Last reviewed · How we verify
NCT01358370
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
trial in Lysosomal Acid Lipase Deficiency in 40 participants. Completed in 1 March 2013.
1 January 2013
Quick facts
| Lead sponsor | Alexion Pharmaceuticals, Inc. |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 40 |
| Start date | 1 November 2010 |
| Primary completion | 1 January 2013 |
| Estimated completion | 1 March 2013 |
| Sites | 14 locations across United States, Canada, France, Italy, United Kingdom |
Conditions studied
- Lysosomal Acid Lipase Deficiency — all drugs for Lysosomal Acid Lipase Deficiency →
- Wolman Disease — all drugs for Wolman Disease →
Sponsor
Alexion Pharmaceuticals, Inc. — full company profile →
Who can join
Eligibility, any sex, with Lysosomal Acid Lipase Deficiency or Wolman Disease. Patients with the condition only — healthy volunteers not accepted.
What's being measured
Primary outcomes are the specific endpoints the trial is designed to prove or disprove.
-
Time to Death
Time frame: Up to two years.
The time to death will be analyzed using Kaplan-Meier curves. Estimates (with exact 95% confidence interval \[CI\]) of the median and the lower and upper quartiles of time to death will be derived.
Sponsor's own description
This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.
Publications & conference data
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
Verify or expand the search:
- PubMed search for NCT01358370
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Lysosomal Acid Lipase Deficiency
Currently open trials in the same condition.
- NCT07455864 — Lysosomal Acid Lipase Deficiency in Risk Groups · recruiting
- NCT01633489 — Lysosomal Acid Lipase (LAL) Deficiency Registry · recruiting
Other Alexion Pharmaceuticals, Inc. trials
Trials by the same sponsor.
- NCT06015750 — Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia · Phase 4 · withdrawn
- NCT07352423 — Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants · Phase 1 · recruiting
- NCT07221838 — A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab · Phase 4 · recruiting
- NCT07413250 — Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis o · active not recruiting
- NCT07308574 — Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS · Phase 4 · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT01358370 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc.
- Last refreshed: 23 June 2016
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01358370.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing