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Cystinosis

8
Pipeline Programs
8
Companies
12
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
4
0
0
4
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

8 companies ranked by most advanced pipeline stage

Amgen
AmgenTHOUSAND OAKS, CA
5 programs
1
4
Cystagon®Phase 31 trial
Cysteamine Bitartrate Delayed-release CapsulesPhase 31 trial
RP103Phase 31 trial
RP103Phase 31 trial
Cystagon®Phase 1/21 trial
Active Trials
NCT00872729Completed9Est. Oct 2009
NCT01000961Completed43Est. Aug 2011
NCT01197378Completed60Est. Jun 2017
+2 more trials
Human BioSciences
Human BioSciencesWV - Martinsburg
3 programs
1
rh Growth HormonePhase 1/2
CysteamineN/A
Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic CystinosisN/A1 trial
Active Trials
NCT00004312Completed400
Genome & Company
Genome & CompanyKorea - Suwon
2 programs
1
rh Growth HormonePhase 1/21 trial
CysteamineN/A1 trial
Active Trials
NCT00359684Recruiting330
NCT02124070Withdrawn0Est. Nov 2015
Nacuity Pharmaceuticals
1 program
1
Cysteamine BitartratePhase 1/21 trial
Active Trials
NCT05994534Recruiting12Est. Aug 2026
Novartis
NovartisBASEL, Switzerland
1 program
Safety and Efficacy AssessmentsN/A
Sandoz
SandozAustria - Kundl
1 program
Safety and Efficacy AssessmentsN/A1 trial
Active Trials
NCT05146830Enrolling By Invitation50Est. Nov 2036
Leadiant Biosciences
Leadiant BiosciencesMD - Rockville
1 program
cysteamine hydrochlorideN/A1 trial
Active Trials
NCT00010426Completed30Est. Feb 2001
Recordati
RecordatiFrance - Saint-Victor
1 program
MercaptaminePHASE_31 trial
Active Trials
NCT04125927Completed5Est. Dec 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
RecordatiMercaptamine
AmgenRP103
AmgenRP103
AmgenCysteamine Bitartrate Delayed-release Capsules
AmgenCystagon®
Nacuity PharmaceuticalsCysteamine Bitartrate
Genome & Companyrh Growth Hormone
AmgenCystagon®
SandozSafety and Efficacy Assessments
Leadiant Biosciencescysteamine hydrochloride
Human BioSciencesEstablishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis
Genome & CompanyCysteamine

Clinical Trials (12)

Total enrollment: 997 patients across 12 trials

NCT04125927RecordatiMercaptamine

Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)

Start: Sep 2020Est. completion: Dec 20225 patients
Phase 3Completed
NCT01733316AmgenRP103

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

Start: Jan 2013Est. completion: Jul 201741 patients
Phase 3Completed
NCT01744782AmgenRP103

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

Start: Dec 2012Est. completion: Dec 201617 patients
Phase 3Completed
NCT01197378AmgenCysteamine Bitartrate Delayed-release Capsules

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

Start: Aug 2010Est. completion: Jun 201760 patients
Phase 3Completed
NCT01000961AmgenCystagon®

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Start: Jun 2010Est. completion: Aug 201143 patients
Phase 3Completed
NCT05994534Nacuity PharmaceuticalsCysteamine Bitartrate

PK and PD Study of NPI-001 and Cysteamine Bitartrate

Start: Oct 2023Est. completion: Aug 202612 patients
Phase 1/2Recruiting
NCT02124070Genome & Companyrh Growth Hormone

Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis

Start: Mar 2014Est. completion: Nov 20150
Phase 1/2Withdrawn
NCT00872729AmgenCystagon®

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Start: May 2009Est. completion: Oct 20099 patients
Phase 1/2Completed
NCT05146830SandozSafety and Efficacy Assessments

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

Start: Nov 2022Est. completion: Nov 203650 patients
N/AEnrolling By Invitation
NCT00010426Leadiant Biosciencescysteamine hydrochloride

Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

Start: Dec 1999Est. completion: Feb 200130 patients
N/ACompleted
NCT00004312Human BioSciencesEstablishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis

Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis

Start: Oct 1999400 patients
N/ACompleted
NCT00359684Genome & CompanyCysteamine

Use of Cysteamine in the Treatment of Cystinosis

Start: Jan 1979330 patients
N/ARecruiting

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

4 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 997 patients
8 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.