Cystinosis

2
Pipeline Programs
4
Companies
10
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Recordati
RecordatiFrance - Saint-Victor
1 program
1
MercaptaminePhase 31 trial
Active Trials
NCT04125927Completed5Est. Dec 2022
Amgen
AmgenTHOUSAND OAKS, CA
1 program
1
RP103Phase 35 trials
Active Trials
NCT02473445Terminated22Est. Mar 2017
NCT02023866Completed36Est. Oct 2016
NCT01733316Completed41Est. Jul 2017
+2 more trials
Novartis
NovartisBASEL, Switzerland
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

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
RecordatiMercaptamine
AmgenRP103
AmgenRP103
AmgenRP103
AmgenRP103
AmgenRP103
AmgenRP103
AmgenRP103
NovartisSafety and Efficacy Assessments
Leadiant Biosciencescysteamine hydrochloride

Clinical Trials (10)

Total enrollment: 313 patients across 10 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
NCT01197378AmgenRP103

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

Start: Aug 2010Est. completion: Jun 201760 patients
Phase 3Completed
NCT01000961AmgenRP103

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
NCT02473445AmgenRP103

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

Start: May 2015Est. completion: Mar 201722 patients
Phase 2Terminated
NCT02023866AmgenRP103

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

Start: May 2014Est. completion: Oct 201636 patients
Phase 2Completed
NCT00872729AmgenRP103

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
NCT05146830NovartisSafety 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

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

2 late-stage (Phase 3) programs — potential near-term approvals
4 companies competing in this space