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NCT03802279: EFFORHAB
Evaluate the Effort Test as a Therapeutic Monitoring Tool in Acute Rhabdomyolyses
trial testing Effort test in Rhabdomyolysis Linked to a Hereditary Disease of Metabolism in 27 participants. Completed in 31 December 2021.
31 December 2021
Quick facts
| Lead sponsor | Assistance Publique - Hôpitaux de Paris |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 27 |
| Start date | 25 October 2019 |
| Primary completion | 31 December 2021 |
| Estimated completion | 31 December 2021 |
| Sites | 1 location across France |
Drugs / interventions tested
- Effort test
- Functional tests on fibroblasts
Conditions studied
- Rhabdomyolysis Linked to a Hereditary Disease of Metabolism — all drugs for Rhabdomyolysis Linked to a Hereditary Disease of Metabolism →
Sponsor
Assistance Publique - Hôpitaux de Paris — full company profile →
Who can join
Adults 6 to 75, any sex, with Rhabdomyolysis Linked to a Hereditary Disease of Metabolism. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The prognosis of rhabdomyolyses related to hereditary diseases of metabolism is poor and treatments are only symptomatic. Rhabdomyolysis outbreaks are frequently precipitated by fever and fasting. They are unpredictable. In spite of the care of patient in an intensive care unit, the occurrence of renal failure and heart rhythm disorders explains a significant acute-phase mortality rate. There is an urgent need to understand the pathophysiological mechanisms of rhabdomyolyses related to hereditary diseases of metabolism, in order to identify specific treatments. Patients with rhabdomyolyses have few clinical signs outside of access. So there is a methodological difficulty in following a treatment test. There is an urgency to identify follow-up parameters in anticipation of new therapies. The objective of this study is to validate the hypothesis that effort test and cardiac function parameters are usable in the treatment monitoring for patients with acute rhabdomyolysis linked to a hereditary disease of metabolism and thus propose the effort test as an assessment tool for future clinical trials. In order to do so, the correlation between the results of the effort tests, performed to each patient with rhabdomyolysis related to a hereditary disease of metabolism, with the severity of the disease will be evaluated. This study is original because it opens up innovative prospects for monitoring in the field of hereditary diseases of metabolism, with the identification of new monitoring tools.
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 NCT03802279
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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 NCT03802279 (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 Assistance Publique - Hôpitaux de Paris
- Last refreshed: 12 September 2025
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/NCT03802279.
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