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NCT01125800 · ClinicalTrials.gov registry record · Phase 1
A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB
A Phase 1 study, sponsored by Novartis Pharmaceuticals.
- Completed
- Registry status
- Phase 1
- Development phase
- 76
- Enrollment target
NCT01125800: Completed Phase 1 study, sponsored by Novartis Pharmaceuticals.
NCT01125800 is a Phase 1 study that has completed, run by Novartis Pharmaceuticals. The registered enrollment target is 76 participants, above the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (27% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01125800, a Phase 1 study, has completed, sponsored by Novartis Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 76 participants
- Enrollment target
Study Summary
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway. Phase II study is to assess preliminary efficacy in both adult and pediatric patients with recurrent or refractory MB.
Primary Outcome
DLT was defined as an adverse event (AE) or abnormal laboratory value assessed as unrelated to disease, disease progression, intercurrent illness, or concomitant medications. DLT included any grade 3 or 4 clinically-evident toxicity, Hematology: ≥ CTCAE grade 3 neutropenia (ANC \<1.0x10\^9/L); ≥ CTCAE grade 3 thrombocytopenia (platelets \<50x10\^9/L); ≥ CTCAE grade 3 anemia (Hgb \<80 g/L); Febrile neutropenia (ANC \<1x10\^9/L, fever ‡ 38.5°C), Renal: ≥ CTCAE grade 3 serum creatinine (\>3xULN), H
Interventions
- DRUG LDE225
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 76 participants |
| Start Date | 2011-02 |
| Est. Completion | 2014-10 |
| Phase | Phase 1 |
What the finished NCT01125800 record still lists
NCT01125800 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 76 participants, a relatively small participant target, above the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (27% higher).
The record links to 0 conditions, and to 1 intervention - of which LDE225 is the first listed.
NCT01125800 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01125800 about?
NCT01125800 is a clinical study titled "A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB". Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway. Phase II stu...
What is the current status of trial NCT01125800?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 76 participants. The study started on 2011-02. Estimated completion is 2014-10.
What interventions are being tested in trial NCT01125800?
The interventions under investigation include: LDE225 (DRUG).
Who is sponsoring clinical trial NCT01125800?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01125800's enrollment target sits among peer trials
76 705th of 2000 higher than 1,290 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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