Phase I clinical trial failures

Phase I Non-CNS Tumor trial by Genmab. Stop reason: EFFICACY/FUTILITY.

After reviewing all available data from Part 1 of the trial, the Sponsor has decided not to proceed to Part 2 (randomized Phase 2) of the trial due to lack of efficacy

Phase I Malaria trial by National Institute of Allergy and Infectious Diseases (NIAID). Stop reason: REGULATORY.

The study remains on hold due to a funding issue after it's been lifted by the FDA.

Phase I Borderline Resectable Pancreatic Adenocarcinoma trial by University of Utah. Stop reason: OPERATIONAL.

Closed due to slow accrual

Phase I Non-Small Cell Lung Cancer trial by National Cancer Institute (NCI). Stop reason: OPERATIONAL.

Revisions to eligibility/design being made prior to enrollment of first participants.

Phase I Advanced Solid Tumor trial by Selecxine. Stop reason: OPERATIONAL.

Business decision

Phase I Multiple Myeloma trial by National Cancer Institute (NCI). Stop reason: OPERATIONAL.

Administrative hold pending non-safety related changes to the study design.

Phase I Metastatic Pancreatic Ductal Adenocarcinoma trial by Fred Hutchinson Cancer Center. Stop reason: OPERATIONAL.

Study terminated due to end of funding.

Phase I Recurrent Plasma Cell Myeloma trial by City of Hope Medical Center. Stop reason: OPERATIONAL.

Slow/Low Accrual

Phase I Acute Lymphoblastic Leukemia trial by City of Hope Medical Center. Stop reason: OPERATIONAL.

Accrual goal

Phase I Recurrent Acute Myeloid Leukemia trial by City of Hope Medical Center. Stop reason: EFFICACY/FUTILITY.

Pending Interim Analysis

Phase I Pancreatic Cancer trial by M.D. Anderson Cancer Center. Stop reason: OPERATIONAL.

PI Request

Phase I Advanced Colorectal Carcinoma trial by University of California, Davis. Stop reason: EFFICACY/FUTILITY.

For internal interim analysis

Phase I Cutaneous Melanoma trial by Seagen, a wholly owned subsidiary of Pfizer. Stop reason: OPERATIONAL.

The trial was terminated for strategic reasons. The decision was not based on any safety concerns.

Phase I HER2-positive Breast Cancer trial by University of Alabama at Birmingham. Stop reason: OPERATIONAL.

Unable to recruit for this study.

Phase I Prostate Cancer trial by Washington University School of Medicine. Stop reason: OPERATIONAL.

Low accrual

Phase I Glioma, Malignant trial by Northwestern University. Stop reason: SAFETY.

Safety issues

Phase I High Risk Neuroblastoma trial by National Cancer Institute (NCI). Stop reason: OPERATIONAL.

Drug supply issues

Phase I Leukodystrophy, Globoid Cell trial by Gemma Biotherapeutics. Stop reason: OPERATIONAL.

The study was stopped due to changes in the company strategy.

Phase I Rectal Neoplasms trial by University of Iowa. Stop reason: SAFETY.

Toxicity in phase 1

Phase I Diastolic Dysfunction trial by Texas Tech University Health Sciences Center. Stop reason: OTHER/UNKNOWN.

Had issue enrolling subjects.

Phase I Metastatic Castration-resistant Prostate Cancer trial by University of Utah. Stop reason: OTHER/UNKNOWN.

DSMC Safety Analysis

Phase I Pain trial by University of Utah. Stop reason: OTHER/UNKNOWN.

Study is paused while preparing for implementation at IND Sponsor's new institution

Phase I Solid Tumor, Adult trial by Virginia Commonwealth University. Stop reason: OTHER/UNKNOWN.

Pending amendment approval

Phase I Glioblastoma Multiforme of Brain trial by Chimeric Therapeutics. Stop reason: OTHER/UNKNOWN.

IND application withdrawn

Phase I Advanced Adenocarcinoma trial by University of California, Davis. Stop reason: SAFETY.

Drugs unavailable

Phase I Healthy trial by Pfizer. Stop reason: OPERATIONAL.

The study was terminated for business reasons; there were no safety concerns.

Phase I Prostatic Neoplasms trial by Eli Lilly and Company. Stop reason: EFFICACY/FUTILITY.

The study was terminated early as related study I3Y-MC-JPCM (NCT03706365) did not meet its primary endpoint.

Phase I Lymphoma, Non-Hodgkin trial by Bristol-Myers Squibb. Stop reason: OTHER/UNKNOWN.

No stop-reason text available in the compact dataset.

Phase I Prostatic Neoplasms trial by National Cancer Institute (NCI). Stop reason: OPERATIONAL.

Slow/insufficient accrual

Phase I Coronavirus trial by Celularity Incorporated. Stop reason: OPERATIONAL.

Lack of enrollment due to COVID treatment options availability.

Phase I Acute Myeloid Leukemia trial by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. Stop reason: OTHER/UNKNOWN.

Upon analysis of the first 7 participants accrued, it was determined that this combination treatment is not efficacious as treatment for patients with relapsed or refractory AML

Phase I Cutaneous Squamous Cell Carcinoma (CSCC) trial by Ankyra Therapeutics, Inc. Stop reason: OTHER/UNKNOWN.

Strategic realignment

Phase I Relapsing Multiple Sclerosis (RMS) trial by Cabaletta Bio. Stop reason: OPERATIONAL.

The study was withdrawn prior to site activation and before enrollment of any participants due to sponsor decision to reprioritize the development portfolio.

Phase I Anatomic Stage III Breast Cancer AJCC v8 trial by National Cancer Institute (NCI). Stop reason: OTHER/UNKNOWN.

Other - DLT assessment and phase 2 dose determination

Phase I Infertility trial by ART Fertility Clinics LLC. Stop reason: OPERATIONAL.

Difficulty of enrolling and recruiting patients

Phase I Metastatic Castration-Resistant Prostate Carcinoma trial by National Cancer Institute (NCI). Stop reason: OPERATIONAL.

Other - To assess the study's feasibility.

Phase I Lymphocyte B CD22 Positive Acute Lymphoblastic Leukemia trial by Nantes University Hospital. Stop reason: OTHER/UNKNOWN.

No stop-reason text available in the compact dataset.

Phase I Carcinoma, Non-Small-Cell Lung trial by Seagen, a wholly owned subsidiary of Pfizer. Stop reason: OPERATIONAL.

The trial was terminated for strategic reasons. The decision was not based on any safety concerns

Phase I Multiple Sclerosis (MS) trial by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology. Stop reason: OTHER/UNKNOWN.

Legend discontinues the development of the IMP.

Phase I Pancreatic Cancer Metastatic trial by HonorHealth Research Institute. Stop reason: EFFICACY/FUTILITY.

The study was terminated early per the recommendation of the Data and Safety Monitoring Board (DSMB) after a planned interim analysis met pre-specified criteria for futility.

Phase I Solid Tumors trial by Boehringer Ingelheim. Stop reason: OPERATIONAL.

Sponsor decision

Phase I Ovarian Cancer trial by Novartis Pharmaceuticals. Stop reason: OPERATIONAL.

Study termination based on sponsor decision due to lack of tolerability observed with \[177Lu\]Lu-EVS459

Phase I Lung Cancer trial by Shenzhen Geno-Immune Medical Institute. Stop reason: OTHER/UNKNOWN.

combine with NCT03356808

Phase I Healthy Adult Volunteers trial by NIBEC Co., Ltd.. Stop reason: OPERATIONAL.

Study was terminated at the end of Part A for strategic reasons (not due to lack of efficacy or safety issues

Phase I Advanced Solid Tumors trial by Debiopharm International SA. Stop reason: OPERATIONAL.

This trial was concluded for strategic reasons.

Phase I Advanced HR+/HER2- Breast Cancer trial by Novartis Pharmaceuticals. Stop reason: OPERATIONAL.

Business decision

Phase I Solid Tumors trial by Amgen. Stop reason: OPERATIONAL.

Sponsor Decision

Phase I GBA1 Parkinson Disease trial by Capsida Biotherapeutics, Inc.. Stop reason: OTHER/UNKNOWN.

Per protocol stopping rule was met for a separate trial for a different disease state and different experimental therapy that utilized a similar technology.

Phase I Advanced Hepatocellular Carcinoma trial by Roswell Park Cancer Institute. Stop reason: OTHER/UNKNOWN.

awaiting agreement with Sponsor

Phase I Head and Neck Cancer trial by Case Comprehensive Cancer Center. Stop reason: OTHER/UNKNOWN.

PI leaving the institution

Phase I Severe Aplastic Anemia (SAA) trial by Regeneron Pharmaceuticals. Stop reason: OPERATIONAL.

Sponsor Decision

Phase I Cerebral Adrenoleukodystrophy trial by Masonic Cancer Center, University of Minnesota. Stop reason: OTHER/UNKNOWN.

Awaiting supplies

Phase I Desmoplastic Small Round Cell Tumor trial by National Cancer Institute (NCI). Stop reason: OPERATIONAL.

Other - Stage 1 has met accrual

Phase I Cancer trial by Biond Biologics. Stop reason: OTHER/UNKNOWN.

Study terminated due to strategic considerations

Phase I Advanced Solid Tumor trial by Cullinan Therapeutics Inc.. Stop reason: OPERATIONAL.

Company decision

Phase I HER2-positive Breast Cancer trial by H. Lee Moffitt Cancer Center and Research Institute. Stop reason: OTHER/UNKNOWN.

Wait for the DLT period with our current patient and will not be enrolling any more.

Phase I HER2-positive Breast Cancer trial by Day One Biopharmaceuticals, Inc.. Stop reason: OTHER/UNKNOWN.

Development discontinued.

Phase I Sickle Cell Disease trial by Fulcrum Therapeutics. Stop reason: OPERATIONAL.

Sponsor decision

Phase I Ovarian Cancer trial by NewLink Genetics Corporation. Stop reason: OPERATIONAL.

Company decision

Phase I Breast Cancer trial by M.D. Anderson Cancer Center. Stop reason: SAFETY.

Toxicity and Lack of Efficacy

Phase I SCLC trial by Y-mAbs Therapeutics. Stop reason: OTHER/UNKNOWN.

Decision by Sponsor

Phase I Metastatic Malignant Solid Neoplasm trial by National Cancer Institute (NCI). Stop reason: OTHER/UNKNOWN.

Other - Correlative sample analysis.

Phase I Advanced Malignant Solid Neoplasm trial by National Cancer Institute (NCI). Stop reason: OTHER/UNKNOWN.

Other - Amendment needed to add new dose level.

Phase I Brain Metastases trial by Medical College of Wisconsin. Stop reason: OPERATIONAL.

Study never started.

Phase I Metastatic Colorectal Adenocarcinoma trial by National Cancer Institute (NCI). Stop reason: OTHER/UNKNOWN.

Other - Pending amendment.

Phase I Perinatal Arterial Ischemic Stroke trial by James Baumgartner, MD. Stop reason: SAFETY.

The FDA required a clinical hold for the IND which remained until it was determined that the study would be closed.

Phase I Hepatitis B trial by GlaxoSmithKline. Stop reason: OPERATIONAL.

The study was terminated as a strategic decision

Phase I Relapsed/Refractory Multiple Myeloma trial by Second Affiliated Hospital of Xi'an Jiaotong University. Stop reason: OTHER/UNKNOWN.

Achieved the proof of concept

Phase I Multi-Drug Resistant Nephrotic Syndrome trial by The Children's Hospital of Zhejiang University School of Medicine. Stop reason: OPERATIONAL.

no participants enrolled

Phase I Cancer trial by Baptist Health South Florida. Stop reason: OPERATIONAL.

Funding

Phase I Relapsed/Refractory Autoimmune Diseases trial by Nanjing Legend Biotech Co.. Stop reason: OTHER/UNKNOWN.

pipeline adjustment

Phase I Solid Tumor trial by Servier Bio-Innovation LLC. Stop reason: OPERATIONAL.

Sponsor decision to terminate during Phase 1

Phase I Hemophilia B trial by Be Biopharma. Stop reason: OPERATIONAL.

The study was terminated due to a strategic business decision and not due to safety concerns

Phase I Stage III Pancreatic Cancer AJCC v8 trial by City of Hope Medical Center. Stop reason: OPERATIONAL.

Accrual Goal met

Phase I Cutaneous T Cell Lymphoma trial by University of Alabama at Birmingham. Stop reason: OTHER/UNKNOWN.

IRB audit

Phase I Healthy Participants trial by UCB Biopharma SRL. Stop reason: OPERATIONAL.

sponsor decision

Phase I Advanced Solid Tumors trial by Vividion Therapeutics, Inc.. Stop reason: OTHER/UNKNOWN.

The study has ended

Phase I Diabetes Mellitus, Type 2 trial by McMaster University. Stop reason: OTHER/UNKNOWN.

Withdrawn

Phase I Advanced Solid Tumor trial by SpringWorks Therapeutics, Inc.. Stop reason: OTHER/UNKNOWN.

Lack of tolerability of BGB-3245 (Brimarafenib) when given in combination with Mirdametinib

Phase I Acute Myeloid Leukemia trial by Northwestern University. Stop reason: OTHER/UNKNOWN.

Premature withdrawal of investigational product per study sponsor.

Phase I Multiple Myeloma trial by Thomas Martin, MD. Stop reason: OTHER/UNKNOWN.

Safety Review

Phase I Glioblastoma Multiforme trial by Genenta Science. Stop reason: OTHER/UNKNOWN.

LVLP (pt GBM-01034) took place on 27may2026. The 2 remaining slots allocated by the protocol for the additional cohort to generate safety data prior to study Part B will not be filled. No safety concerns. Benefit-risk profile unchanged.

Phase I Advanced Solid Tumors trial by Astellas Pharma Global Development, Inc.. Stop reason: OTHER/UNKNOWN.

Lack of clinical benefit

Phase I Anemia, Sickle Cell trial by Columbia University. Stop reason: OPERATIONAL.

Closed by the industry collaborator

Phase I Constitutional Mismatch Repair Deficiency Syndrome trial by National Cancer Institute (NCI). Stop reason: OPERATIONAL.

Inadequate accrual rate

Phase I Developmental and Epileptic Encephalopathy trial by Capsida Biotherapeutics, Inc.. Stop reason: OTHER/UNKNOWN.

Stopping rule for study was met

Phase I Periorbital Burns trial by Virginia Commonwealth University. Stop reason: OTHER/UNKNOWN.

Inventory not available

Phase I Solid Tumor trial by Boundless Bio, Inc.. Stop reason: OPERATIONAL.

Strategic decision made following an assessment of preliminary PK data from the Part 1 portion of the trial showing a lack of dose proportional exposure and the increasing complexity and associated development costs in this landscape.

Phase I Triple Negative Breast Cancer (TNBC) trial by Boundless Bio, Inc.. Stop reason: OPERATIONAL.

Decision was made to halt the study based on overall clinical experience and market considerations. This decision was not driven by any safety signal.

Phase I Astrocytoma, Grade IV trial by Celularity Incorporated. Stop reason: OPERATIONAL.

No participants were enrolled. Study was withdrawn prior to treatment initiation.

Phase I Atypical Choroid Plexus Papilloma trial by Weill Medical College of Cornell University. Stop reason: OPERATIONAL.

Study terminated due to low accrual

Phase I Sleep Disorders trial by University of California, San Francisco. Stop reason: OPERATIONAL.

Lack of funding

Phase I Ovarian Cancer Recurrent trial by University of Alabama at Birmingham. Stop reason: OTHER/UNKNOWN.

never opened

Phase I Recurrent Chronic Myelomonocytic Leukemia trial by M.D. Anderson Cancer Center. Stop reason: OTHER/UNKNOWN.

\<75% participation

Phase I Atopic Dermatitis trial by Yellow Jersey Therapeutics AG. Stop reason: OTHER/UNKNOWN.

Asset acquisition by Johnson \& Johnson. Minimum study objectives necessary to inform safety, tolerability, and dose selection for Phase 2 dose-ranging evaluation of NM26-2198 were achieved, with full completion of all healthy volunteer cohorts.

Phase I Acute Myeloid Leukaemia trial by Sanofi. Stop reason: OPERATIONAL.

Sponsor's decision

Phase I Pediatric Acute Leukemia trial by M.D. Anderson Cancer Center. Stop reason: OPERATIONAL.

PI requested

Phase I Distal Bile Duct Adenocarcinoma trial by City of Hope Medical Center. Stop reason: EFFICACY/FUTILITY.

Pending Interim analysis

Phase I Lung Cancer trial by University of Nebraska. Stop reason: OPERATIONAL.

Poor accrual

Phase I Recurrent Acute Lymphoblastic Leukemia trial by City of Hope Medical Center. Stop reason: EFFICACY/FUTILITY.

Pending Interim Analysis