A Study of Pomalidomide (CC-4047) Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03257631|
Recruitment Status : Recruiting
First Posted : August 22, 2017
Last Update Posted : April 5, 2018
A Phase 2 study that will assess the efficacy, safety and tolerability of pomalidomide in children and young adults aged 1 to < 21 years with recurrent or progressive primary brain tumors.
The study will consist of 4 parallel groups, one for each of the following primary brain tumor types: high-grade glioma, medulloblastoma, ependymoma and DIPG. A Simon's Optimal two-stage study design will be applied to each group.
|Condition or disease||Intervention/treatment||Phase|
|Central Nervous System Neoplasms Medulloblastoma||Drug: Pomalidomide||Phase 2|
This is a Phase 2 multi-center, open-label, parallel-group study that will assess the efficacy, safety and tolerability of pomalidomide in children and young adults aged 1 to < 21 years with recurrent or progressive primary brain tumors after at least one prior standard therapy.
The study will consist of 4 parallel groups, one for each of the following primary brain tumor types: high-grade glioma, medulloblastoma, ependymoma and DIPG. A Simon's Optimal two-stage study design will be applied to each group and enrollment will occur as follows:
- Stage 1: Nine subjects will be enrolled.
- Stage 2: If during Stage 1, ≥ 2 subjects achieves either an objective response (either CR or PR) within the first 6 cycles of treatment (within first 3 cycles for DIPG), or a long-term SD, an additional 11 subjects shall be enrolled; otherwise no additional subjects will be enrolled into that group.
- If a total of 5 or more subjects across all 20 subjects in a given group (Stage 1 and 2) evaluable for the primary endpoint are observed as having either an objective response (either CR or PR) within the first 6 cycles of treatment (within first 3 cycles for DIPG) or a long-term SD, pomalidomide will be considered effective in that disease indication. Subjects who do not meet the criteria for an objective response or disease progression by the end of Cycle 6 (end of Cycle 3 for DIPG subjects) will be considered as having long-term SD.
Response evaluations will be based on MRI results obtained at each site and will be assessed both locally and by an independent central reviewer. Corticosteroid use and clinical assessments (ie, neurologic status) will also be considered when determining overall response.
Tumor assessments will be conducted by standard MRI with and without contrast using three MRI sequences (T1-weighted pre- and post-contrast, T2-weighted, fluid-attenuated inversion recovery [FLAIR]). Overall radiographic objective response will be assessed utilizing the sequence(s) best representative of tumor in the opinion of the neuroradiologist.
Once treatment has been discontinued, subjects will be followed up for up to 5 years from enrollment of the last subject.
Subjects who withdraw from either stage for reasons other than disease progression prior to completing Cycle 1 of study treatment will be replaced.
An independent Data Monitoring Committee will monitor the study conduct.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||36 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A PHASE 2 CLINICAL STUDY OF POMALIDOMIDE (CC-4047) MONOTHERAPY FOR CHILDREN AND YOUNG ADULTS WITH RECURRENT OR PROGRESSIVE PRIMARY BRAIN TUMORS|
|Actual Study Start Date :||August 22, 2017|
|Estimated Primary Completion Date :||August 13, 2018|
|Estimated Study Completion Date :||August 13, 2018|
Pomalidomide will administered at a starting dose of 2.6 m2/day. Pomalidomide will be provided as either a capsule (0.5 mg, 1 mg, 2 mg, 3 mg or 4 mg) or as an oral suspension (2 mg/mL).
: Subjects will be administered pomalidomide on Days 1 to 21, followed by a 7-day rest period, of each 28-day treatment cycle and will continue treatment for up to 24 cycles or until disease progression, withdrawal of consent/assent or unresolved toxicities as described in the protocol.
Other Name: CC-4047
- Objective Response (OR), Long-term Stable Disease (SD) Rate (ORSDR) [ Time Frame: Up to approximately 6 months ]The total number of subjects achieving either an OR (CR or PR) or long-term SD, analyzed per stratum
- Objective Response Rate (ORR) [ Time Frame: Up to approximately 5 years ]Total number of subjects achieving an OR (either CR or PR) analyzed per stratum
- Long-term stable disease rate [ Time Frame: Up to approximately 6 months ]Total number of subjects achieving a long-term SD analyzed per stratum
- Duration of response [ Time Frame: Up to approximately 5 years ]The time from first achievement of CR or PR under study treatment, whichever occurs first, until first documentation of progressive disease (PD) or death of any cause
- Progression-free survival [ Time Frame: Up to approximately 5 years ]The time from enrollment until first documentation of PD and/or death of any cause
- Overall Survival [ Time Frame: Up to approximately 5 years ]The time from enrollment until death of any cause
- Adverse Events (AEs) [ Time Frame: Up to approximately 5 years ]Type, frequency, and severity of AEs and relationship to study drug
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03257631
|Contact: Associate Director Clinical Trial Disclosurefirstname.lastname@example.org|
|United States, California|
|Stanford University Cancer Center||Recruiting|
|Stanford, California, United States, 94305-5750|
|United States, Florida|
|University of Florida||Recruiting|
|Gainesville, Florida, United States, 32611|
|United States, Illinois|
|Ann and Robert H Lurie Childrens Hospital of Chicago||Recruiting|
|Chicago, Illinois, United States, 60611|
|United States, Maryland|
|National Cancer Institute||Recruiting|
|Bethesda, Maryland, United States, 20892|
|United States, Massachusetts|
|Dana Farber Cancer Institute||Recruiting|
|Boston, Massachusetts, United States, 02115|
|United States, Texas|
|Baylor College of Medicine||Recruiting|
|Houston, Texas, United States, 77030|
|Centre Oscar Lambret||Recruiting|
|Lille, France, 59020|
|Centre Leon Berard||Recruiting|
|Lyon, France, 69008|
|Hopital d'Enfants de la Timone||Recruiting|
|Marseille Cedex 01, France, 13005|
|Paris, France, 75005|
|Hopital des Enfants||Recruiting|
|Toulouse, France, 31059|
|CHU Nancy Hematology||Recruiting|
|Vandoeuvre les Nancy, France, 54511|
|Institut Gustave Roussy Faculte de Medecine Paris Sud Service de pneumologie||Recruiting|
|Villejuif Cedex, France, 94805|
|Istituto G. Gaslini Ospedale Pediatrico IRCCS||Recruiting|
|Fondazione IRCCS Istituto Nazionale dei Tumori||Recruiting|
|Milan, Italy, 20133|
|Ospedale Bambin Gesu||Recruiting|
|Roma, Italy, 00165|
|Hospital Universitario Vall D Hebron||Recruiting|
|Barcelona, Spain, 8035|
|Hospital Infantil Universitario Nino Jesus||Recruiting|
|Madrid, Spain, 28009|
|Hospital Universitario La Fe||Recruiting|
|Valencia, Spain, 46026|
|Leeds General Infirmary||Recruiting|
|Leeds, United Kingdom, LS7 4SA|
|Great Ormond Street Hospital||Recruiting|
|London, United Kingdom, WC1N 3JH|
|Royal Victoria Infirmary Department of Haematology||Not yet recruiting|
|Newcastle-upon-Tyne, United Kingdom, NE1 4LP|
|The Royal Marsden Hospital||Recruiting|
|Sutton-Surrey, United Kingdom, SM2 5PT|
|Study Director:||Bouchra Benettaib, MD||Celgene Corporation|