Back to discover
Disease

Hereditary diffuse leukoencephalopathy with axonal spheroids and pigmented glia

Late-stage therapeutic development
Also known as ALSP, Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia, Autosomal dominant leukoencephalopathy with neuroaxonal spheroids, FPSG+8 more

ALSP, Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia, Autosomal dominant leukoencephalopathy with neuroaxonal spheroids, FPSG, Familial dementia, Neumann type, Familial progressive subcortical gliosis, GPSC, HDLS, Hereditary diffuse leukoencephalopathy with spheroids, POLD, Pigmentary orthochromatic leukodystrophy, Subcortical gliosis of Neumann.

24
Clinical trials
3
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 4 clinical trials expected to report results, the earliest in Q1 2029.
Upcoming Milestones
Clinical Milestones14View all 14
Results posted 4
Results expected 4
Recently completed 3
Other updates 3
+6 more in the activity timeline below
Activity timeline14

Clinical trials

10 sponsors · 2 new · 5 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme
24
All trials
6
Active
17
Late-stage
6
Completed
Late-stage studies

Randomized Trial of Consolidation Targeted Adjuvant Therapy With Encorafenib and Cetuximab Versus Usual Care for Patients With Stage II/III BRAF V600E Colon Cancer

Phase 2/3 · Active, not recruiting · Alliance for Clinical Trials in Oncology

A Phase III Randomized Trial of Eribulin (NSC #707389) With Gemcitabine Versus Standard of Care (Physician's Choice) for Treatment of Metastatic Urothelial Carcinoma Refractory to, or Ineligible for, Anti PD1/PDL1 Therapy

Phase 3 · Active, not recruiting · National Cancer Institute (NCI)

Phase III Randomized Trial of Immunotherapy With or Without Consolidative Radiotherapy for Oligometastatic Head and Neck Squamous Cell Carcinoma

Phase 3 · Active, not recruiting · ECOG-ACRIN Cancer Research Group

Phase II/III Second-Line NABPLAGEM vs. Nab-Paclitaxel/Gemcitabine in BRCA1/2 or PALB2 Mutant Metastatic Pancreatic Ductal Adenocarcinoma (PLATINUM)

Phase 2/3 · Active, not recruiting · Alliance for Clinical Trials in Oncology

A Randomized Phase 2/3 Study of Olaparib Plus Temozolomide Versus Investigator's Choice for the Treatment of Patients With Advanced Uterine Leiomyosarcoma After Progression on Prior Chemotherapy

Phase 2/3 · Active, not recruiting · National Cancer Institute (NCI)

A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy With Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) Plus VINO-CPO Maintenance in Patients With High Risk Rhabdomyosarcoma (HR-RMS)

Phase 3 · Active, not recruiting · Children's Oncology Group
Recently completed

A Multicenter, Randomized, Double-Blind, Positive-Controlled, Cross-Over Phase Ⅲ Clinical Trial Evaluating the Efficacy and Safety of HRS-9231 for Central Nervous System (CNS) Magnetic Resonance Imaging (MRI)

Phase 3 · Completed · Shanghai Shengdi Pharmaceutical Co., Ltd

A Multicenter, Randomized, Double-Blind, Positive-Controlled, Cross-Over Phase III Clinical Trial Evaluating the Efficacy and Safety of HRS-9231 for Body Parts (Non-Central Nervous System) Magnetic Resonance Imaging (MRI)

Phase 3 · Completed · Shanghai Shengdi Pharmaceutical Co., Ltd

A Multicenter, Prospective, Open-label Study to Evaluate the Pharmacokinetics and Safety of Gadoquatrane in Pediatric Participants (From Birth to <18 Years) Undergoing Contrast-enhanced Magnetic Resonance Imaging (CE-MRI)

Phase 3 · Completed · Bayer

A Phase II-R and a Phase III Trial Evaluating Both Erlotinib (Ph II-R) and Chemoradiation (Ph III) as Adjuvant Treatment for Patients With Resected Head of Pancreas Adenocarcinoma

Phase 3 · Completed · National Cancer Institute (NCI)

Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

Phase 3 · Completed · Children's Oncology Group

Treatment for Very Low and Standard Risk Favorable Histology Wilms Tumor

Phase 3 · Completed · Children's Oncology Group

Associated genes

3 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

CSF1R0.83STAM0.05SELENOT0.04

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Macrophage colony-stimulating factor 1 receptorvia CSF1R0.83

Reference

Authoritative identity, definition & identifiers.

Synonyms

ALSP, Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia, Autosomal dominant leukoencephalopathy with neuroaxonal spheroids, FPSG, Familial dementia, Neumann type, Familial progressive subcortical gliosis, GPSC, HDLS, Hereditary diffuse leukoencephalopathy with spheroids, POLD, Pigmentary orthochromatic leukodystrophy, Subcortical gliosis of Neumann

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.