Phase 2 Thyroid Cancer Trial, Active, Not Recruiting NCT04284774 Sponsor: National Cancer Institute (NCI) Condition: Thyroid Cancer
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Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial enrolled 5 children and young people (referred to as paediatric patients) who received a drug called tipifarnib. The trial was designed to measure how many participants' tumours shrank or disappeared (known as an "objective response"), how long participants went without their disease getting worse (called "progression-free survival"), and what proportion experienced serious side effects. All 5 participants are recorded as not having completed the study, and none are recorded as having completed it — the data does not explain the reasons for this. The reported data shows that the primary outcome — the percentage of patients whose tumours shrank or disappeared — was 0%, meaning none of the 5 participants met the criteria for a response during the study. For the secondary outcomes, including progression-free survival and the rate of serious side effects (graded 3 or higher), no numerical results were reported on ClinicalTrials.gov. The same applies to the additional exploratory measures looking at tumour genetics and biomarkers — no data was reported for those outcomes either. Because the trial ended with no participants completing it and most outcome data was not reported, the information available from this submission is very limited. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 2 Thyroid Cancer Trial, Active, Not Recruiting

NCT04284774
Active, Not Recruiting Phase 2

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You must have already enrolled in a related study (called APEC1621SC / NCT03155620) and been matched to this treatment based on a specific genetic change found in your cancer.
  • You must be at least 12 months old and no older than 21 years old when joining the study.
  • Your body surface area (a measure based on height and weight) must meet a minimum size requirement (confirm with trial site).
  • Your cancer must be measurable on a scan (such as an MRI or CT), meaning there is at least one tumor that can be seen and measured; children with neuroblastoma may still qualify if their cancer shows up on a specific type of scan called an MIBG scan even if it cannot be measured in the usual way.
  • You must be well enough to carry out at least some daily activities, as measured by a standard ability scale used for your age group.
  • You must have recovered from side effects of any previous cancer treatments, and enough time must have passed since your last treatment (the exact waiting period depends on what type of treatment you had — confirm with trial site).
  • You must never have received the study drug, tipifarnib, before.
  • Your blood cell counts (white cells and platelets) must be at or above certain levels in the week before joining (specific numbers depend on whether your bone marrow is affected by cancer — confirm with trial site).
  • Your kidneys must be working well enough, based on a blood or urine test done within the past week.
  • Your liver must be working well enough, based on blood tests done within the past week.
  • Your blood protein (albumin) level must be at or above a minimum level, based on a blood test done within the past week.
  • If you have a seizure condition, you may still be eligible as long as it is well controlled with medication.
  • Any nerve-related side effects from past treatments must be mild (grade 2 or lower).
  • You must be able to swallow tablets whole or crushed and mixed into a drink or soft food, or receive them through a feeding tube.
  • You (and/or your parent or legal guardian) must be willing and able to sign a consent form agreeing to take part.

Who may not be able to join:

  • Women who are pregnant or breastfeeding cannot join this trial.
  • Anyone who could become pregnant or father a child must agree to use two reliable forms of contraception during treatment and for at least 4 weeks after the last dose; males who wish to preserve their fertility should discuss sperm banking before starting treatment.
  • You are not eligible if you are taking steroid medications (such as prednisone) unless you have been on a stable or reducing dose for at least 7 days before joining (or at least 14 days if the steroids were used to treat side effects of a previous cancer therapy).
  • You cannot join if you are currently taking part in another clinical trial using an experimental drug.
  • You cannot join if you are currently receiving any other cancer treatment.
  • You are not eligible if you are taking medications to prevent graft-versus-host disease after a bone marrow transplant (such as cyclosporine or tacrolimus).
  • You cannot join if you are taking certain medications that strongly affect how the body processes the study drug (specific drug interactions — confirm with trial site).
  • You are not eligible if you have had an allergic reaction to tipifarnib (the study drug) or any of its ingredients.
  • You are not eligible if you have had an allergic reaction to a group of antifungal medicines called imidazoles (such as clotrimazole, ketoconazole, or miconazole).
  • You cannot join if you currently have an infection that is not under control.
  • You are not eligible if you have previously had a transplant of a solid organ (such as a kidney, heart, or liver).
  • You may not be eligible if the study doctors feel you would not be able to safely follow the study's monitoring and safety requirements.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 27 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

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Trial details

Status
Active, Not Recruiting
Phase
Phase 2
Registry
ClinicalTrials.gov
Start date
13 October 2020
Est. completion
31 March 2024

Where this trial is recruiting

Puerto Rico 🇺🇸 United States

Primary endpoints

Objective Response Rate (Complete Response + Partial Response) in Pediatric Patients Treated With Tipifarnib

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov