Last reviewed · How we verify
NCT04372446
Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency
trial in Generalized Arterial Calcification in Infancy in 38 participants. Completed in 30 September 2020.
30 September 2020
Quick facts
| Lead sponsor | Inozyme Pharma |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 38 |
| Start date | 6 May 2020 |
| Primary completion | 30 September 2020 |
| Estimated completion | 30 September 2020 |
| Sites | 1 location across United States |
Conditions studied
- Generalized Arterial Calcification in Infancy — all drugs for Generalized Arterial Calcification in Infancy →
- Autosomal Recessive Hypophosphatemic Rickets Type 2 — all drugs for Autosomal Recessive Hypophosphatemic Rickets Type 2 →
Sponsor
Inozyme Pharma — full company profile →
Who can join
Eligibility, any sex, with Generalized Arterial Calcification in Infancy or Autosomal Recessive Hypophosphatemic Rickets Type 2. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
To date, the investigators lack characterization of, the burden of, and the systemic progression of disease in ENPP1 deficiency and ABCC6 deficiency from a patient and/or parent perspective. This study aims to document this characterization, progression as well as the burden of disease. Link to the study registration- https://www.engagehealth.com/survey/TakeSurvey.aspx?SurveyID=8252n62
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Lifelong impact of ENPP1 Deficiency and the early onset form of ABCC6 Deficiency from patient or caregiver perspective.
O'Brien C, Khursigara G, Huertas P, Leiro B, et al · · 2022 · cited 3× · PMID 35895733 · DOI 10.1371/journal.pone.0270632
Verify or expand the search:
- PubMed search for NCT04372446
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other Inozyme Pharma trials
Trials by the same sponsor.
- NCT07473973 — ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency · Phase 3 · recruiting
- NCT06739980 — The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency · Phase 2 · withdrawn
- NCT06283589 — The SEAPORT 1 Study: Evaluation of the Safety and Tolerability of INZ-701 in Adults With End-Stage Kidney Disease Underg · Phase 1 · completed
- NCT05734196 — The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency · Phase 1 · recruiting
- NCT05050669 — Natural History Study of ENPP1 Deficiency and the Early-onset Form of ABCC6 Deficiency · completed
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 NCT04372446 (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 Inozyme Pharma
- Last refreshed: 1 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/NCT04372446.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing