A Study of Pralsetinib Versus Standard of Care (SOC) for Treatment of RET-Mutated Medullary Thyroid Cancer (MTC).

Browse trials for

• Thyroid Neoplasms 802
• Carcinoma, Neuroendocrine 236
• Thyroid Diseases 967

Thyroid Neoplasms

802 studies on the registry are indexed under Thyroid Neoplasms; 216 are open to participants now.

Lead sponsor

Hoffmann-La Roche is the lead sponsor of 2,061 studies on the registry; 85 are open to participants now.

Of its 319 completed or terminated interventional studies of FDA-regulated products, 239 (75%) have results posted.

Study arms

• Experimental
  Arm A (Pralsetinib)

  Participants will receive pralsetinib at a dose of 400 milligrams (mg) orally once daily (PO QD) in 28-day cycles.

  Drug: Pralsetinib

• Active comparator
  Arm B (SOC: Cabozantinib/Vandetanib)

  Adult participants will receive investigator's choice of SOC MKI therapy with either 140 mg cabozantinib PO QD or 300 mg vandetanib PO QD in 28-day cycles. Adolescents participants (≥ 12 and \< 18 years of age) will receive vandetanib, PO QD or every other day, in 28-day cycles depending on the body surface area (BSA), at a dose determined according to the dosing nomogram available in the E.U. Vandetanib SmPC.

  Drug: Cabozantinib · Drug: Vandetanib

Interventions

• DrugPralsetinib

  Participants will receive pralsetinib at a dose of 400 mg, as per the dosing schedule described above.

• DrugCabozantinib

  Adult participants will receive cabozantinib at a dose of 140 mg, as per the dosing schedule described above.

• DrugVandetanib

  Adult participants will receive vandetanib at a dose of 300 mg and adolescent participants will receive vandetanib as per the dosing schedule described above.

Primary outcomes

1. Progression-Free Survival (PFS)

   Defined as the time from randomization date to the first documented PD (Progression of Disease), as assessed by BICR according to Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1) or death due to any cause, whichever occurs first.

   Time frame: Up to 5 years

Secondary outcomes

1. Time-To-Treatment Failure (TTF)

   Defined as the time from randomization date to the discontinuation of study drug during the treatment period due to death, treatment-related adverse event, or PD, as assessed by BICR according to RECIST v1.1, whichever occurs first.

   Time frame: Up to 13 years

2. Objective Response Rate (ORR)

   Defined as the proportion of participants who achieve a CR (Complete Response) or a PR (Partial Response) prior to progressive disease and/or other anti-cancer therapy, as assessed by BICR according to RECIST v1.1.

   Time frame: Up to 13 years

3. Overall Survival (OS)

   Defined as the time from randomization date to death due to any cause.

   Time frame: Up to 13 years

4. Percentage of Participants With Adverse Events (AEs)

   Incidence and severity determined according to the NCI CTCAE v5.0 (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0) criteria.

   Time frame: Up to 13 years

5. Duration of Response (DOR)

   Defined as the number of weeks from the time criteria are first met for either a CR or a PR, until the date of documented PD, as assessed by BICR according to RECIST v1.1, or death due to any cause, whichever occurs first.

   Time frame: Up to 13 years

6. Disease Control Rate (DCR)

   Defined as the proportion of participants who experience a best response of CR, PR, or stable disease, as assessed by BICR according to RECIST v1.1.

   Time frame: Up to 13 years

7. Clinical Benefit Rate (CBR)

   Defined as the proportion of participants who experience a CR or a PR or a best response of stable disease with a minimum duration of 6 months, as assessed by BICR according to RECIST v1.1.

   Time frame: Up to 13 years

8. Time to Deterioration of Function

   Defined as the time from randomization to the date of a participant's first \>= 10-point decrease from baseline score, as assessed through the use of the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30).

   Time frame: Up to 13 years

9. Quality of Life (QoL)

   Defined as the time from randomization to the date of a participant's first \>= 10-point decrease from baseline score, as assessed through the use of the GHS (Global Health Status)/QoL scales.

   Time frame: Up to 13 years

10. Acceptability and Palatability of Pralsetinib Capsules

    Acceptability Survey scores on Day 1 of Cycle 1 and Crossover Cycle 1 (each cycle is 28 days).

    Time frame: Up to 13 years

Individual participant data

Plan to share: Yes — Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).