# Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

> **NCT01307098** · PHASE1,PHASE2 · COMPLETED · sponsor: **Alexion Pharmaceuticals, Inc.** · enrollment: 9 (actual)

## Conditions studied

- Cholesterol Ester Storage Disease(CESD)
- Lysosomal Acid Lipase Deficiency
- LAL-Deficiency

## Interventions

- **DRUG:** Sebelipase alfa 0.35 mg/kg
- **DRUG:** Sebelipase alfa 1 mg/kg
- **DRUG:** Sebelipase alfa 3 mg/kg

## Key facts

- **NCT ID:** NCT01307098
- **Lead sponsor:** Alexion Pharmaceuticals, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2011-04-25
- **Primary completion:** 2012-01-06
- **Final completion:** 2012-01-06
- **Target enrollment:** 9 (ACTUAL)
- **Last updated:** 2018-12-11


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01307098

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01307098, "Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency". Retrieved via AI Analytics 2026-07-07 from https://api.ai-analytics.org/clinical/NCT01307098. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*