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Cerebral Adrenoleukodystrophy (CALD)

3
Pipeline Programs
3
Companies
3
Clinical Trials
1 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
0
0
1
2
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

BioTherapeutics Inc
BioTherapeutics IncVA - Blacksburg
2 programs
1
1
Lenti-DPhase 31 trial
Lenti-D Drug ProductPhase 2/31 trial
Active Trials
NCT01896102Completed32Est. Mar 2021
NCT03852498Completed35Est. Jul 2023
Minoryx Therapeutics
Minoryx TherapeuticsSpain - Barcelona
1 program
1
LeriglitazonePhase 31 trial
Active Trials
NCT05819866Recruiting40Est. May 2027
Genetix Biotherapeutics
2 programs
Lenti-D Drug ProductPHASE_2_3
Lenti-DPHASE_3

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
Minoryx TherapeuticsLeriglitazone
BioTherapeutics IncLenti-D
BioTherapeutics IncLenti-D Drug Product

Clinical Trials (3)

Total enrollment: 107 patients across 3 trials

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

Start: Jul 2023Est. completion: May 202740 patients
Phase 3Recruiting

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Start: Jan 2019Est. completion: Jul 202335 patients
Phase 3Completed
NCT01896102BioTherapeutics IncLenti-D Drug Product

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Start: Aug 2013Est. completion: Mar 202132 patients
Phase 2/3Completed

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 107 patients
3 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.