A Phase 1/2 interventional study of Nandrolone Decanoate in Aplastic Anemia, Bone Marrow Failure Syndromes and Idiopathic Pulmonary Fibrosis, sponsored by University of Sao Paulo. Completed at 1 site in Brazil. Open to participants aged 2 Years to 99 Years. Per ClinicalTrials.gov, last updated 2023-08-31.
Sponsored by University of Sao Paulo · Phase 1/2, Interventional, and Treatment
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.
Telomeres are repeated nucleotide sequences of non-coding DNA at the ends of chromosomes that have protective functions and avoid chromosomes recombinations and fusions.
Loss-of-function mutations in genes of the telomerase complex, a enzyme responsible for maintaining telomere length, has been associated with bone marrow failures, notedly mutations in DKC1 gene, detected in a rare inherited form of marrow aplasia, called dyskeratosis congenita. These findings implicated telomerase dysfunction and shortening telomere length in failed hematopoiesis.
In family members of probands with aplastic anemia, marrow aplasia and telomerase mutations also have been observed and associated to varying degrees of cytopenias, IPF and/or cirrhosis. Moreover, patients with varying degrees of cytopenias, with significant family history for cytopenias, IPF and/or cirrhosis, have been identified with very short telomeres and some mutations in telomerase complex genes. Additionally, telomere length has been associated with human cancer.
In vitro studies suggest that telomere length could be modulated with sex hormones. Normal lymphocytes and human bone marrow progenitor cells exposed to androgens increased telomerase activity in vitro, and in individuals with telomerase mutations (TERT) androgens increased telomerase activity.This could be the explanation for the hematologic improvement observed in some aplastic anemia patients treated over 40 years ago with male hormones.
Therefore, we hypothesize that androgens therapy might modulate telomere attrition in vivo and ameliorate progression or reverse the clinical consequences of shortening telomere length, and we propose androgens therapy in patients with cytopenias and/or IPF with a short age adjusted telomere length, with or without telomerase gene mutations.
The primary biologic endpoint will be the reduction of telomere attrition over time compared to known rates of telomere erosion in normal individuals and in those who carry mutation in the telomerase genes. Secondary endpoints will be tolerability of nandrolone decanoate over two years, improvement in blood counts and/or pulmonary function.
371 studies on the registry are indexed under Anemia, Aplastic; 98 are open to participants now.
This study's enrollment of 20 is below the median of 37 across 281 interventional studies indexed under Anemia, Aplastic.
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AND
Anemia (symptoms of anemia with \<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 ( counts \<30.000/μL or \<50.000/μL associated with bleeding, or megakaryocytes reduction in the bone marrow).
Neutropenia ( \<1.000/μL).
OR
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Nandrolone Decanoate intramuscularly administered, every two weeks, 5 mg/kg/dose
Drug: Nandrolone Decanoate
Reduction in telomere attrition
The biologic endpoint is reduction in telomere attrition rate yearly compared to known rates of telomere erosion in normal individuals and in those who carry mutation in the telomerase genes
Time frame: 2 years
Hematologic response
The hematologic response will be determined by one or more of the following: 1. absolute neutrophil counts (increase of more than 500/μL above initial value) 2. platelets (increase of more than 20.000/μL above initial value) 3. Hemoglobin: * Increase in hemoglobin of more than 1.5 g/dL above initial value OR * Transfusion independence in transfusion-dependent patients (more than 2 months without transfusion) OR * Reduction of the transfusion needs in more than 50%
Time frame: 2 years
Clonal evolution
Number of participants that evolute to myelodysplasia or acute leukemia.
Time frame: 2 years
Improvement in lung function
The pulmonary response will be determined by the presence of one or more of the following: 1. Improvement of dyspnea severity, objectively evaluated by "Baseline Dyspnea Index"; 2. forced vital capacity (10% absolute increase) 3. Diffusion of carbon monoxide (DLCO) corrected for hemoglobin (15% increase) 4. No worsening of pulmonary fibrosis and reduction of assessed ground-glass opacities in computed tomography of the chest
Time frame: 2 years
Survival
Time frame: 2 years
Safety
Number of participants with adverse effects attributed to the use of nandrolone decanoate during the 24 months treatment period.
Time frame: 2 years
This study is completed, as verified in Aug 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Nandrolone Decanoate