Pediatric Trials in Latin America Clinical Research in Pediatric Oncology

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1 Pediatric Trials in Latin America Clinical Research in Pediatric Oncology Prof. Dr. Antonio Sérgio Petrilli

2 Clinical Research Regulations Timeline Best Pharmaceuticals for Children Act estabilished programme and funding for study of off-lpatent drugs. Pediatric Research Equitity Act Pediatric Rule FDA requiring manufactures to assess the safety and effectiveness of new drugs and biological products for pediatric labeling. Marketing authorization applications must contain a pediatric plan or waiver. Page 2

3 Objectives 1. When to perform pediatric research? 2. Timing of initiation; 3. Types of studies; 4. Age categories; 5. Ethical considerations. Page 3

4 1. When to perform pediatric research? Take into account: The prevalence of the condition to be treated in the pediatric population; Seriousness of the condition; Availability of alternative treatments in the pediatric population, including efficacy and adverse event profile. Page 4

5 1. When to perform pediatric research? Take into account: Whether the medicinal product is novel or one of a class of compounds with known properties; Age ranges of pediatric patients likely to be treated; Unique pediatric safety concerns (developmental) Page 5

6 2. Timing of initiation Concomitantly After initial safety analysis Phase I Phase II Phase III Diseases predominantly or exclusively affecting pediatric patients. Serious or life-threatening diseases, occurring in both adults and pediatric patients. After efficacy and safety analysis Other diseases and conditions. Page 6 Risk x Benefit

7 3. Types of studies Pharmacokinetics Consider: Number of patients and samples x patient: sparse sampling, sufficient to generate a comparison with adult parameters. Routine collections, wherever possible, at the same time as samples for pharmacokinetic analysis. Trained staff and sensitive assays in order to decrease the volume of blood required per sample. Page 7

8 3. Types of studies Efficacy Consider: Measurement of subjective symtoms (ie: pain) Specific Assessment instruments. Ie: pain, quality of life. Page 8 Caran, E, et al. Pediatr Blood Cancer, 45:925 32, 2005

9 3. Types of studies Performance Score Karnofsky (pacientes> 16 anos) Lansky (pacientes< 16 anos) 100 Totalmente ativo; normal. Sem queixas; sem evidência de doença; 90 Restrições leves em atividades físicas vigorosas. Atividade normal; sinais ou sintomas da doença leves. 80 Ativo, mas cansa mais rápido. Atividade normal com esforço; alguns sinais ou sintomas da doença 70 Grandes restrições e tempo menor nas atividades físicas. 60 Em pé e caminhando, mas mínima atividade, ocupa-se com atividades mais calmas. 50 Veste-se, mas fica muito deitado durante o dia; capaz de participar em todas as atividades calmas 40 Maior parte na cama; participa de atividades calmas 30 Na cama; necessita de assistência mesmo para atividades calmas Page 9 Cuida-se; capaz de exercer atividades normais ou de fazer trabalho ativo Necessita de assistência ocasional, mas tem capacidade de cuidas de muitas necessidades pessoais. Necessita de assistência considerável e cuidados médicos frequentes; atividades calmas. Incapacitado; necessita de cuidado e assistência especiais Gravemente incapacitado; é indicada internação embora a morte não seja iminente. 20 Dorme com frequência; atividades passivas Muito doente; necessária internação, necessário tratamento de suporte ativo 10 Sem atividade, não deixa a cama Moribundo 0 Não responsivo Morto

10 3. Types of studies Efficacy Consider: - Dosing Weight x BSA FIG. 1. The percentage of full dose administered resulting from three methods of dose reduction in response to weight loss (A) and the drug exposure, or area under the curve (AUC), resulting from the administration of these doses (B). Page 10 Dose Reduction of Chemotherapeutic Agents after Weight Loss. O Marcaigh, Aengus; Gilchrist, Gerald, American Journal of Clinical Oncology. 20(2): , 195, April 1997.

11 3. Types of studies Safety Consider: Normal lab values age appropriate; Different metabolism (Ex: renal and liver clearance) Long-term safety studies (Ex: chemotherapy cardiotoxicity) Page 11 Pediatr Blood Cancer 2005;

12 4. Age categories. Preterm Newborn Infants; Term Newborn Infants (0 to 27 days); Infants and toddlers (28 days to 23 months); Children (2 to 11 years); Adolescents (12 to 18 years). Page 12

13 5. Ethical Considerations Vulnerable population Consent and Assent: appropriate age? Economic aspects (patents extension) Page 13

14 Necessary actions from involved parts Sponsors Stimulate the development of pediatric studies Protocols designed to minimize risks. IRBs Knowledge of ethical, clinical and psico-social pediatric aspects Consider risks from the children s point of view Familiarity with study designs that minimize risks Faster approval processes without ethical prejudice. Investigators Training and experience in pediatric studies. Page 14

15 Pediatric Oncology Studies Our experience Protocols Phases I, II, III and IV Page 15

16 Phases I and II Protocols Our experience Objectives Risks Benefits Observations Phase I - Pharmacokinets/ dynamics, - Initial safety analysis Unknown toxicities Direct feeling to have tried all possible options Oncology nonresponders to other therapies. Phase II Efficacy and short-term safety Unproven efficacy Direct possibility of survival/quality of life benefits Still non-responders, but drugs already registered for adult use. Page 16

17 Phase I Protocols Page 17 J Clin Oncol 18: by American Society of Clinical Oncology

18 Difficulties Page 18 J Clin Oncol 18: by American Society of Clinical Oncology

19 Phases III and IV Protocols Our experience Objectives Risks Benefits Observations Phase III Short and long term risk/benefit analysis Known treatment toxicities Direct patients have access to drugs not otherwise available Phase IV Marketing and postmarketing surveillance Known treatment toxicities Direct medications without costs. Page 19

20 Pediatric Oncology Studies Our experience Protocols Phases I, II, III and IV Epidemiologic studies Page 20

21 Epidemiological Studies Our experience Objectives Risks Benefits Observations Increase knowledge about population and disease characteristics Disclosure of personal data Indirect Publication, health services Page 21

22 Pediatric Oncology Studies Our experience Protocols Phases I, II, III and IV Epidemiologic studies Collaborative treatment studies Page 22

23 Page 23

24 Collaborative treatment studies COG Since the 50s, increase from 10% to more than 77% on the cure rates. Participating centers Patients Children s Oncology Group: Chemotherapy benefits for children with cancer were first proven; Discovery of the first tumor suppressor gene; Multi-drug chemotherapy. Page 24

25 Collaborative treatment studies COG 3% Relative mortality rate reduction - USA % 2.6% Média Anual em % 2% 1% Why? 1.8% 1.5% 0.9% 1.1% 1.5% 0.4% 0% Idade (Anos) Page 25 Bleyer, W.A. The impact of childhood cancer on the United States and the world Ca,40: ,1990; Bleyer, W.A. Conferência ministrada durante a 30ª ASCO 2000

26 Collaborative treatment studies COG Inclusions on Treatment Protocols NCI Clinical Trials Inclusions Idade (Anos) Page 26 Bleyer, W.A. The impact of childhood cancer on the United States and the world Ca,40: ,1990; Bleyer, W.A. Conferência ministrada durante a 30ª ASCO 2000

27 Collaborative Treatment Protocols COG Correlation between Mortality rates reduction and Clinical Trials Admissions Annual Mediam % Reduction of Mortality 3% 2% 1% 0% % Reduction on Mortality AGE Page 27 Bleyer, W.A. The impact of childhood cancer on the United States and the world Ca,40: ,1990; Bleyer, W.A. Conferência ministrada durante a 30ª ASCO % 20% 15% 10% 5% 0% r =.91, p =.001 1,000 10,000 Admissions (log) Admissions on Clinical Trials

28 Study III GCBTO Study N Sites Study III *39 (9.3%)/421 Enrolled patients/brazil expected cases/year ( ) Page 28 IBGE Censos 1991, 1996 e 2000 / SEER - Malignant Bone Tumors Chapter

29 Study IV GCBTO Study N Sites ( ) Study III *39 (9.3%)/421 Enrolled patients/brazil expected cases/year Study N Sites Study IV *29 (6.8%)/427 Enrolled patients/brazil expected cases/year Page 29 IBGE Censos 1991, 1996 e 2000 / SEER - Malignant Bone Tumors Chapter

30 Study V GCBTO Study N Sites ( ) Study III *39 (9.3%)/421 Enrolled patients/brazil expected cases/year Study N Sites Study IV *29 (6.8%)/427 Enrolled patients/brazil expected cases/year Study N Sites Study V *74 (16.5%)/447 Enrolled patients/brazil expected cases/year Page 30 IBGE Censos 1991, 1996 e 2000 / SEER - Malignant Bone Tumors Chapter

31 Overall Survival x Protocol N=553 Kaplan-Meier survival estimates (2 yo)p=0.012 (5 yo)p<0.001 Study Page 31 Meta (%) NMeta (%) III IV V % 58% 50% 48% 57% 46% analysis time Study= III Study = IV Study= V

32 Overall Survival x Presence of Metastases N=553 Kaplan-Meier survival estimates P< % 37% analysis time Nmeta Meta 59% 22% Page 32

33 Collaborative Treatment Protocols GALOP Protocol GLATO sites Brasil 2 sites Argentina 1 site Uruguai Page 33

34 Inclusion Rates X # of Sites Study N Sites Study Page 34

35 Studies III, IV, V and 2006 Study Sites N Study III Study IV Study V Study Total 1009 Page 35

36 Important Publications Page 36

37 Important Recent Citations Page 37

38 Pediatric Oncology Studies Our experience Protocols Phases I, II, III and IV Epidemiologic studies Collaborative treatment studies Studies of Supportive care medications Page 38

39 Support meds. Studies Our experience Objectives Risks Benefits Observations Supportive care (infectious diseases, emesis, nutrition, palliative care) Treatment toxicities and disclosure of personal data Direct improvements on life quality Increase knowledge about medications on patients with concomitant conditions (cancer, neutropenia, etc) Page 39

40 Pediatric Oncology Studies Our experience Protocols Phases I, II, III and IV Epidemiologic studies Collaborative treatment studies Studies of Supportive care medications Molecular biology studies Page 40

41 Molecular Biology Studies Our experience Objectives Risks Benefits Observations Determine molecular causes of pathologies, staging and prognosis. Disclosure of personal data Direct staging and prognosis Indirectscientific Biological Targets Obs: tumor banking Page 41

42 Tumor Bank Osteosarcoma BX Srg Meta Page 42 Specimen N % Biopsy (BX) Surgeries (Srg) Metastatic Disease (Meta) Total

43 Clinical Research Center IOP GRAACC GRAACC UNIFESP Page 43

44 IOP GRAACC Unifesp 115 Studies since 2004 being: - 81 Internal protocols - 22 Sponsored - 12 Cooperative International and National Groups 54 ongoing studies: Page 44

45 Of all the forms of inequity, injustice in health care is the most shocking and inhumane. Dr. Martin Luther King Jr. Tel: (11) Fax: (11) Page 45

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