The objectives for Amendment 1 were: 1. To update the Background Information based on the final results obtained from the RP103-01 pilot study. 2. To revise the protocol design of version 1.1 to incorporate the following changes:  - Monthly visits for at least six (6) months for each subject followed by synchronized quarterly visits.  - Modify and clarify inclusion and exclusion criteria.  - Eliminate the GSRS from the study. - Utilize the self-report PedsQL QoL instrument instead of the parent proxy report. - Addition of safety and PK/PD data reviews, during or prior to, each study visit to allow for greater subject oversight for dose adjustments. 3. To update the bioanalytical laboratory information. - Provide specific information concerning where samples are to be sent in the US and Europe. - To update the plasma cysteamine and WBC processing instructions. 4. To update the list of abbreviations. 5. To clarify statements and instructions concerning protocol specified procedures. 6. To update administrative information. 7. To update the reference list. 8. To correct typographical and formatting errors.

The objectives for Amendment 2 were: 1. To update the protocol to synchronize with changes incorporated in the precursor pivotal study RP103-03 Amendment 2, which included: - Allowance for subjects that are receiving Cystagon® at the conclusion of study RP103-03 to roll over into this study without the need for a Day 1 study visit.  - Clarification that WBC cystine values <1 nmol ½ cystine/mg protein represented a meaningful reduction in WBC cystine levels.  - Changing the time for RP103 trough sampling (i.e., 1 hour changed to 0.5 hours post RP103 dose administration). 2. To update and correct the inclusion and exclusion criteria. 3. To update the list of abbreviations. 4. To correct and update the total amount of blood collected over the study duration. 5. To correct typographical and formatting errors.

The objectives for Amendment 3 were: 1. To revise the protocol to restrict enrollment for subjects that did not participate in the precursor study (RP103-03) to occur only after all subjects enrolled in Study RP103-03 have completed their participation in that study and the data analyzed. 2. To add stopping criteria consistent with Study no. RP103-03. 3. To correct the list of safety endpoints. 4. To update the list of abbreviations. 5. To update the responsible party for medical monitoring. 6. To clarify inclusion criteria language. 7. To make language consistency changes between protocols RP103-03 and RP103-04. 8. To correct typographical and formatting errors.

The objectives for Amendment 4 were: 1. To change the regular daily dose of Cystagon® to 80% (instead of 70% previously)and a dose adjustment to 100% of their regular daily dose of Cystagon® (instead of 20to 25% of the dose of RP103). 2. To clarify instructions for subjects not entering the RP103-04 trial from Study RP103-03. 3. To describe a food effect on cysteamine absorption. 4. To include total protein test method comparison and describe the correction factor. 5. To include the updated prefigured guideline for the dosage of the subjects. 6. To update one table and include two new figures. 7. To update the List of Abbreviations. 8. To clarify statements and instructions. 9. To correct formatting errors.

The objectives for Amendment 5 were: 1. To include published results of recently completed clinical trials: - Bioequivalence studies RP103-02 and RP103-05, in healthy volunteers. - Pivotal Phase 3 study RP103-03, in cystinosis subjects. 2. To provide final data and guidance in the following areas, which in the previous protocol amendment were based on interim results of the recently completed clinical trials: - Starting total daily dose of RP103 changed to 70% of the stable baseline - Cystagon® dose and subsequent dose adjustment increased to 100% of that stable Cystagon® dose. - Food effect on cysteamine absorption has been detailed.  - RP103 dosing recommendations with respect to food intake and timing have been updated. 3. To expand enrollment to subjects unable or unwilling to take intact capsules, the following areas were revised:  - Total number of expected study participants has been increased. - Inclusion and Exclusion criteria were updated to permit enrollment of subjects who do not receive their cysteamine dose as intact capsules, and those who receive it via gastric tube. 4. To change the Schedule of Events for subjects not entering the trial from Study RP103-03, adding a Dose Confirmation Period which includes study visits on Day 4 and Day 5. 5. To change all references to “nephropathic cystinosis” to simply “cystinosis” 6. To clarify statements and instructions. 7. To correct formatting and typographical errors.

The objectives for Amendment 6 were: 1. To insert a minimum required age for study participants; 2. To increase maximum study duration from 24 to 36 months; 3. To revise investigational product description to reflect currently manufactured lots; 4. To revise maximum blood volume to be collected from subjects, reflecting the change in maximum study duration to 36 months and to include the optional PK substudy; 5. To insert an optional PK substudy visit for all subjects 6 years old and younger; 6. To insert a newly published reference (previously reported as “submitted for publication”); 7. To update the bioanalytical laboratory contact details to reflect that all PK-PD samples were to be shipped to the US location (because the EU laboratory had closed); 8. To clarify statements and instructions, specifically pertaining to RP103 dosing; 9. To correct formatting and typographical errors.

The objectives for Amendment 7 were: 1. To insert changes to Sponsor company name and contact details; 2. To insert a change in Institution and contact details for the Lead Investigator; 3. To increase the maximum study duration to 48 months; 4. To provide updated information regarding the acceptable storage temperature of the investigational product, RP103; 5. To revise maximum blood volume to be collected from subjects, reflecting the change in maximum study duration to 48 months; 6. To remove references to the specific central bioanalytical laboratory “BASi” in the event that a different laboratory would be utilized in future (in which case the site laboratory instructions would be updated); 7. To clarify statements and instructions, specifically pertaining to RP103 dosing; 8. To correct minor formatting and typographical errors.

The objectives for Amendment 8 were: 1. To document a change in Sponsor Medical Officer; 2. To document a change in the Sponsor medical representative responsible for receipt of serious adverse event (SAE) reports; 3. To insert changes to Sponsor physical address; 4. To increase the maximum study duration to 60 months; 5. To insert the EudraCT number associated with the trial; 6. To revise maximum blood volume to be collected from subjects, reflecting the change in maximum study duration to 60 months; 7. To correct minor formatting and typographical errors.

The purpose of RP103-04 Protocol Amendment 9 were as follows: - To update Raptor Chief Medical Officer; - To clarify study duration; - To update anticipated blood collection volumes for the entire study based on clarification to study duration; - To clarify Adverse Event reporting; - Update reference to Storage Conditions and Identity of Investigational Product (RP103); - To correct formatting and typographical errors.