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NCT00006450COMPLETEDanonymous

Phase II Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Progressive or Recurrent CNS Malignancy

Sponsor

Source record

National Cancer Institute (NCI)

Phase

Source record

Phase 2

Modality

AI-normalized

small molecule

Target

AI-normalized

Phenylbutyrate acts primarily through its metabolite phenylacetate, which induces tumor cytostasis and differentiation in various cancer cell lines, including malignant gliomas and neuroblastomas. It may also induce apoptosis through distinct molecular activities.

Indication / condition

AI-normalized

Brain Tumor

Intervention

Source record

Phenylbutyrate

Source & freshness

Source record

NCT ID

NCT00006450

Original source

ClinicalTrials.gov

Source last updated

Mar 04, 2008

Ingested at

Jun 13, 2026

Internal sync

Jun 13, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

Open original registry record
View original source fields

NCT ID

NCT00006450

Title

Phase II Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Progressive or Recurrent CNS Malignancy

Sponsor

National Cancer Institute (NCI)

Status

COMPLETED

Phase

Phase 2

Condition raw

Brain Tumor

Condition normalized

Brain Tumor

Modality raw

small molecule

Modality normalized

small molecule

Target raw

Phenylbutyrate acts primarily through its metabolite phenylacetate, which induces tumor cytostasis and differentiation in various cancer cell lines, including malignant gliomas and neuroblastomas. It may also induce apoptosis through distinct molecular activities.

Target normalized

Phenylbutyrate acts primarily through its metabolite phenylacetate, which induces tumor cytostasis and differentiation in various cancer cell lines, including malignant gliomas and neuroblastomas. It may also induce apoptosis through distinct molecular activities.

Interventions

Phenylbutyrate

Public preview

Source record

The Phase II trial of phenylbutyrate for pediatric patients with progressive or recurrent CNS malignancies addresses a significant unmet need in the treatment of childhood brain tumors. Given the limited therapeutic options available for this patient population, successful outcomes could position phenylbutyrate as a novel treatment modality, enhancing its commercial viability. The pediatric oncology market is characterized by high demand for innovative therapies, especially for rare and aggressive tumor types. Competitive analysis should focus on existing treatments for similar indications, including chemotherapeutic agents and emerging therapies. Diligence should assess the potential for partnership or licensing opportunities with larger pharmaceutical companies, particularly those with established oncology portfolios.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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TrialSignal

Clinical trial intelligence report

Phase II Trial of Phenylbutyrate Given as a Continuous Infusion in Pediatric Patients With Progressive or Recurrent CNS Malignancy

Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.

Generated

Jun 16, 2026

NCT ID

NCT00006450

Status

COMPLETED

Phase

Phase 2

Sponsor

National Cancer Institute (NCI)

Executive brief

Investment-Ready Snapshot

The Phase II trial of phenylbutyrate for pediatric patients with progressive or recurrent CNS malignancies addresses a significant unmet need in the treatment of childhood brain tumors. Given the limited therapeutic options available for this patient population, successful outcomes could position phenylbutyrate as a novel treatment modality, enhancing its commercial viability. The pediatric oncology market is characterized by high demand for innovative therapies, especially for rare and aggressive tumor types. Competitive analysis should focus on existing treatments for similar indications, including chemotherapeutic agents and emerging therapies. Diligence should assess the potential for partnership or licensing opportunities with larger pharmaceutical companies, particularly those with established oncology portfolios.

Source & freshness

Provenance

SourceClinicalTrials.gov
Source updatedMar 04, 2008
Internal syncJun 13, 2026
Model versiontrialsignal-ai-v1

https://clinicaltrials.gov/study/NCT00006450

Indication

Brain Tumor

Modality

small molecule

Target

Phenylbutyrate acts primarily through its metabolite phenylacetate, which induces tumor cytostasis and differentiation in various cancer cell lines, including malignant gliomas and neuroblastomas. It may also induce apoptosis through distinct molecular activities.

Intervention

Phenylbutyrate

Source record

Protocol Description

Detailed source ingestion pending.

Source record

Outcome Measures

Detailed source ingestion pending.

Source record

Eligibility

Detailed source ingestion pending.

AI analysis

Known Results And Readout Context

Detailed source ingestion pending.

IP intelligence

Patent And IP Landscape

Detailed source ingestion pending.

Source record

Contacts

Detailed source ingestion pending.