| Withdrawn |
NCT02124070 -
Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
|
Phase 1/Phase 2 | |
| Completed |
NCT01614431 -
N Acetyl Cysteine for Cystinosis Patients
|
Phase 4 | |
| Recruiting |
NCT05843851 -
Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria
|
N/A | |
| Completed |
NCT01000961 -
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
|
Phase 3 | |
| Active, not recruiting |
NCT03897361 -
Stem Cell Gene Therapy for Cystinosis
|
Phase 1/Phase 2 | |
| Recruiting |
NCT01793168 -
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
|