Autosomal Dominant Polycystic KidneyREORIENTEDNCT05646420
Thyroid Hormones in ADPKD
A study testing Blood sampling in people with Autosomal Dominant Polycystic Kidney, run by Mario Negri Institute for Pharmacological Research at 1 site in Italy.
Trial summary
Autosomal dominant polycystic kidney disease (ADPKD) is a monogenic, rare and life-threatening disease, characterized by the pathological formation of multiple fluid-filled cysts that arise from renal tubules and alter kidney architecture and function. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney
- What is being tested?
- Blood sampling
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 91 participants
- Where
- 1 siteItaly
- Study dates
- 1 Feb 2023to 25 Mar 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male and female ≥18 years old;
- Diagnosis of ADPKD based on renal ultrasonography or genetic test;
- Written informed consent
Who cannot join
- Diagnosis of Hashimoto's disease, hyperthyroidism or pituitary disease or any other condition undergoing levothyroxine replacement
- Patient with hypothyroidism treated with drug therapy
- Active treatment with Tolvaptan and/or Octreotide-LAR;
- Regular treatment with amiodarone, lithium, interferon or immunosuppressive drugs including steroids;
- Active malignancy or acute or chronic inflammatory disease, HIV;
- Dialysis or kidney transplantation;
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- Diabetes mellitus;
- Hypocaloric diet or current dietary approaches to obtain weight loss.
- Legal incapacity or any evidence that the patient will not be able to understand the study aims and procedures.
Trial locations and contacts
1 site in 1 country.
Italy1 site
Centro di Ricerche Cliniche per le Malattie Rare "Aldo e Cele Daccò"
Ranica, BG, 24020
About this study
Official title: Deciphering the Role of Thyroid Hormones in Autosomal Dominant Polycystic Kidney Disease
Autosomal dominant polycystic kidney disease (ADPKD) is a monogenic, rare and life-threatening disease, characterized by the pathological formation of multiple fluid-filled cysts that arise from renal tubules and alter kidney architecture and function. In most patients, the progressive deterioration of renal function ultimately leads to end-stage kidney disease (ESKD) and the need for dialysis or kidney transplantation.
Save the conventional anti-hypertensive strategies, there are currently two disease-specific treatments for ADPKD (Tolvaptan and Octreotide-LAR). However, these drugs are only available to patients at high risk of progression to ESKD, while a remarkable number of ADPKD patients progress to ESKD despite the treatments.
Read more
Cyst formation in ADPKD is determined by mutations in two genes encoding two transmembrane proteins: polycystin1 and polycystin2. The pathogenesis of the disease involves a series of phenotypic alterations, including the de-differentiation of epithelial cells, uncontrolled proliferation and abnormal secretion of fluids in the cysts, metabolic remodeling, all phenomena that lead to the progressive loss of renal structure and function . Therefore, to try to investigate the mechanisms of the disease, the investigators should go in search of pleiotropic molecules capable of simultaneously modulating structure, function and metabolism.
Research done so far suggests that thyroid hormones (TH) may also act as pleiotropic modulators in the patho-biology of ADPKD. TH signals play a crucial role in the regulation of cell de-differentiation and cell cycle reactivation, as well as in the metabolism and evolution of cardiac and renal diseases. Interestingly, changes in TH levels have been detected in approximately 80% of patients with chronic renal failure (CKD), whereas patients with ADPKD show a higher incidence of clinical and subclinical hypothyroidism. Despite these evidences, the ability of TH to modulate anti-cystogenic and renoprotective processes in ADPKD has not yet been studied.
The objective of this study is to determine the levels of THs in the serum of ADPKD patients with normal renal function and mild, moderate or severe renal dysfunction, and to correlate them with renal functional parameters.
- Conditions
- Autosomal Dominant Polycystic Kidney
- Being tested
- Blood sampling
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood samplingOther
A blood sample of 15 ml will be collected for each patient.
| Study group | Role | What they receive |
|---|---|---|
| ADPKD patients | Experimental | The study will include 90 patients with diagnosis of ADPKD based on renal ultrasonography findings or genetic test.
Specifically, five groups of patients will be identified according to KDIGO classification:
* 18 subjects with normal or high renal function: eGFR ≥90 ml/min/1.73m2 - CKD G1 stage
* 18 subjects with mildly decreased renal function: eGFR 89-60 ml/min/1.73m2 - CKD G2 stage
* 18 subjects with mildly to moderately decreased renal function: eGFR 59 to 45 ml/min/1.73m2 - CKD G3a stage
* 18 subjects with moderately to severely decreased renal function: eGFR 44 to 30 ml/min/1.73m2 - CKD G3b stage
* 18 subjects with severely decreased renal function: eGFR 29 to 15 ml/min/1.73m2 - CKD G4 stage. Receives: Other: Blood sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum levels of total and free triiodothyronine (T3).
Measured: Once during the study.
Serum levels of total and free L-thyroxine (T4).
Measured: Once during the study.
Serum levels of total and free thyroid stimulating hormone (TSH).
Measured: Once during the study.
Serum levels of total and free reverse T3 (rT3).
Measured: Once during the study.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2022
- Study start1 Feb 2023
- Primary completion16 Feb 2024
- Study completion25 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autosomal Dominant Polycystic KidneyStudies 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 91 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mario Negri Institute for Pharmacological Research
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05646420
- Sponsor's protocol ID
- REORIENTED
- Acronym
- REORIENTED
- Status last verified
- Mar 2024
- First submitted
- 5 Dec 2022
- Last updated
- 28 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lavecchia AM, Locatelli L, Trillini M, Del Vecchio A, Cerullo D, Villa A, Peracchi T, Brizi V, Butto S, Corna D, Brunelli L, Guarinoni C, Rubis N, Remuzzi G, Xinaris C. Thyroid Hormones Exhibit Promising Prognostic and Therapeutic Potential in Autosomal Dominant Polycystic Kidney Disease. Thyroid. 2026 Aug;36(8):840-852. doi: 10.1177/10507256261460200. Epub 2026 Jun 15.
PubMed 42296046
Study at a glance
From the registry record, last updated by the sponsor on 28 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Deciphering the Role of Thyroid Hormones in Autosomal Dominant Polycystic Kidney Disease
- ClinicalTrials.gov ID
- NCT05646420
- Acronym / sponsor ID
- REORIENTED / REORIENTED
- Conditions
- Autosomal Dominant Polycystic Kidney
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 91 participants (actual)
- Lead sponsor
- Mario Negri Institute for Pharmacological ResearchUniversity, hospital or other
- Study start
- 1 Feb 2023 (actual)
- Primary completion
- 16 Feb 2024 (actual)
- Study completion
- 25 Mar 2024 (actual)
- First posted
- 12 Dec 2022 (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.
Autosomal dominant polycystic kidney disease (ADPKD) is a monogenic, rare and life-threatening disease, characterized by the pathological formation of multiple fluid-filled cysts that arise from renal tubules and alter kidney architecture and function. In most patients, the progressive deterioration of renal function ultimately leads to end-stage kidney disease (ESKD) and the need for dialysis or kidney transplantation.
Save the conventional anti-hypertensive strategies, there are currently two disease-specific treatments for ADPKD (Tolvaptan and Octreotide-LAR). However, these drugs are only available to patients at high risk of progression to ESKD, while a remarkable number of ADPKD patients progress to ESKD despite the treatments.
Cyst formation in ADPKD is determined by mutations in two genes encoding two transmembrane proteins: polycystin1 and polycystin2. The pathogenesis of the disease involves a series of phenotypic alterations, including the de-differentiation of epithelial cells, uncontrolled proliferation and abnormal secretion of fluids in the cysts, metabolic remodeling, all phenomena that lead to the progressive loss of renal structure and function . Therefore, to try to investigate the mechanisms of the disease, the investigators should go in search of pleiotropic molecules capable of simultaneously modulating structure, function and metabolism.
Research done so far suggests that thyroid hormones (TH) may also act as pleiotropic modulators in the patho-biology of ADPKD. TH signals play a crucial role in the regulation of cell de-differentiation and cell cycle reactivation, as well as in the metabolism and evolution of cardiac and renal diseases. Interestingly, changes in TH levels have been detected in approximately 80% of patients with chronic renal failure (CKD), whereas patients with ADPKD show a higher incidence of clinical and subclinical hypothyroidism. Despite these evidences, the ability of TH to modulate anti-cystogenic and renoprotective processes in ADPKD has not yet been studied.
The objective of this study is to determine the levels of THs in the serum of ADPKD patients with normal renal function and mild, moderate or severe renal dysfunction, and to correlate them with renal functional parameters.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ADPKD patients | Experimental | Other: Blood samplingThe study will include 90 patients with diagnosis of ADPKD based on renal ultrasonography findings or genetic test. Specifically, five groups of patients will be identified according to KDIGO classification: * 18 subjects with normal or high renal function: eGFR ≥90 ml/min/1.73m2 - CKD G1 stage * 18 subjects with mildly decreased renal function: eGFR 89-60 ml/min/1.73m2 - CKD G2 stage * 18 subjects with mildly to moderately decreased renal function: eGFR 59 to 45 ml/min/1.73m2 - CKD G3a stage * 18 subjects with moderately to severely decreased renal function: eGFR 44 to 30 ml/min/1.73m2 - CKD G3b stage * 18 subjects with severely decreased renal function: eGFR 29 to 15 ml/min/1.73m2 - CKD G4 stage. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male and female ≥18 years old;
- Diagnosis of ADPKD based on renal ultrasonography or genetic test;
- Written informed consent
Key exclusion criteria (9 listed)
- Diagnosis of Hashimoto's disease, hyperthyroidism or pituitary disease or any other condition undergoing levothyroxine replacement
- Patient with hypothyroidism treated with drug therapy
- Active treatment with Tolvaptan and/or Octreotide-LAR;
- Regular treatment with amiodarone, lithium, interferon or immunosuppressive drugs including steroids;
- Active malignancy or acute or chronic inflammatory disease, HIV;
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 |
|---|---|---|
| Blood samplingA blood sample of 15 ml will be collected for each patient. | Other | ADPKD patients |
Primary outcome
Serum levels of total and free triiodothyronine (T3).
Time frame: Once during the study.
Serum levels of total and free L-thyroxine (T4).
Time frame: Once during the study.
Serum levels of total and free thyroid stimulating hormone (TSH).
Time frame: Once during the study.
Serum levels of total and free reverse T3 (rT3).
Time frame: Once during the study.
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 28 Mar 2024.
- Derived from this study: PubMed 42296046Lavecchia AM, Locatelli L, Trillini M, Del Vecchio A, Cerullo D, Villa A, Peracchi T, Brizi V, Butto S, Corna D, Brunelli L, Guarinoni C, Rubis N, Remuzzi G, Xinaris C. Thyroid Hormones Exhibit Promising Prognostic and Therapeutic Potential in Autosomal Dominant Polycystic Kidney Disease. Thyroid. 2026 Aug;36(8):840-852. doi: 10.1177/10507256261460200. Epub 2026 Jun 15.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Blood sampling in people with Autosomal Dominant Polycystic Kidney, run by Mario Negri Institute for Pharmacological Research at 1 site in Italy.
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 3 inclusion and 9 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 28 Mar 2024. 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 Italy. 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?
Serum levels of total and free triiodothyronine (T3). (time frame: Once during the study.). The study lists 4 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 NCT05646420, which also lists 1 linked publication.
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 28 Mar 2024.