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

A study testing NUV001 in people with Sickle Cell Disease, run by LGD at 1 site in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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… Read more about this study

Who is it for?: Ages 18 to 60 years
Conditions the study is for: Sickle Cell Disease
What is being tested?: NUV001
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 12 participants
Where: 1 siteFrance
Study dates: 30 Mar 2023to 6 May 2024
Phase: Interventional
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 60 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Participants meeting the following criteria could be included:
Male or female between 18 and 60 years old.
Females of childbearing potential should be using one of the following acceptable methods of birth control:
Intrauterine Device in place for at least 60 days prior to the first dose of the study (Visit 1) throughout the study and for 30 days after completion of the study.
Hormonal contraceptives for at least 90 days prior to the first dose of the study (Visit 1) throughout the study, and for 30 days after study completion.
Patients whose weight is greater than 50 kg.

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Patients diagnosed with homozygous sickle cell anemia of SS genotype (documented by genotyping).
Patients who have been treated with an anti-sickling agent (Siklos®) within six months of the screening visit (Visit 0) must maintain the therapy continuous and unmodified for at least six months with the intent to continue for the duration of the study.
Patients who are available to attend on an outpatient basis for visits provided for in the protocol and can complete the data collection documents (and quality of life scale).
Patients have given written informed consent.
Patients with a health insurance coverage.

Who cannot join

Participants meeting the following criteria could not be included:
Patients with known or suspected allergies to any ingredient of the food supplement (β-NMN, Isomalt, Magnesium stearate, microcrystalline cellulose).
Patients who have consumed food supplements containing tryptophan, glutamine or vitamin B3 in various forms (nicotinic acid/niacin and nicotinamide) during the month before selection.
Patients have a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit (Visit 0).
Patients have serum albumin < 3.0 g/dl (< 30 g/L).
Patients have been transfused and received any blood products within three months of the Screening Visit (Visit 0).

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Patients have been hospitalized for acute vaso-occlusive crisis within one month of the Screening Visit (Visit 0).
Patient has clinically significant cardiovascular or liver disease, renal or lung insufficiency or lymphopenia (with clinically significant abnormal results on the screening bioassays: CBC, transaminases (AST, ALT, GGT, ALP), bilirubin, creatinine, CPK, ionogram, blood glucose, lipid profile).
Patients with a diagnosed cancer in the past 2 years.
Pregnant or lactating woman. Women of childbearing potential should have a negative serum or urine pregnancy test at screening and a negative urine pregnancy test at inclusion prior to administration of the study product.

Trial locations and contacts

1 site in 1 country.

France1 site

Aphm Hopital La Timone Adultes Sce Medecine Interne (Umap)
Marseille, 13005

Other trials in France

About this study

Official title: EFFECT OF NUV001 SUPPLEMENTATION FOR 120 DAYS IN PATIENTS SUFFERING FROM SICKLE CELL DISEASE (SCD) SS GENOTYPE: A PILOT STUDY

Read the 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 food supplement in adult patients with SCD, 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
Being tested: NUV001
Keywords: safety, tolerance, SS genotype of sickle cell disease
Medical subject headings: Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NUV001Dietary supplement
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

Study groups in NCT05789355
Study group	Role	What they receive
NUV001	Experimental	Daily supplementation with NUV001 1000 mg Receives: Dietary Supplement: NUV001

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of adverse events and treatment-emergent adverse events through Day 120.
Participant incidence of AEs/TEAEs, including vaso-occlusive crises and SCD-related hospitalizations, from first dose through Day 120.
Measured: First dose through Day 120.
Clinically significant changes in laboratory safety parameters through Day 120.
Clinically significant changes from baseline through Day 120 in hematology, CRP, liver function tests, renal function tests, CPK, electrolytes, fasting glucose, and albumin.
Measured: Baseline through Day 120.
Clinically significant changes in vital signs through Day 120.
Clinically significant changes from baseline through Day 120 in systolic blood pressure, diastolic blood pressure, pulse rate, body temperature, and body weight if retained as part of tolerability assessment.
Measured: Baseline through Day 120.

Secondary outcomes (10)

Change from baseline in hematologic parameters through Day 120.
Change from baseline through Day 120 in Red Blood Cell count, Hemoglobin, Hematocrit, Mean Corpuscular Volume, Mean Corpuscular Hemoglobin, Mean Corpuscular Hemoglobin Concentration, and Reticulocyte count.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in markers of hemolysis through Day 120.
Change from baseline through Day 120 in Lactate DeHydrogenase and Bilirubin parameters as markers of hemolysis.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in Fetal Hemoglobin-related erythroid parameters through Day 120.
Change from baseline through Day 120 in percentage of F-cells, percentage of F-reticulocytes, and distribution of hemoglobin F expression within F-cells.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in sickling-related Red Blood Cell parameters through Day 120.
Change from baseline through Day 120 in percentage of irreversibly sickled cells and in vitro hypoxia-induced Red Blood Cell sickling.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in blood β-NMN and NAD+ concentrations through Day 120.
Change from baseline through Day 120 in whole blood β-Nicotinamide mononucleotide (β-NMN) and nicotinamide adenine dinucleotide (NAD+) concentrations.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in plasma NAD-related metabolites through Day 120.
Change from baseline through Day 120 in plasma nicotinamide (NAM) and 1-methylnicotinamide (MeNAM) concentrations.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in urinary NAD-related metabolites through Day 120.
Change from baseline through Day 120 in urinary 1-methylnicotinamide (MeNAM) and 2PY concentrations.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in health-related quality of life through Day 120.
Change from baseline through Day 120 in Short Form-36 (SF-36) questionnaire domain scores and summary scores.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, and Day 120.
Change from baseline in pain outcomes through Day 120.
Change from baseline through Day 120 in pain severity and pain interference scores assessed using the Brief Pain Inventory questionnaire.
Measured: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, and Day 120
Change from baseline in analgesic use through Day 120.
Change from baseline through Day 120 in use of analgesic and opioid medications, assessed using recorded concomitant medication use and equianalgesic conversion where applicable.
Measured: Baseline (Day 0) through Day 120.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

30 Mar 20236 May 2024

First posted29 Mar 2023
Study start30 Mar 2023
Primary completion6 May 2024
Study completion6 May 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Prospective single-arm, open-label and monocentric pilot study.
Masking: None (open label)
Enrolment: 12 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05789355
Sponsor's protocol ID: Safety-DR-NUV001-SCD
Other IDs: 2022-A01570-43
Status last verified: Jun 2026
First submitted: 28 Feb 2023
Last updated: 17 Jun 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

All Sickle Cell Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: EFFECT OF NUV001 SUPPLEMENTATION FOR 120 DAYS IN PATIENTS SUFFERING FROM SICKLE CELL DISEASE (SCD) SS GENOTYPE: A PILOT STUDY
ClinicalTrials.gov ID: NCT05789355
Acronym / sponsor ID: Safety-DR-NUV001-SCD
Conditions: Sickle Cell Disease
Keywords: safety, tolerance, SS genotype of sickle cell disease
Study type: Interventional
Recruitment status: Completed
Enrolment: 12 participants (actual)
Lead sponsor: LGDIndustry (pharma, biotech, devices)
Collaborators: Assistance Publique Hopitaux De Marseille, Etablissement Français du Sang, CEN Biotech
Study start: 30 Mar 2023 (actual)
Primary completion: 6 May 2024 (actual)
Study completion: 6 May 2024 (actual)
First posted: 29 Mar 2023 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Prospective single-arm, open-label and monocentric pilot study.

Arm	Type	Receives
NUV001	Experimental	Dietary Supplement: NUV001Daily supplementation with NUV001 1000 mg

Who may qualify

Ages: 18 to 60 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (11 listed)

Participants meeting the following criteria could be included:
Male or female between 18 and 60 years old.
Females of childbearing potential should be using one of the following acceptable methods of birth control:
Patients whose weight is greater than 50 kg.
Patients diagnosed with homozygous sickle cell anemia of SS genotype (documented by genotyping).

Key exclusion criteria (10 listed)

Participants meeting the following criteria could not be included:
Patients with known or suspected allergies to any ingredient of the food supplement (β-NMN, Isomalt, Magnesium stearate, microcrystalline cellulose).
Patients who have consumed food supplements containing tryptophan, glutamine or vitamin B3 in various forms (nicotinic acid/niacin and nicotinamide) during the month before selection.
Patients have a significant medical condition that required hospitalization (other than sickle cell crisis) within two months of the screening visit (Visit 0).
Patients have serum albumin < 3.0 g/dl (< 30 g/L).

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
NUV001Daily 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	Dietary supplement	NUV001

Primary outcome

Incidence of adverse events and treatment-emergent adverse events through Day 120.
Time frame: First dose through Day 120.
Participant incidence of AEs/TEAEs, including vaso-occlusive crises and SCD-related hospitalizations, from first dose through Day 120.
Clinically significant changes in laboratory safety parameters through Day 120.
Time frame: Baseline through Day 120.
Clinically significant changes from baseline through Day 120 in hematology, CRP, liver function tests, renal function tests, CPK, electrolytes, fasting glucose, and albumin.
Clinically significant changes in vital signs through Day 120.
Time frame: Baseline through Day 120.
Clinically significant changes from baseline through Day 120 in systolic blood pressure, diastolic blood pressure, pulse rate, body temperature, and body weight if retained as part of tolerability assessment.

10 secondary outcomes

Change from baseline in hematologic parameters through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in markers of hemolysis through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in Fetal Hemoglobin-related erythroid parameters through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in sickling-related Red Blood Cell parameters through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in blood β-NMN and NAD+ concentrations through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in plasma NAD-related metabolites through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in urinary NAD-related metabolites through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, Day 120.
Change from baseline in health-related quality of life through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, and Day 120.
Change from baseline in pain outcomes through Day 120.
Time frame: Baseline (Day 0), Day 15, Day 30, Day 60, Day 90, and Day 120
Change from baseline in analgesic use through Day 120.
Time frame: Baseline (Day 0) through Day 120.

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2026.
Individual participant dataSharing planned: No

Related trials for Sickle Cell Disease

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Matthias CANAULT, PhD-HDR study director

LGD

Estelle JEAN, MD principal investigator

Assistance Publique Hopitaux Marseille

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A study testing NUV001 in people with Sickle Cell Disease, run by LGD at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events and treatment-emergent adverse events through Day 120. (time frame: First dose through Day 120.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05789355.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jun 2026.