Telomere DiseasePhase 2NCT03312400
Low-Dose Danazol for the Treatment of Telomere Related Diseases
A Phase 2 study testing Danazol in people with Telomere Disease, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Background: DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Read more about this study
- Who is it for?
- Ages 3 to 99 years
- Conditions the study is for
- Telomere Disease
- What is being tested?
- Danazol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Feb 2018to 31 Oct 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age-adjusted telomere length less than or equal to the first percentile by flow-FISH method. In patients with a known pathogenic or likely pathogenic mutation in a telomere maintenance gene, age adjusted telomere length less than or equal to the 10th percentile is sufficient.
- A mutation in telomere maintenance genes (TERT, TERC, DKC1, TINF2, NHP2, NOP10, WRAP53, TERF2, PARN, RTEL1, ACD, CTC1, USB1) as tested in a CLIA (or international equivalent) certified laboratory
- Age greater than or equal to 3 years
- Weight greater than or equal to 12 Kg
- AND
- At least one of the following criteria:
Show all 13Show fewer
- Anemia with a hemoglobin less than or equal to 10 g/dL without red blood cell transfusion
- Thrombocytopenia with a platelet count less than or equal to 50,000/microliter without transfusion
- Neutropenia with an absolute neutrophil count less than or equal to 1,000/ microliter
- OR
- Pulmonary fibrosis diagnosed by either a lung biopsy or computed tomography scan of the chest according to guidelines from the American Thoracic Society and European Respiratory Society.
- OR
- Hepatic fibrosis diagnosed by Transient Elastography by Fibroscan value greater than 10 kpa or US evidence of cirrhotic liver or splenomegaly, or transjugular liver biopsy demonstrating fibrosis.
Who cannot join
- Patients on androgen hormones to include testosterone or high dose estrogen (estradiol 0.5 mg/day or greater) for the12 months prior to enrollment
- Patients with active thrombosis or thromboembolic disease and history of such events, undiagnosed abnormal genital bleeding, porphyria, androgendependent tumor, or prostatic hypertrophy
- Patients with pulmonary fibrosis who are receiving anti-fibrotic drug treatment, such as pirfenidone or nintedanib unless stable on anti-fibrotic drug for at least 6 months prior to starting on danazol as demonstrated by PFTs.
- Patients with active hepatitis B or C
- Patients who have received a bone marrow transplant
- Patient with other hereditary bone marrow failure syndromes such as Fanconi anemia or Diamond Blackfan anemia
Show all 13Show fewer
- Patients with infections not adequately responding to appropriate therapy
- Current pregnancy, or unwillingness to take oral contraceptives or use the barrier methods of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of the study
- Lactating women, due to the potentially harmful effects on the nursing child
- Patients with cancer who are actively receiving systemic chemotherapeutic treatment or who take drugs with hematological effects
- Patients with decompensated liver disease to include persistent ascites, encephalopathy, variceal hemorrhage, or MELD score of 10 or greater
- Inability to understand the investigational nature of the study or to give informed consent or without a legally authorized representative or surrogate that can provide informed consent
- Inability to swallow a capsule
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Low Dose Danazol for the Treatment of Telomere Related Diseases
Background:
DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers want to see if low doses of the hormone drug danazol can help.
Read more
Objective:
To study the safety and effect of low dose danazol.
Eligibility:
People ages 3 and older with a telomere disease who have either very short telomeres and a specific gene change. They must also show signs of aplastic anemia, lung, or liver disease.
Design:
Participants will be screened in another protocol.
Participants will have:
- Medical history
- Physical exam
- Blood tests
- Lung exam. They will breathe into an instrument that records the amount and rate of air breathed in and out over a period of time. 6-minute walking test.
- Abdominal ultrasound and liver scan. These tests use sound waves to measure the fibrosis in the liver.
Some participants will have:
- Pregnancy test
- Small sample of the liver removed
- Bone marrow biopsy. The bone will be numbed and a small needle will take a sample of the marrow.
All participants will have hormone levels checked.
All child participants will see a pediatric endocrinologist. Children may need to have a hand x-ray.
We will monitor patients for 6 months before starting danazol.
Participants will take danazol by mouth twice a day for 1 year.
Participants must return to the clinic at 6 months and 12 months while on danazol and 6 months after stopping it. They will have blood and urine tests, a lung exam, abdominal ultrasound, and liver scan.
Read the detailed description
Telomere disease is caused by accelerated telomere attrition and results in multi-organ dysfunction. Telomeres are nucleotide repeats of non-coding DNA at the end of the chromosomes which function as protective caps to prevent erosion of genomic DNA during cell division and to protect chromosomes from recognition as single stranded DNA. Telomeric DNA is elongated by the telomerase complex, which is comprised of a reverse transcriptase catalytic subunit (encoded by TERT), an RNA template (encoded by TERC) and associated proteins.
Telomerase activity is crucial in maintaining telomere length in cells with a high proliferative capacity, such as hematopoietic stem cells (HSCs) and lymphocytes. Presentation of telomeropathies can vary from severe aplastic anemia (SAA) and dyskeratosis congenital (DKC) early in childhood, to pulmonary or hepatic fibrosis later in life. There is no standard of care for the treatment of telomere disease.
Considerable evidence suggests that sex hormones regulate telomerase. Calado et al. demonstrated that human lymphocytes and CD34+ hematopoietic cells up regulate both TERT gene expression and telomerase enzymatic activity in response to androgens in vitro. A recent observational cohort study demonstrated hematologic response in 14 of 16 pediatric patients with DKC treated with androgens. In a prospective trial from our Branch, Townsley et al demonstrated that patients with telomere diseases who were treated with the synthetic sex hormone danazol showed telomere elongation, and hematologic response were seen in 79% of patients after only three months of treatment. This study used the highest dose of danazol, 800 mg daily, and known adverse effects, such as elevated liver enzyme levels and muscle cramps, occurred in 41% and 33% of patients, respectively. Overall the treatment was well tolerated, but some patients did require dose reduction. After 27 patients were enrolled, the study was halted early, because telomere attrition was reduced in all 12 patients who could be evaluated for the primary endpoint. Because of the limited power, we were unable to draw definitive conclusions regarding further clinical effect of danazol but stabilization or improvement was observed in a few cases in other organ function, measured by DLCO for pulmonary fibrosis and Fibroscan for cirrhosis.
We now propose a phase II study designed to determine the efficacy of low dose danazol in decreasing the rate of telomere attrition in subjects with a short age-adjusted telomere length. The secondary aim is to determine the clinical effect of this therapy in conditions that are related to short telomeres, to include cytopenia(s), pulmonary fibrosis, and/or hepatic fibrosis.
- Conditions
- Telomere Disease
- Being tested
- Danazol
- Keywords
- Telomeres, Aplastic Anemia, Pulmonary Fibrosis, Androgens, Hepatic Fibrosis
- Medical subject headings
- Anemia, Aplastic, Pulmonary Fibrosis, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DanazolDrug
Participants will be randomized to one of two treatment arms, and after 6 months of treatment they will switch to the other treatment arm. One arm will be danazol 100 mg twice a day, the other arm will be danazol 200 mg twice a day. Every patient will receive 6 months danazol 200 mg/day and 6 months 400 mg/day, thereby completing 12 months of danazol treatment. Pediatric participants less than 50 kg will be dosed at 4 or 8 mg/kg/day not to exceed 200 or 400 mg/day, respectively.
| Study group | Role | What they receive |
|---|---|---|
| Danazol 200 mg then Danazol 400 mg | Active comparator | Participants will receive danazol 100 mg twice daily for the first 24 weeks. At Week 24, the dose will be increased to 200 mg twice daily. Receives: Drug: Danazol |
| Danazol 400 mg then Danazol 200 mg | Active comparator | Participants will receive danazol 200 mg twice daily for the first 24 weeks. At Week 24, the dose will be reduced to 100 mg twice daily. Receives: Drug: Danazol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With a Reduction in Telomere Attrition Rate at 6 Months
Telomere attrition rate will be compared between participants receiving danazol 400 mg and 200 mg.
A reduction in telomere attrition rate is defined as a ≥50% reduction from baseline.
Measured: Baseline, 24 Weeks
Secondary outcomes (4)
Hematologic Response
Response is if on at least two serial measures one week apart:
- Participant with severe pancytopenia, defined as Absolute neutrophil count (ANC) <500/μL , platelet count <20,000/μL, and reticulocyte count <20, 000, will be considered to have a response when criteria is no longer for at least 2 of the following criteria: Absolute neutrophil count >500/μL; Platelet count > 20,000/μL; Reticulocyte count > 60,000/μL
- Participants with moderate pancytopenia, or those not qualified as severe, response is defined as improvement in one or more of the below:
- If baseline ANC is below 500/μL, increase in ANC greater or equal to 300/μL
- If baseline ANC is above 500/μL, increase in ANC greater or equal to 500/μL
- If platelet response is increased by greater or equal to 20,000/μL
- If any increase in hemoglobin by 1.5 g/dL of blood in transfusion independent participants and in absolute reticulocyte count to greater or equal to 60,000 /μL of blood in transfusion-dependent patients
Measured: 6 and 12 months
Pulmonary Function Testing Response in Participants With Pulmonary Fibrosis
Defined as a positive response of the rate of Forced vital capacity (FVC) decline on drug is 5% less than baseline rate of decline.
Measured: After 6 and 12 months of treatment
Hepatic Response in Participants With Hepatic Fibrosis
Defined as improvement in one or more of the listed parameters:
- Reduction in transient elastography by Fibroscan of greater or equal to 20% OR
- Reduction in hepatic venous portal gradient by greater or equal to 20% in subjects who had liver biopsy for significant liver disease. OR
- Reduction in transient elastography by Acoustic Radiation Force Impulse imaging greater or equal to 20%.
Measured: After 6 and 12 months of treatment
Proportion of Adverse Events, Including Serious Adverse Events (SAEs) and Treatment Related SAEs
Proportion of adverse events, including serious adverse events (SAEs) and treatment related SAEs using Common Terminology Criteria for Adverse Events (CTCAE) v4.0. CTCAE grades side effects from 1 to 5: mild, moderate, severe, life-threatening, or fatal.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2017
- Study start8 Feb 2018
- Primary completion31 Oct 2025
- Study completion31 Oct 2025
Why it stopped: Study was stopped due to poor accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Telomere DiseaseStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Bhavisha Patel, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03312400
- Sponsor's protocol ID
- 180004
- Other IDs
- 18-H-0004
- Status last verified
- Oct 2026
- First submitted
- 14 Oct 2017
- Last updated
- 27 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD used in the results publication will be shared
Publications
Calado RT, Yewdell WT, Wilkerson KL, Regal JA, Kajigaya S, Stratakis CA, Young NS. Sex hormones, acting on the TERT gene, increase telomerase activity in human primary hematopoietic cells. Blood. 2009 Sep 10;114(11):2236-43. doi: 10.1182/blood-2008-09-178871. Epub 2009 Jun 26.
PubMed 19561322Townsley DM, Dumitriu B, Liu D, Biancotto A, Weinstein B, Chen C, Hardy N, Mihalek AD, Lingala S, Kim YJ, Yao J, Jones E, Gochuico BR, Heller T, Wu CO, Calado RT, Scheinberg P, Young NS. Danazol Treatment for Telomere Diseases. N Engl J Med. 2016 May 19;374(20):1922-31. doi: 10.1056/NEJMoa1515319.
PubMed 27192671Khincha PP, Wentzensen IM, Giri N, Alter BP, Savage SA. Response to androgen therapy in patients with dyskeratosis congenita. Br J Haematol. 2014 May;165(3):349-57. doi: 10.1111/bjh.12748. Epub 2014 Feb 12.
PubMed 24666134
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Low Dose Danazol for the Treatment of Telomere Related Diseases
- ClinicalTrials.gov ID
- NCT03312400
- Acronym / sponsor ID
- 180004
- Conditions
- Telomere Disease
- Keywords
- Telomeres, Aplastic Anemia, Pulmonary Fibrosis, Androgens, Hepatic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was stopped due to poor accrual
- Enrolment
- 18 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 8 Feb 2018 (actual)
- Primary completion
- 31 Oct 2025 (actual)
- Study completion
- 31 Oct 2025 (actual)
- First posted
- 17 Oct 2017 (actual)
- Results
- Posted 27 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers want to see if low doses of the hormone drug danazol can help.
Objective:
To study the safety and effect of low dose danazol.
Eligibility:
People ages 3 and older with a telomere disease who have either very short telomeres and a specific gene change. They must also show signs of aplastic anemia, lung, or liver disease.
Design:
Participants will be screened in another protocol.
Participants will have:
- Medical history
- Physical exam
- Blood tests
- Lung exam. They will breathe into an instrument that records the amount and rate of air breathed in and out over a period of time. 6-minute walking test.
- Abdominal ultrasound and liver scan. These tests use sound waves to measure the fibrosis in the liver.
Some participants will have:
- Pregnancy test
- Small sample of the liver removed
- Bone marrow biopsy. The bone will be numbed and a small needle will take a sample of the marrow.
All participants will have hormone levels checked.
All child participants will see a pediatric endocrinologist. Children may need to have a hand x-ray.
We will monitor patients for 6 months before starting danazol.
Participants will take danazol by mouth twice a day for 1 year.
Participants must return to the clinic at 6 months and 12 months while on danazol and 6 months after stopping it. They will have blood and urine tests, a lung exam, abdominal ultrasound, and liver scan.
Detailed description
Telomere disease is caused by accelerated telomere attrition and results in multi-organ dysfunction. Telomeres are nucleotide repeats of non-coding DNA at the end of the chromosomes which function as protective caps to prevent erosion of genomic DNA during cell division and to protect chromosomes from recognition as single stranded DNA. Telomeric DNA is elongated by the telomerase complex, which is comprised of a reverse transcriptase catalytic subunit (encoded by TERT), an RNA template (encoded by TERC) and associated proteins.
Telomerase activity is crucial in maintaining telomere length in cells with a high proliferative capacity, such as hematopoietic stem cells (HSCs) and lymphocytes. Presentation of telomeropathies can vary from severe aplastic anemia (SAA) and dyskeratosis congenital (DKC) early in childhood, to pulmonary or hepatic fibrosis later in life. There is no standard of care for the treatment of telomere disease.
Considerable evidence suggests that sex hormones regulate telomerase. Calado et al. demonstrated that human lymphocytes and CD34+ hematopoietic cells up regulate both TERT gene expression and telomerase enzymatic activity in response to androgens in vitro. A recent observational cohort study demonstrated hematologic response in 14 of 16 pediatric patients with DKC treated with androgens. In a prospective trial from our Branch, Townsley et al demonstrated that patients with telomere diseases who were treated with the synthetic sex hormone danazol showed telomere elongation, and hematologic response were seen in 79% of patients after only three months of treatment. This study used the highest dose of danazol, 800 mg daily, and known adverse effects, such as elevated liver enzyme levels and muscle cramps, occurred in 41% and 33% of patients, respectively. Overall the treatment was well tolerated, but some patients did require dose reduction. After 27 patients were enrolled, the study was halted early, because telomere attrition was reduced in all 12 patients who could be evaluated for the primary endpoint. Because of the limited power, we were unable to draw definitive conclusions regarding further clinical effect of danazol but stabilization or improvement was observed in a few cases in other organ function, measured by DLCO for pulmonary fibrosis and Fibroscan for cirrhosis.
We now propose a phase II study designed to determine the efficacy of low dose danazol in decreasing the rate of telomere attrition in subjects with a short age-adjusted telomere length. The secondary aim is to determine the clinical effect of this therapy in conditions that are related to short telomeres, to include cytopenia(s), pulmonary fibrosis, and/or hepatic fibrosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Danazol 200 mg then Danazol 400 mg | Active comparator | Drug: DanazolParticipants will receive danazol 100 mg twice daily for the first 24 weeks. At Week 24, the dose will be increased to 200 mg twice daily. |
| Danazol 400 mg then Danazol 200 mg | Active comparator | Drug: DanazolParticipants will receive danazol 200 mg twice daily for the first 24 weeks. At Week 24, the dose will be reduced to 100 mg twice daily. |
Who may qualify
- Ages
- 3 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- AND
- OR
- Pulmonary fibrosis diagnosed by either a lung biopsy or computed tomography scan of the chest according to guidelines from the American Thoracic Society and European Respiratory Society.
- OR
Key exclusion criteria (13 listed)
- Patients on androgen hormones to include testosterone or high dose estrogen (estradiol 0.5 mg/day or greater) for the12 months prior to enrollment
- Patients with active thrombosis or thromboembolic disease and history of such events, undiagnosed abnormal genital bleeding, porphyria, androgendependent tumor, or prostatic hypertrophy
- Patients with pulmonary fibrosis who are receiving anti-fibrotic drug treatment, such as pirfenidone or nintedanib unless stable on anti-fibrotic drug for at least 6 months prior to starting on danazol as demonstrated by PFTs.
- Patients with active hepatitis B or C
- Patients who have received a bone marrow transplant
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 |
|---|---|---|
| DanazolParticipants will be randomized to one of two treatment arms, and after 6 months of treatment they will switch to the other treatment arm. One arm will be danazol 100 mg twice a day, the other arm will be danazol 200 mg twice a day. Every patient will receive 6 months danazol 200 mg/day and 6 months 400 mg/day, thereby completing 12 months of danazol treatment. Pediatric participants less than 50 kg will be dosed at 4 or 8 mg/kg/day not to exceed 200 or 400 mg/day, respectively. | Drug | Danazol 200 mg then Danazol 400 mg, Danazol 400 mg then Danazol 200 mg |
Primary outcome
Number of Participants With a Reduction in Telomere Attrition Rate at 6 Months
Time frame: Baseline, 24 Weeks
Telomere attrition rate will be compared between participants receiving danazol 400 mg and 200 mg. A reduction in telomere attrition rate is defined as a ≥50% reduction from baseline.
4 secondary outcomes
Hematologic Response
Time frame: 6 and 12 months
Pulmonary Function Testing Response in Participants With Pulmonary Fibrosis
Time frame: After 6 and 12 months of treatment
Hepatic Response in Participants With Hepatic Fibrosis
Time frame: After 6 and 12 months of treatment
Proportion of Adverse Events, Including Serious Adverse Events (SAEs) and Treatment Related SAEs
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 19561322Calado RT, Yewdell WT, Wilkerson KL, Regal JA, Kajigaya S, Stratakis CA, Young NS. Sex hormones, acting on the TERT gene, increase telomerase activity in human primary hematopoietic cells. Blood. 2009 Sep 10;114(11):2236-43. doi: 10.1182/blood-2008-09-178871. Epub 2009 Jun 26.
- Background: PubMed 27192671Townsley DM, Dumitriu B, Liu D, Biancotto A, Weinstein B, Chen C, Hardy N, Mihalek AD, Lingala S, Kim YJ, Yao J, Jones E, Gochuico BR, Heller T, Wu CO, Calado RT, Scheinberg P, Young NS. Danazol Treatment for Telomere Diseases. N Engl J Med. 2016 May 19;374(20):1922-31. doi: 10.1056/NEJMoa1515319.
- Background: PubMed 24666134Khincha PP, Wentzensen IM, Giri N, Alter BP, Savage SA. Response to androgen therapy in patients with dyskeratosis congenita. Br J Haematol. 2014 May;165(3):349-57. doi: 10.1111/bjh.12748. Epub 2014 Feb 12.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. IPD used in the results publication will be shared
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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.
Bhavisha Patel, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Danazol in people with Telomere Disease, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
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 13 inclusion and 13 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: stopped early, as of the sponsor's last update on 27 Oct 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 United States. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With a Reduction in Telomere Attrition Rate at 6 Months (time frame: Baseline, 24 Weeks). 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 NCT03312400, which also lists 3 linked publications.
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 27 Oct 2026.