---
title: CYSTEA-BONE Clinical Study
nct_id: NCT03919981
overall_status: RECRUITING
sponsor: Hospices Civils de Lyon
study_type: OBSERVATIONAL
primary_condition: Nephropathic Cystinosis
countries: France, Germany, Italy, Turkey (Türkiye)
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT03919981.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT03919981"
ct_last_update_post_date: 2025-03-03
last_seen_at: "2026-05-12T06:30:07.514Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# CYSTEA-BONE Clinical Study

**Official Title:** A European, Multicenter, Prospective Clinical Study to Evaluate Cysteamine Toxicity on Human Osteoclasts. The CYSTEA-BONE Clinical Study.

**NCT ID:** [NCT03919981](https://clinicaltrials.gov/study/NCT03919981)

## Key Facts

- **Status:** RECRUITING
- **Study Type:** OBSERVATIONAL
- **Target Enrollment:** 50
- **Lead Sponsor:** Hospices Civils de Lyon
- **Conditions:** Nephropathic Cystinosis
- **Start Date:** 2019-04-05
- **Completion Date:** 2026-10-05
- **CT.gov Last Update:** 2025-03-03

## Brief Summary

Nephropathic Cystinosis (NC) is an orphan inherited autosomal recessive disease characterised as a generalized lysosomal storage disease due to a deficiency of the cystine lysosomal transport protein, cystinosin.

Patients with NC usually receive cysteamine. Bone impairment was recently recognized as a late complication of NC, occurring at adolescence or early adulthood. Even though the exact underlying pathophysiology is unclear, at least six hypotheses are discussed, and mainly cysteamine toxicity and/or direct bone effect of the Cystinosin (CTNS) mutation. Because of the potential dramatic impact on quality of life of this novel complication, research should aim to better understand bone disease in NC.

The primary objective of this study is to evaluate the action of cysteamine on osteoclastic differentiation and resorption activity of NC patients, depending on the underlying genotype. The Secondary objective is to describe the clinical bone status of NC patients depending on their underlying genotype.

## Eligibility

- **Minimum age:** 2 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
Inclusion Criteria:

* Male and female subjects with confirmed diagnosis of nephropathic cystinosis (defined by clinical signs, White Blood Cells (WBC) cystine level and/or mutation), currently receiving oral cysteamine.
* Age \> 2 years.
* Subjects and/or their parents/ legal guardian must provide non opposition prior to participation in the study.

Exclusion Criteria:

* Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
```

## Arms

- **nephropathic cystinosis patients receiving cysteamine** — nephropathic cystinosis patients receiving cysteamine. The blood samples of the group will be used to evaluate the action of cysteamine on osteoclastic differentiation and resorption activity of NC patients, depending on the underlying genotype.

## Interventions

- **Blood sampling** (OTHER) — 25 mL blood sample will be collected on citrate tubes for osteoclastic analysis.

## Primary Outcomes

- **Number of positive Tartrate-resistant acid phosphatase (TRAP) cells** _(time frame: 1 day)_ — Number of positive TRAP cells will be assessed at the end of osteoclast differentiation from circulating monocytes

## Locations (13)

- CHU de Besançon, Besançon, France — _NOT_YET_RECRUITING_
- CHU Bordeaux - Hôpital Pellegrin tripode, Bordeaux, France — _RECRUITING_
- Hôpital Femme Mère Enfant, Bron, France — _RECRUITING_
- Hôpital Jeanne de Flandre, Lille, France — _RECRUITING_
- Hopital Edouard Herriot, Lyon, France — _RECRUITING_
- AP-HM - Timone Enfants, Marseille, France — _NOT_YET_RECRUITING_
- CHU Paris - Hôpital Robert Debré, Paris, France — _RECRUITING_
- CHU Paris - Hôpital Necker-Enfants Malades, Paris, France — _RECRUITING_
- Hôpital des Enfants, Toulouse, France — _NOT_YET_RECRUITING_
- CHRU Nancy - Hôpital Brabois Enfants, Vandœuvre-lès-Nancy, France — _RECRUITING_
- Klinik für Pädiatrische Nieren-, Leber- und Stoffwechselerkrankungen, Hanover, Germany — _NOT_YET_RECRUITING_
- IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy — _NOT_YET_RECRUITING_
- Hacettepe University Faculty of Medicine, Ankara, Turkey (Türkiye) — _NOT_YET_RECRUITING_

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.chu de besançon|besançon||france` — added _(2026-05-12)_
- `locations.chu bordeaux - hôpital pellegrin tripode|bordeaux||france` — added _(2026-05-12)_
- `locations.hôpital femme mère enfant|bron||france` — added _(2026-05-12)_
- `locations.hôpital jeanne de flandre|lille||france` — added _(2026-05-12)_
- `locations.hopital edouard herriot|lyon||france` — added _(2026-05-12)_
- `locations.ap-hm - timone enfants|marseille||france` — added _(2026-05-12)_
- `locations.chu paris - hôpital robert debré|paris||france` — added _(2026-05-12)_
- `locations.chu paris - hôpital necker-enfants malades|paris||france` — added _(2026-05-12)_
- `locations.hôpital des enfants|toulouse||france` — added _(2026-05-12)_
- `locations.chru nancy - hôpital brabois enfants|vandœuvre-lès-nancy||france` — added _(2026-05-12)_
- `locations.klinik für pädiatrische nieren-, leber- und stoffwechselerkrankungen|hanover||germany` — added _(2026-05-12)_
- `locations.irccs ospedale pediatrico bambino gesù|roma||italy` — added _(2026-05-12)_
- `locations.hacettepe university faculty of medicine|ankara||turkey (türkiye)` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT03919981.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT03919981*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*