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Clinical Trial Search

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

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 1 N=32 A Dose-escalating Clinical Trial With KH176

MELAS · LHON · Leigh Syndrome
Primary: SAD: Change From Baseline in ECG Results by Timepoint: Corrected QT Interval According to Fridericia's Formula (QTcF) — 386.2; 395.0; 385.8; 399.3 msec
Khondrion BV Results Nov 2020 0.0% serious AE View details

Phase 4 N=13 High Dose or High Dose Frequency Study of Alglucosidase Alfa

Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis 2 Acid Maltase Deficiency
Primary: Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment — 0; 0; 0; 1…
Genzyme, a Sanofi Company Results Apr 2011 25.0% serious AE View details

N/A N=41 Fatty Acid Oxidation Defects and Insulin Sensitivity

Very Long-chain Acyl-CoA Dehydrogenase Deficiency · Trifunctional Protein Deficiency · Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
Primary: Glucose Disposal Rate (Rd)- the Rate of Glucose Infusion to Maintain Euglycemia During Steady State Insulin Infusion in mg/Min — 709; 429; 842; 497 mg/min — p=0.136
Oregon Health and Science University Results Jan 2024 5.1% serious AE View details