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New treatment option for Telomere Biology Disorders
Official title Nucleoside Therapy in Patients With Telomere Biology Disorders
ClinicalTrials.gov ID: NCT06817590
What this study is testing
What is deoxycytidine?
deoxycytidine is an investigational medicine, given as an once-weekly pill taken by mouth, being studied as a potential treatment for telomere biology disorders.
Also referred to as dC.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The goal of this clinical trial is to learn if a combination therapy of deoxycytidine (dC) plus deoxythymidine (dT) is safe in patients with telomere biology disorders. The main questions it aims to answer are: Is the therapy safe with tolerable side effects in patients with telomere biology disorders?
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 70
You may be able to join if
- Age ≥ 1 year and ≤ 70 years
- Karnofsky performance status ≥ 50 for participants ≥16 years of age and Lansky performance status ≥ 50 for participants \<16 years of age
- Diagnosis requirement. Participants must meet at least one of the following requirements for a diagnosis of a telomere biology disorder:
- Age-adjusted mean telomere length \< 1%ile in peripheral blood lymphocytes by flow cytometry-fluorescence in situ hybridization (flow-FISH), as...
- Pathogenic or likely pathogenic variant(s) in one of the follow telomere biology associated genes: DKC1, TERC, TERT, NOP10, NHP2, WRAP53/TCAB1...
You likely can't join if
- Participants must not have very severe aplastic anemia necessitating bone marrow transplant at the time of enrollment. Very severe aplastic anemia is...
- Participants must not otherwise be expected to undergo bone marrow transplantation within 6 months of enrollment.
- Participants must not be taking concurrent medications intended to improve hematopoiesis such as androgens or growth factors, including granulocyte...
- Participants must not have chronic diarrhea or an average baseline stool output of more than 4 stools per day.
- Participants must not have gastrointestinal disorders that may impair enteral absorption of dC/dT, such as inflammatory bowel disease or short bowel...
- Participants must not have chronic kidney disease with an estimated glomerular filtration rate \< 60 mL/min/1.73 m2.
See the full eligibility criteria
- Age ≥ 1 year and ≤ 70 years
- Karnofsky performance status ≥ 50 for participants ≥16 years of age and Lansky performance status ≥ 50 for participants \<16 years of age
- Diagnosis requirement. Participants must meet at least one of the following requirements for a diagnosis of a telomere biology disorder:
- Age-adjusted mean telomere length \< 1%ile in peripheral blood lymphocytes by flow cytometry-fluorescence in situ hybridization (flow-FISH), as reported by a Clinical Laboratory Improvement Amendments (CLIA)-approved...
- Pathogenic or likely pathogenic variant(s) in one of the follow telomere biology associated genes: DKC1, TERC, TERT, NOP10, NHP2, WRAP53/TCAB1, TINF2, CTC1, RTEL1, ACD, PARN, NAF1, STN1, ZCCHC8, POT1, RPA1, DCLRE1B...
- Participants must exhibit at least one active clinical manifestation associated with a telomere biology disorder, in the judgment of the PI, which includes but is not limited to the following: one or more peripheral...
- Participants must be able to take enteral liquids by mouth or enteral feeding tube.
- Female participants who are sexually active and could become pregnant must use two effective methods of contraception, at least one of which must be considered a highly effective method.
- Participants (or parent/legally authorized representative for minors) must demonstrate the ability to understand and willingness to provide informed consent, which will be documented using an institutionally approved...
- Participants must not have very severe aplastic anemia necessitating bone marrow transplant at the time of enrollment. Very severe aplastic anemia is defined by the presence of at least 2 of the following: ANC \<200...
- Participants must not otherwise be expected to undergo bone marrow transplantation within 6 months of enrollment.
- Participants must not be taking concurrent medications intended to improve hematopoiesis such as androgens or growth factors, including granulocyte colony stimulating factor, erythropoietin, or thrombopoietin mimetics...
- Participants must not have chronic diarrhea or an average baseline stool output of more than 4 stools per day.
- Participants must not have gastrointestinal disorders that may impair enteral absorption of dC/dT, such as inflammatory bowel disease or short bowel syndrome.
- Participants must not have chronic kidney disease with an estimated glomerular filtration rate \< 60 mL/min/1.73 m2.
- Participants must not be on other medications or study agents or have other uncontrolled intercurrent illness that could interfere with study interpretation, in the opinion of the study Principal Investigator (PI)
- Participants must not have high-risk myelodysplastic syndrome or leukemia or other active malignancy.
- Pregnant individuals will not be eligible for enrollment given the physiological changes in blood counts that occur during pregnancy.
- Breastfeeding mothers will not be eligible for enrollment due to the unknown risk to nursing infants.
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.