Pilot study to investigate the feasibility of 13-cis-Retinoic acid pharmacokinetic monitoring in high-risk neuroblastoma - 13 cis-Retinoic acid monitoring study

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2008-003606-33-GB

Enrollment

Registered

2008-09-04

Start date

2009-01-15

Completion date

Unknown

Last updated

2015-08-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

High risk neuroblastoma MedDRA version: 9.1 Level: LLT Classification code 10029260 Term: Neuroblastoma

Interventions

Trade Name: Roaccutane Pharmaceutical Form: Capsule, soft INN or Proposed INN: isotretinoin CAS Number: 4759482 Current Sponsor code: PK 2008 03 Other descriptive name: 13-cis-retinoic acid Concentrat

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: a) Age less than or equal to 18 years. b) Diagnosis of high-risk neuroblastoma. c) Receiving 13-cis-RA (Roaccutane) as part of clinical treatment. d) Single or double lumen central venous catheter in place. e) Written informed consent. f) Protocol approval by national and local ethics committee, regulatory authority and Trust R&D departments. g) A negative pregnancy test for women of child bearing potential, and sexually active patients and partners agreeing to undertake adequate contraceptive measures Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Failure to comply with any of the inclusion criteria

Design outcomes

Primary

Measure	Time frame
Secondary Objective: not applicable;Primary end point(s): Target accrual of up to 75 patients reached Effective individualised dosing of isotretinoin in high-risk neuroblastoma patients to achieve consistent plasma 13-cis-retinoic acid concentrations >2µM with acceptable toxicity;Main Objective: The study is designed as a pilot study to: •Investigate the feasibility of implementing 13-cis-Retinoic acid dose modification following course 1 of treatment, based on targeted 13-cis-Retinoic acid plasma concentrations and observed toxicity. • Minimize the large inter-patient variation in plasma concentrations of 13-cis-Retinoic acid observed following standard treatment for high-risk neuroblastoma. This will ensure that patients are not exposed to potentially sub-optimal plasma concentrations of 13-cis-RA during long-term treatment, particularly for those children who are not able to swallow 13-cis-RA capsules. • Obtain preliminary data investigating the potential impact of 13-cis-RA therapeutic monitoring on clinical response and toxicity in children with high-risk neuroblastoma	—

Measure

Time frame

Secondary Objective: not applicable;Primary end point(s): Target accrual of up to 75 patients reached Effective individualised dosing of isotretinoin in high-risk neuroblastoma patients to achieve consistent plasma 13-cis-retinoic acid concentrations >2µM with acceptable toxicity;Main Objective: The study is designed as a pilot study to: •Investigate the feasibility of implementing 13-cis-Retinoic acid dose modification following course 1 of treatment, based on targeted 13-cis-Retinoic acid plasma concentrations and observed toxicity. • Minimize the large inter-patient variation in plasma concentrations of 13-cis-Retinoic acid observed following standard treatment for high-risk neuroblastoma. This will ensure that patients are not exposed to potentially sub-optimal plasma concentrations of 13-cis-RA during long-term treatment, particularly for those children who are not able to swallow 13-cis-RA capsules. • Obtain preliminary data investigating the potential impact of 13-cis-RA therapeutic monitoring on clinical response and toxicity in children with high-risk neuroblastoma

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Countries

United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026