Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Diseases and Abnormalities at or Before Birth

Immune System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Nutritional and Metabolic Diseases

Rare Diseases

Syndrome

Leukemia

Preleukemia

Myelodysplastic Syndromes

Cytopenia

Anemia, Sickle Cell

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Anemia

Lymphoma

Lymphoma, Non-Hodgkin

Thalassemia

Hematologic Neoplasms

Immunologic Deficiency Syndromes

Anemia, Aplastic

Hemoglobinopathies

Primary Immunodeficiency Diseases

Hematologic Diseases

Fanconi Syndrome

Fanconi Anemia

Anemia, Diamond-Blackfan

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Bone Marrow Diseases

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Hemolysis

Anemia, Hemolytic

Anemia, Hemolytic, Congenital

Genetic Diseases, Inborn

Anemia, Hypoplastic, Congenital

Bone Marrow Failure Disorders

Pancytopenia

Congenital Bone Marrow Failure Syndromes

DNA Repair-Deficiency Disorders

Metabolic Diseases

Red-Cell Aplasia, Pure

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Lymphosarcoma

Acute Graft Versus Host Disease

Congenital Aplastic Anemia

Diamond-Blackfan Anemia

Pure Red Cell Aplasia

Rimiducid: to treat uncontrolled GVHD in patients who have received rivogenlecleucel Rimiducid will be given at 0.4 mg/kg weight (intravenous infusion)

No further rivogenlecleucel infusions are planned. Patients who received rivogenlecleucel in the BP-004 study will be evaluated for long-term safety and efficacy.

Rimiducid is administered to treat chronic graft versus host disease

Rimiducid

donor T-cells modified with iCasp safety switch

rivogenlecleucel

Bellicum Pharmaceuticals

This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.

Subjects enrolled on the BP-004 study who have completed or discontinued from the study, and are beyond Day 180 will be requested to enroll on this long-term follow up protocol. Long term follow up for gene therapy clinical and safety endpoints will continue up to 15 years.

Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders

Overall survival (OS) in both malignant and non-malignant subpopulations at 1 and 2 years in the Intent-to-Treat (ITT) Population

KM Parameter Estimates of disease-free survival (DFS) in the non-malignant subpopulation at 1 and 2 years in the Intent-to-Treat (ITT) Population

Kaplan-Meier Parameter Estimates of Relapse-free survival rate (number of patients survived without experiencing a recurrence) at the 1-year and 2-year timepoints in the Intent-to-Treat (ITT) Population in the malignant study arm (patients with a malignant reason for their transplant).

ITT Population: Includes all patients treated with HSCT who received rivogenlecleucel at the dose of 1×10E6 cells/kg

All patients who received HSCT and went on to receive any dose of rivogenlecleucel.

Rivogenlecleucel (BPX-501) Safety Population

All patients who received at least 1 dose of rimiducid for the treatment of acute or chronic GvHD refractory to SoC treatment after rivogenlecleucel administration.

Rimiducid Safety Population

Rimiducid and Rivogenlecleucel

Subject eligibility criteria: patients less than or equal to 18 years of age and greater than or equal to 1 month of age.

Age, Continuous

< 2 years

2 to < 12 years

12 to ≤ 18 years

18 to ≤ 26 years

Age, Customized

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

HSCT safety population: All enrolled patients who received hematopoietic stem cell transplantation

Rivogenlecleucel Study Team

1 Year

2 Years

Malignant

Non-malignant

ITT Population: Includes all patients treated with HSCT who received rivogenlecleucel at the dose of 1×10E6 cells/kg

Study BP-404 is the long-term follow-up study of patients of the BP-004 study who were enrolled at the Italian and Saudi Arabia sites. There is a second long-term follow-up study (BP-004 UK) for patients enrolled at the UK sites. The below results include data from both the BP-404 study and the BP-004 UK study. Individual results for either study are not available.

Overall Survival

Incidence of Disease-free Survival

Relapse-free Survival

Overall Study

Skin and Connective Tissue Diseases

Behaviors and Mental Disorders

Osteosarcoma

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Sarcoma

Quality of Life

Soft Tissue Sarcoma

Sending of 2 questionnaires (SF 36 and TESS)

Quality of life questionnaire

Stéphane SUPIOT, PHD-MD

Surgery and radiation therapy play a major role in the treatment of bone and soft tissue tumors. Osteosarcomas, rhabdomyosarcomas, and Ewing tumors are the most common histologic types. Surgery may require multiple techniques and radiation therapy may be conformational, or more recently IMRT (Intensity Modulated Radiation Therapy). If surgery is possible, lower limb surgery is generally the recommended treatment, even if a poor functional result can be expected.

The literature is very poor regarding the impact of radiotherapy on quality of life and functional results, mainly with the use of prosthetic materials.

Tools such as the Toronto Extremity Salvage Score (TESS) are now available for self-assessment of functional outcomes. The presence of large cohorts such as FCCSS, COHOPER and SALTO facilitates these studies.

The SF-36 is a short 36-question health questionnaire that consists of a generic, consistent, and easy-to-administer set of measures. These measures are based on self-report by patients and are now widely used by organizations managing the care of adult patients.

The TESS Functional Questionnaire is a patient-completed self-questionnaire widely used for motor stimulation in patients with musculoskeletal tumors. This score is based on the definitions of handicap, impairment and handicap as documented by the World Health Organization (WHO). It includes 30 questions assessing overall function and daily activities. The final score varies from 0% to 100%, 100% being the best possible score. So far, several studies have reported the validation of TESS in Portuguese, Danish, Korean, Japanese and since this year in French. Indeed, the TESS questionnaire was validated in French by the study "Transcultural validation of TESS and MSTS questionnaires" promoted by the Nantes University Hospital.

Long-term Evaluation of the Quality of Life of Former Patients Treated With Orthopedic Surgery, With or Without Radiotherapy

Long-term Evaluation of the Quality of Life and the Functional Repercussions of Former Patients Treated With Orthopedic Surgery, With or Without Radiotherapy

Institut Cancerologie de l'Ouest

Digestive System Diseases

Carcinoma

Prostatic Neoplasms

Carcinoma, Hepatocellular

Neoplasms, Glandular and Epithelial

Genital Neoplasms, Male

Urogenital Neoplasms

Genital Diseases

Genital Diseases, Male

Urogenital Diseases

Prostatic Diseases

Male Urogenital Diseases

Adenocarcinoma

Liver Neoplasms

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Liver Diseases

Follow-up for up to 15 years of subjects who received AZD5851 in study NCT06084884

Follow-up for up to 15 years of subjects who received AZD0754 in study NCT06267729

Safety follow-up for up to 15 years of subjects who received AZD5851 in study NCT06084884.

AZD5851

Safety follow-up for up to 15 years of subjects who received AZD0754 in study NCT06267729.

AZD0754

AstraZeneca Clinical Study Information Center

Master LTFU study will monitor the long-term safety and tolerability of cell or gene therapy study participants from AstraZeneca for up to 15 years post last cell or gene therapy treatment.

This is a multi-centre and multinational basket study that will monitor the long-term safety and tolerability of cell or gene therapies in participants that have taken part in other AstraZeneca cell or gene therapy studies for up to 15 years.

LTFU for All Cell and Gene Therapy Studies

A LOng-term Follow-up Master Protocol for Participants Who Received Cell or Gene Therapies in Other AstraZeneca StUdieS (LOCUS)

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal

Vivli.org. All requests will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. "Yes", indicates that AZ are accepting requests for IPD, but this does not mean all requests will be approved.

To determine long-term safety of previous treatment with applicable cell and gene therapy products

To determine long-term efficacy follow-up after previous treatment with a cell or gene therapy product

To characterise the long-term persistence of the cell or gene therapy product.

AstraZeneca

Long term follow-up of subjects with solid or hematological malignancies who have received lentivirus-mediated genetically engineered T Cell Receptors in a previous trial

No study drug is administered in this study. Subjects who received lentivirus-mediated genetically engineered T Cell Receptors in a previous trial will be evaluated in this trial for long-term safety and efficacy.

Genetically engineered T Cell Receptors

Marcus Butler, MD

Subjects who previously took part in an Adaptimmune study and received genetically changed T cells (including but not limited to MAGE-A10ᶜ⁷⁹⁶T and MAGE-A4ᶜ¹º³²T) are asked to take part in this long term follow-up study. Subjects will be asked to join this study once they complete the parent interventional study.

The purpose of this study is to find out if the genetically changed T cells that subjects received in the parent study have any long-term side effects. No additional study drug will be given, but subjects can receive other therapies for their cancer while they are being followed for long term safety in this study.

For a period of 15 years starting from last administration of the genetically changed T cells, subjects will visit their study doctor for a check-up and to have blood tests to look for any changes that might have happened because of the genetically changed T cells.

This is a non-therapeutic, multi-center, long-term follow-up (LTFU) study of subjects who have received lentivirus-mediated genetically engineered T Cell Receptors in an Adaptimmune sponsored clinical trial. The study is designed in accordance with FDA and EMA guidance on gene therapy trials.

The study involves up to 15 years post-infusion monitoring of subjects who have been exposed to lentivirus-mediated gene transfer in Adaptimmune clinical studies. The study will include subjects who have received various T cell receptors including but not limited to MAGE-A10ᶜ⁷⁹⁶T and MAGE-A4ᶜ¹º³²T. Subjects will undergo clinical evaluation (i.e., new medical history, physical exam, adverse events, and exposure to mutagenic agents, anti-cancer therapies and investigational products in other clinical studies) with careful attention to adverse events possibly related to gene transfer or lentivirus-induced diseases. Blood samples will be collected for evaluating persistence of cells with lentiviral vector sequences, the detection of replication competent lentivirus (RCL), and chemistry and hematology laboratory assessments. Subjects will be followed for survival.

Long Term Follow up of Subjects Exposed to Genetically Engineered T Cell Receptors

Long Term Follow-up of Subjects Exposed to Genetically Engineered Tumor Antigen Specific T Cell Receptors

* New malignancies
* New incidence or exacerbation of a pre-existing neurologic disorder
* New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder
* New incidence of a hematologic disorder
* Opportunistic and/or serious infections
* Unanticipated illness and/or hospitalization deemed related to gene modified cell therapy

Subjects' peripheral blood samples will be used to evaluate RCL

Peripheral blood samples will be used to evaluate persistence

Number of samples positive for vector integration by PCR

OS defined as the interval between the date of first T cell infusion and date of death due to any cause

Adaptimmune

No study drug is administered in this study. Participants who received GSK adoptive cell therapy in a previous trial will be evaluated in this trial for long-term safety and efficacy.

GSK adoptive cell therapy

US GSK Clinical Trials Call Center

EU GSK Clinical Trials Call Center

EU GSK Clinical Trials Call Centre

Melinda Yushak

Aaron Rapoport

Scott Schuetze

Brian Van Tine

Sandra P D'Angelo

Richard Riedel

Lara Davis

Edward A Stadtmauer

Melissa Burgess

Theodore Laetsch

Dejka Araujo

John A Charlson

Jayesh Desai

Adrian Sacher

Jonathan Noujaim

Lars Lindner

Roland Ullrich

Silvia Stacchiotti

John B.A.G. Haanen

Enriqueta Felip

Victor Moreno Garcia

Emiliano Calvo Aller

Pilar Sancho Márquez

Sandra Strauss

Fiona Thistlethwaite

Andrew Furness

GSK Clinical Trials

This trial will evaluate long term safety of participants who have received GlaxoSmithKline (GSK) adoptive cell therapy for up to 15 years following last adoptive cell therapy infusion.

Participants who received a GSK adoptive cell therapy will be enrolled in this non-therapeutic, multi-center, long term follow-up (LTFU) study and will be followed for up to 15 years post-infusion of lentiviral vector-based adoptive cell therapy. Participants will be monitored for safety following last adoptive cell therapy infusion.

Adoptive Cell Therapy Long-term Follow-up (LTFU) Study

Long-Term Follow-Up (LTFU) of Participants Treated With GSK Adoptive Cell Therapies

IPD for this study will be made available via the Clinical Study Data Request site.

Peripheral blood samples will be collected for the assessment of VSV-G DNA copies.

Peripheral blood samples will be collected for the assessment of WPRE or Psi DNA.

Peripheral blood samples will be collected for the assessment of Integrated vector sequences and vector integration patterns (e.g., polyclonal, oligoclonal, or monoclonal).

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study	NCT03733249	Acute Lymphobla... Leukemia, Acute... Lymphoma, Non-H... Myelodysplastic... Primary Immunod... Anemia, Aplasti... Hemoglobinopath... Cytopenia Fanconi Anemia Diamond Blackfa... Thalassemia Anemia, Sickle ...	Rimiducid rivogenlecleuce...	1 Month - 18 Years	Bellicum Pharmaceuticals
Long-term Evaluation of the Quality of Life of Former Patients Treated With Orthopedic Surgery, With or Without Radiotherapy	NCT04644913	Osteosarcoma in...	Quality of life...	18 Years -	Institut Cancerologie de l'Ouest
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study	NCT03733249	Acute Lymphobla... Leukemia, Acute... Lymphoma, Non-H... Myelodysplastic... Primary Immunod... Anemia, Aplasti... Hemoglobinopath... Cytopenia Fanconi Anemia Diamond Blackfa... Thalassemia Anemia, Sickle ...	Rimiducid rivogenlecleuce...	1 Month - 18 Years	Bellicum Pharmaceuticals
LTFU for All Cell and Gene Therapy Studies	NCT06194461	Hepatocellular ... Prostate Cancer	AZD5851 AZD0754	18 Years - 130 Years	AstraZeneca
Long-term Evaluation of the Quality of Life of Former Patients Treated With Orthopedic Surgery, With or Without Radiotherapy	NCT04644913	Osteosarcoma in...	Quality of life...	18 Years -	Institut Cancerologie de l'Ouest
Long Term Follow up of Subjects Exposed to Genetically Engineered T Cell Receptors	NCT03391791	Solid and Hemat...	Genetically eng...	18 Years -	Adaptimmune
Adoptive Cell Therapy Long-term Follow-up (LTFU) Study	NCT03391778	Neoplasms	GSK adoptive ce...	-	GlaxoSmithKline
Natural History of NFAI: 10 Year Follow-up Results	NCT04616703	Adrenal Inciden...	CT imaging	18 Years -	University Medical Centre Ljubljana
Natural History of NFAI: 10 Year Follow-up Results	NCT04616703	Adrenal Inciden...	CT imaging	18 Years -	University Medical Centre Ljubljana
Long Term Follow up of Subjects Exposed to Genetically Engineered T Cell Receptors	NCT03391791	Solid and Hemat...	Genetically eng...	18 Years -	Adaptimmune

Spots Global Cancer Trial Database for long term follow up