Phase 2b of RAPA-201 Cell Therapy in Post-PD-(L)-1 Melanoma

NCT06708455 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: RAPA-201-MEL-PH2-01
• Study Type: interventional
• Target: 65
• Locations: 1 country
• Sites: 1

Brief Summary

The protocol is a Simon's 2-stage, non-randomized, open label, multi-site, phase 2 trial for patients with advanced metastatic, recurrent and unresectable malignant melanoma that has recurred or relapsed after prior anti-PD-(L)1 therapy.

Conditions

Malignant Melanoma Stage IIIc; Malignant Melanoma Stage IV; Malignant Melanoma With Metastasis; Malignant Melanoma

Keywords

Adoptive T Cell Therapy; Rapamycin; Regenerative Medicine; Malignant Malanoma; RMAT

Outcome Measures

Primary Outcomes (1)

• Efficacy of RAPA-201 Cell Therapy

  In an intent-to-treat (ITT) manner, the efficacy of RAPA-201 cell therapy will be determined by overall response rate (ORR) in metastatic melanoma patients with progressive disease after anti-PD-(L)1 therapy, as assessed by the independent review committee (IRC) per iRECIST v1.1.

  18 months (6 mo. of treatment; 12 mo of clinical follow-up)

Secondary Outcomes (3)

• Characterize RAPA-201 Efficacy

  One (1) year after the last dose of RAPA-201 cells.

• Safety of RAPA-201 Cell Therapy

  One (1) year after the last dose of RAPA-201 cells.

• Quality of Life (QOL)

  One (1) year after the last dose of RAPA-201 cells.

Other Outcomes (1)

• Immune Reconstitution

  One (1) year after the last dose of RAPA-201 cells.

Study Arms (1)

Administration of RAPA-201 cells

EXPERIMENTAL

RAPA-201 cells will be administered at a target flat dose between 80 and 400 x 10\^6 cells per infusion.

Biological: RAPA-201 Rapamycin Resistant T Cells; Drug: Chemotherapy Prior to RAPA-201 Therapy

Interventions

RAPA-201 Rapamycin Resistant T Cells BIOLOGICAL

Autologous Rapamycin-Resistant Th1/Tc1 Cells

Administration of RAPA-201 cells

Chemotherapy Prior to RAPA-201 Therapy DRUG

Carboplatin + Paclitaxel Regimen (CP Regimen)

Administration of RAPA-201 cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients ≥ 18 years of age.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 and an estimated life expectancy of ≥ 3 months.
• Patients with unresectable or metastatic melanoma (Stage IIIc or Stage IV).
• Prior to enrollment, documented refractory status to the most recent regimen, which must include an anti-PD-(L)1 monoclonal antibody, as defined by lack of response after at least two cycles of therapy or relapse within 12-months of initiation of the anti-PD- (L)1-containing therapy.
• For patients with BRAF V600 mutation-positive tumors, prior therapy with a BRAF inhibitor alone or in combination with a MEK inhibitor.
• Presence of measurable disease to permit monitoring by iRECISTv1.1 Criteria.
• Must have a potential source of autologous T cells potentially sufficient to manufacture RAPA-201 cells, as defined by a circulating absolute lymphocyte count (ALC) of ≥ 500 cells/μL.
• Patients must be ≥ two weeks from last solid tumor cancer chemotherapy, major surgery, radiation therapy and/or participation in investigational trials.
• Patients must have recovered from clinical immunotherapy-related toxicities \[resolution of CTCAE (v5) toxicity to a value of ≤ 1; with the exception of alopecia, vitiligo, and endocrinopathy stable on hormone replacement\].
• Hematologic parameters of: Absolute neutrophil count (ANC) of ≥ 1500 cells/μL, Platelet count ≥ 100,000 cells/μL, and Hemoglobin of ≥ 9 grams/μL.
• Calculated creatinine clearance of ≥ 40 mL/min.
• Ejection fraction (EF) by MUGA or 2-D echocardiogram within institution normal limits, with an EF level of ≥ 45%.
• Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) ≤ 3 x upper limit of normal (ULN) (or ≤ ULN if patient has liver metastasis).
• Bilirubin ≤ 2.0 mg/dL (if Gilbert\'s disease, ≤ 3.0 mg/dL).
• No history of abnormal bleeding tendency (as defined by any inherited coagulation defect or history of internal bleeding).

You may not qualify if:

• Other active malignancy (except non-melanoma skin cancer).
• Life expectancy \< 3 months.
• Seropositivity for HIV, hepatitis B, or hepatitis C, unless such conditions are in stable condition using adequate treatment.
• Uncontrolled hypertension.
• Cerebrovascular accident within 6 months of enrollment.
• Myocardial infarction within 6 months of enrollment.
• NYHA class III/IV congestive heart failure.
• Uncontrolled angina/ischemic heart disease.
• Cancer metastasis to the central nervous system, unless such metastasis has been adequately treated.
• Pregnant or breastfeeding patients.
• Women of childbearing potential, or males who have a partner of childbearing potential, who are unwilling to practice contraception.
• Patients may be excluded at the discretion of the PI or if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk.

Sponsors & Collaborators

• Rapa Therapeutics LLC: lead

Study Sites (1)

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

MeSH Terms

Conditions

Melanoma

Condition Hierarchy (Ancestors)

Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Nevi and Melanomas; Skin Neoplasms; Neoplasms by Site; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Daniel Fowler, M.D.

  Rapa Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Daniel Fowler, M.D.

CONTACT

(301)-518-3104; dan@rapatherapeutics.com

Jennifer Sunga

CONTACT

jsunga@rapatherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2024
• First Posted: November 27, 2024
• Study Start (Estimated): March 1, 2027
• Primary Completion (Estimated): March 1, 2029
• Study Completion (Estimated): September 30, 2029
• Last Updated: March 19, 2026

Record last verified: 2026-03

Locations

US (1)