NCT05789355

Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)

Effect of NUV001 Supplementation for 120 Days in Patients Suffering From Sickle Cell Disease (SCD) S Homozygous (SS) Genotype: A Pilot Study

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: LGD · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety and tolerability parameters as well as to measure the plasma and urinary residues of daily oral doses of NUV001. Secondly, the study will evaluate the impact of NUV001 on biological parameters and quality of life of patients.

Detailed description

This is a monocentric, prospective, open label pilot study designed for 12 adult patients suffering of Sickle Cell disease (SCD) SS genotype each 12 receiving the active supplementation of NUV001, 1000mg/day (4 x 250 mg tablet) for 3 months of follow-up plus 1 month after supplementation. A stratification according to the medical treatment is planned. At least 2 patients suffering of SCD SS genotype without hydroxyurea treatment and maximum 10 patients suffering of SCD SS genotype in association with hydroxyurea treatment. If a subject is withdrawn from this study part, the subject may be replaced as necessary with another subject assigned to the same treatment at the discretion of the sponsor's team in consultation with the investigator. The current study is designed to assess in the first part, the safety, tolerability, plasma, and urine residual rate parameters of daily oral doses of NUV001 as dietary supplement n adult patients suffering of sickle cell disease SS genotype. In a second part, the study will assess the pharmacological impact of NUV001 on biological parameters and the quality of life in patient suffering of sickle cell disease SS genotype.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	NUV001	Daily supplementation with NUV001 at 1000 mg (4 tablets of 250 mg each) for 90 days with a prolonged follow-up of 1 month (30 days) after stopping the supplementation

Timeline

• Start date: 2023-04-01
• Primary completion: 2024-05-10
• Completion: 2024-06-30
• First posted: 2023-03-29
• Last updated: 2024-05-28

Locations

1 site across 1 country: France