Last reviewed · How we verify
NCT03872531: LISA
Lifetime Impact Study for Achondroplasia
trial in Achondroplasia in 173 participants. Completed in 6 February 2021.
6 February 2021
Quick facts
| Lead sponsor | BioMarin Pharmaceutical |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 173 |
| Start date | 31 March 2019 |
| Primary completion | 6 February 2021 |
| Estimated completion | 6 February 2021 |
| Sites | 4 locations across Argentina, Colombia, Brazil |
Conditions studied
- Achondroplasia — all drugs for Achondroplasia →
Sponsor
BioMarin Pharmaceutical — full company profile →
Who can join
Adults 3 to 100, any sex, with Achondroplasia. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Observational study looking at the burden of illness in achondroplasia subjects aged 3 and above. The study will include a 3 year review of historical clinical data as well as a single point collection of questionnaire data to look at the impact on the following in individuals with achondroplasia versus a normative population: * Quality of life * Clinical burden * Healthcare resource use * Socio-economic burden * Psychosocial burden Up to 175 subjects will be enrolled in sites in Argentina, Colombia and Brazil.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Lifetime Impact Study for Achondroplasia (LISA): Findings from an observational and multinational study focused on health-related quality of life in individuals with achondroplasia in Latin America.
Llerena J, Rosselli P, Aragão A, Valenzuela C, et al · · 2024 · cited 1× · PMID 39669637 · DOI 10.1016/j.gimo.2023.100843
Verify or expand the search:
- PubMed search for NCT03872531
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Achondroplasia
Currently open trials in the same condition.
- NCT07301463 — A Study in Children With Achondroplasia · recruiting
- NCT06926491 — Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study） · Phase 3 · recruiting
- NCT05328050 — Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy) · recruiting
Other BioMarin Pharmaceutical trials
Trials by the same sponsor.
- NCT07477691 — Immune Modulation During Palynziq® Treatment in Adults (IMPALA) · Phase 4 · not yet recruiting
- NCT07126262 — A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months · Phase 2 · recruiting
- NCT06309979 — A Study to Assess Growth in Children With Idiopathic Short Stature · recruiting
- NCT06455059 — Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia · Phase 3 · active not recruiting
- NCT06305234 — A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) · 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 NCT03872531 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by BioMarin Pharmaceutical
- Last refreshed: 5 April 2021
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/NCT03872531.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing