CompletedPhase 1/2

Nandrolone Decanoate in the Treatment of Telomeropathies

Brazil20 participantsStarted 2014-02-01

Plain-language summary

Decrease in blood cell counts due to deficient bone marrow function, called bone marrow failure, as well as some lung diseases, called idiopathic pulmonary fibrosis, can be caused by genetic defects in telomere biology genes, eventually causing telomere erosion. These disorders are collectively termed "telomeropathies". There is evidence that male hormones may improve blood cell counts in marrow failure, and these hormones are able to stimulate telomerase function in hematopoietic cells in vitro. We propose this study to the use of male hormone in patients with aplastic anemia and pulmonary fibrosis associated with defects in telomeres.

Who can participate

Age range

2 Years – 99 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Peripheral blood leukocytes telomeres short for age, below the first percentile of a curve based on 500 healthy individuals between 0 and 100 years, with or without a telomerase gene mutation. AND * One or more of the following cytopenias: Anemia (symptoms of anemia with hemoglobin \<9.5 g/dL, or need for transfusion \> 2 units of packed red blood cells/month for at least two months, or absolute reticulocytes count \<60.000/μL). Thrombocytopenia (platelets counts \<30.000/μL or \<50.000/μL associated with bleeding, or megakaryocytes reduction in the bone marrow). Neutropenia (absolute neutrophil counts \<1.000/μL). OR * Idiopathic pulmonary fibrosis diagnosed according to the American Thoracic Society (ATS) criteria. Exclusion Criteria: * Terminal disease or liver disease, renal, cardiac, neurological, infectious or concomitant metabolic state whose gravity prevents the ability of the patient to tolerate the treatment protocol, or probable death within 30 days. * People with cancer who are undergoing chemotherapy. * Pregnancy, or desire to not prevent pregnancy in childbearing age. * Aplastic Anemia patients with indication for bone marrow transplantation and matched donor.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Reduction in telomere attrition

Timeframe: 2 years

Trial details

NCT IDNCT02055456

SponsorUniversity of Sao Paulo

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-02-01

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