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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.

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30 closest matches · ranked by relevance

N/A N=117 Alpha-1 Carrier Genomics Study

Alpha-1 Antitrypsin Deficiency · Emphysema · COPD
Primary: Number of Participants With Abnormal Sequences in SERPINA1 Genes — 6; 0 participants — p=0.054
Medical University of South Carolina Results Jul 2019 0.0% serious AE View details

Phase 2 N=63 Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

Alpha-1 Antitrypsin Deficiency (AATD) · Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations · Emphysema or COPD
Primary: Within-individual % Change in Plasma Desmosine/Isodesmosine — 10.0619; 8.5627 % change
University of Alabama at Birmingham Results Jul 2024 0.0% serious AE View details

Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors

Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Masonic Cancer Center, University of Minnesota Results Jul 2019 26.3% serious AE View details

Phase 2 N=60 ALS Reversals - Lunasin Regimen

ALS (Amyotrophic Lateral Sclerosis)
Primary: Change in Revised ALS Functional Rating Scale (ALSFRS-R) — 0.44 points per month — p=0.99
Richard Bedlack, M.D., Ph.D. Results Dec 2018 26.0% serious AE View details

Phase 2 N=23 A Study of Alirocumab in Participants With Autosomal Dominant Hypercholesterolemia (ADH) and Gain-of-Function Mutations (GOFm) of the Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Gene or Loss-of-Function Mutations (LOFm) of the Apolipoprotein (Apo) B Gene

Hypercholesterolemia
Primary: Percent Change in Measured Serum Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 15 — -62.48; -8.77; -48.21; -4.93 percent change — p== 0.0009
Regeneron Pharmaceuticals Results Jun 2020 9.1% serious AE View details