About this data Sourced primarily from ClinicalTrials.gov — a listing isn't proof of safety or FDA approval.

Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.

Not yet reported

cALD HR-D

Company: Masonic Cancer Center, University of Minnesota
Mechanism: Mechanism not yet reported
Route: Route not yet reported
Recruiting trials: None currently listed

Efficacy results: Not yet reported

No recruiting studies currently listed

See all studies for this drug

Data source: ClinicalTrials.gov · trial data as of Nov 4, 2026

Trial details

Active, not recruiting NCT NCT02171104

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Not yet reported Masonic Cancer Center, University of Minnesota 149 participants Est. completion 2031-07-14 1 site Stand-alone

Obstructive sleep apnea Other conditions: Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
View official trial listing →

Efficacy history

Not yet reported.

Safety

Not yet reported.

Sources

ClinicalTrials.gov — NCT02171104

Also known as: cALD HR-D, cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)

Related On The Pen coverage

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This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.