Common Protocol Template Pediatric Library v001

Common Protocol Template Pediatric Library v001CPT Section Library Content2. Introduction Instructional text for pediatric-specific protocol

requirements2.2. Background Instructional text for background, including disease and

intervention in adult population and rationale for dose selection in pediatric population

3. Objectives and Endpoints Pediatric-specific example text for objectives and endpoints

4.3. Justification for Dose Instructional text specifying considerations that should be made in dosing decisions, with a focus on differences in absorption, distribution, metabolism, and excretion (ADME) in children of different ages and implications of under- or over-dosing

5. Study Population Instructional text regarding justification of study population and stratification of subgroups

5.1. Inclusion Criteria Language including instructional text, template text, age group guidelines, and informed consent guidance

5.3.3. Activity Instructional text regarding exercise in the pediatric population

6.1. Study Intervention Administered

Instructional text specifying that dosing instructions should be given to both the child and caregiver

6.1.1. Medical Devices Instructional text regarding device (if applicable)6.2. Preparation / Handling / Storage / Accountability

Instructional text related to pediatric-specific and general guidelines for preparation, handling, storage, and accountability of the study intervention

6.5. Concomitant Therapy Guidance related primarily to vaccines6.7. Intervention after the End of the Study

Discussion of potential intervention options after the study

8.1. Efficacy Assessments Guidance regarding observer assessments and growth parameters

8.2. Safety Assessments Instructional text specifying what safety evaluations should be included

8.2.1. Physical Examinations Guidance regarding physical examinations and Tanner Staging

8.2.2. Vital Signs Instructional text for measuring vital signs in the pediatric population

8.2.3. Electrocardiograms Guidance regarding electrocardiogram (ECG) assessments (if required) in the pediatric population

8.2.4. Clinical Safety Laboratory Assessments

Guidance regarding laboratory assessments in the pediatric population

8.2.5. Suicidal Risk Monitoring Guidance for monitoring suicide risk and relevance across age groups

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Common Protocol Template Pediatric Library v001

CPT Section Library Content8.3.5. Pregnancy Instructional text explaining relevance of section given

the pediatric population and investigator responsibility to report pregnancy test results

8.4. Treatment of Overdose Recommendation to have caregiver store study intervention out of reach of the child

8.9. [Health Economics] OR [Medical Resource Utilization and Health Economics]

Guidance for assessment of health economics in children and their caregivers

9.2. Sample Size Determination Instructional text for age groups and number of subjects enrolled in each group

9.4. Statistical Analyses Instructional text for data analysis and stratification.10.1 Appendix 1: Regulatory, Ethical, and Study Oversight Considerations

Instructional and template text regarding data monitoring committee (DMC), informed consent, medical records, and data protection

10.2 Appendix 2: Clinical Laboratory Tests

Instructional and template text for clinical laboratory test processes

10.4 Appendix 4: Contraceptive Guidance and Collection of Pregnancy Information

Sample text for pregnancy reporting

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Common Protocol Template Pediatric Library v001

2. Introduction

2.2 Background

3. Objectives and EndpointsObjectives Endpoints

Primary

To evaluate the efficacy of [dose/amount of Study Intervention] compared with placebo administered [frequency] to children and adolescents with [condition/diagnosis]

The change in [variable, absolute, or percentage] from baseline to [Week X]

Secondary

To evaluate the efficacy of [dose/amount of Study Intervention] compared with placebo administered [frequency] to children and adolescents with [condition/diagnosis] as measured by [variable]

The change in [variable, absolute, or percentage] from baseline to [Week X]

To characterize the pharmacokinetics (PK) of [dose/amount of Study Intervention] in children and adolescents with [condition/diagnosis]

Plasma PK parameters [list parameters] for [Study Intervention]

To characterize the pharmacokinetic-pharmacodynamic (PK-PD) relationship of [outcome] to plasma concentrations of [Study Intervention]

[As determined based on the planned correlative analyses]

To evaluate the safety and tolerability of [dose/amount of Study Intervention] given [frequency] in the treatment of children and adolescents with [condition/diagnosis]

Adverse events (AEs), safety laboratory assessments, electrocardiograms (ECGs), and vital signs

To characterize the [growth rate or other similar variable] in children and adolescents with [condition/diagnosis] who are taking [dose/amount of Study Intervention] given [frequency]

The change in [variable, absolute, or percentage] from baseline to [Week X]

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Common Protocol Template Pediatric Library v001

4.3 Justification for Dose

5. Study Population

5.1 Inclusion CriteriaAge

Male or female [outpatient/inpatient] participants who are [insert age category/requirements] of age at the screening visit.

Placebo Swallow Test (if applicable): The placebo swallow test will be conducted at the study site during the screening phase (anytime between the screening and baseline visits) to ensure that participants are able to swallow [x tablets and x capsules] (one at a time) of the same size as [study intervention, control, placebo, etc.]. Participants who initially fail the swallow test but are otherwise eligible may re-test at the study site during the screening phase, but they must pass the test by the baseline visit to be eligible to continue in the study.

Type of Participant and Disease Characteristics

Must meet [the criteria for the therapeutic need that is that target of the pediatric clinical development plan] as determined by medical evaluation including [medical history, physical examination, laboratory tests, and other assessments].

Weight

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Common Protocol Template Pediatric Library v001

Minimum [Weight/BMI]

Maximum [Weight/BMI]

Age Definition Comment

Gestational age (GA)

Weeks after start of last menstrual period as determined by history, ultrasound scan, best obstetric estimate, or best neonatal estimate

Post-natal age (PNA) = Chronological age (months and years)

Age since birth This is the most common definition after the first few months, but the other definitions will be relevant in some non-neonatal studies.

Postmenstrual Age

GA + PNA Usually used before expected date of delivery

Corrected Gestational Age

GA + PNA Usually used after expected date of delivery

Sex

2. [Male and/or female]

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a. Male participants:

A male participant must agree to use contraception as detailed in Appendix 4 of this protocol during the treatment period and for at least [X days/weeks, corresponding to time needed to eliminate study intervention for both genotoxic and teratogenic study interventions plus an additional 90 days (a spermatogenesis cycle) for study interventions with genotoxic potential] after the last dose of study intervention and refrain from donating sperm during this period.

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Common Protocol Template Pediatric Library v001

b. Female participants:

A female participant is eligible to participate if she is not pregnant (see Appendix 4), not breastfeeding, and at least one of the following conditions applies:

i. Not a woman of childbearing potential (WOCBP) as defined in Appendix 4

OR

ii. A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 during the treatment period and for at least [X days/weeks,(5 terminal half-lives and, for genotoxic products, an additional 30 days], corresponding to time needed to eliminate study intervention plus 30 days for study interventions with genotoxic potential after the last dose of study intervention.

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Informed Consent

The investigator, or a person designated by the investigator, will obtain written informed consent from each study participant [or the participant's legally acceptable representative, parent(s), or legal guardian] [and the subject’s assent, when applicable,] before any study-specific activity is performed [unless a waiver of informed consent has been granted by an Institutional Review Board (IRB)/Ethics Committee (EC)]. The investigator will retain the original copy of each participant's signed consent[/assent] document.

5.3.3 Activity

6.1 Study Intervention Administered6.1.1 Medical Devices

6.2 Preparation/Handling/Storage/AccountabilityThe excipients used in the pediatric formulation are safe for administration in the pediatric population participating in the study. [Study intervention] will be [self-administered by the child or administered to the child by the parent/caregiver].

6.5 Concomitant Therapy

6.7 Intervention after the End of the Study

8.1 Efficacy Assessments

8.2 Safety Assessments8.2.1 Physical Examinations

A hearing test will be conducted at Screening and [Week X].

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Common Protocol Template Pediatric Library v001

Tanner Staging will be conducted at Screening and [Week X].

8.2.2 Vital Signs

8.2.3 Electrocardiograms

8.2.4 Clinical Safety Laboratory Assessments

8.2.5 Suicidal Risk Monitoring

Families and caregivers of participants must be instructed to monitor participants for the emergence of unusual changes in behavior, as well as the emergence of suicidal ideation and behavior, and to report such symptoms immediately to the investigator. Consideration may be given to discontinuing participants who experience suicidal ideation or behavior. The [name of scale/assessment tool] will be used during the study to [assess or monitor] suicidal ideation and behavior. Refer to [Section X or Appendix X] for more information.

8.3.5 Pregnancy

8.4 Treatment of Overdose

8.9 [Health Economics] OR [Medical Resource Utilization and Health Economics]

9.2 Sample Size Determination

9.4 Statistical Analyses

10.1 Appendix 1: Regulatory, Ethical, and Study Oversight Considerations10.1.1 Regulatory and Ethical Considerations

The investigator will be responsible for reporting cases of suspected child abuse and/or neglect according to local medical association (e.g., AAP) or health department guidelines.

10.1.3 Informed Consent Process

Participants must be informed that their participation is voluntary. Participants or their legally authorized representative [defined as xxx] will be required to provide a statement of informed consent/assent that meets the requirements of 21 CFR 50, local regulations, ICH guidelines, Health Insurance Portability and Accountability Act (HIPAA) requirements, where applicable, and the IRB/IEC or study center.

The medical record must include a statement that legally authorized representative (parent/guardian) consent and child/adolescent assent (if deemed appropriate by local ethics review) was obtained before the participant was enrolled in the study and the date

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Common Protocol Template Pediatric Library v001

the written consent was obtained. The medical record should also describe how the clinical investigator determined that the person signing the ICF was the participant’s legally authorized representative (parent/guardian). The authorized person obtaining the informed consent must also sign the ICF.

Participants and their legally authorized representative (parent/guardian) must be re-consented and re-assented to the most current version of the ICF(s) during their participation in the study.

Minor participants must be re-consented if they reach the age of majority during the course of the study, in order to continue participating.

A copy of the ICF(s) must be provided to the participant or the participant’s parent/guardian.

10.1.4 Data Protection

10.2 Appendix 2: Clinical Laboratory Tests

10.4 Appendix 4: Contraceptive Guidance and Collection of Pregnancy InformationFor a female participant who becomes pregnant, this information will be shared with the study participant’s parent/guardian if the participant’s age is [insert age category or range].

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