21 Spinal Muscular Atrophy Clinical Trials in Philadelphia, PA (2026)

Q: Are there spinal muscular atrophy clinical trials in Philadelphia?

Yes, there are 21 spinal muscular atrophy clinical trials currently recruiting in Philadelphia, PA.

Q: How do I join a clinical trial in Philadelphia?

Browse available studies, click one that interests you, and complete the free eligibility form. A study coordinator at the Philadelphia research site will contact you.

Q: Are clinical trials in Philadelphia free?

Yes, clinical trial participation is always free. Study-related treatments, medical tests, and doctor visits are provided at no cost.

Recruiting Spinal Muscular Atrophy Studies in Philadelphia

NCT07037758Recruiting

A Study of Tarlatamab in Combination With AB248 in Participants With Extensive Stage Small Cell Lung Cancer (DeLLphi-311)

The primary objective for dose exploration and dose expansion is to evaluate the safety and tolerability of tarlatamab in combination with AB248. The primary objective for dose exploration only is to...

Philadelphia, PAPHASE1380 participantsAmgen

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NCT06043817Recruiting

First-In-Human Study of STX-721 in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer Harboring EGFR Exon 20 Insertion Mutations

Study STX-721-101 is an open label, Phase 1/2 study evaluating the safety, tolerability, pharmacokinetic (PK) exposure, and preliminary antitumor activity of STX-721 in participants with non-small cel...

Philadelphia, PAPHASE1345 participantsAntares Therapeutics, Inc

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NCT07174583Recruiting

A Study of IDE849 in Patients With DLL3 Expressing Tumors Including Small Cell Lung Cancer

This is Phase 1/2, multicenter, clinical study to evaluate the safety, efficacy, PK, and immunogenicity of IDE849 in subjects with DLL3-expressing tumors including SCLC....

Philadelphia, PAPHASE1262 participantsIDEAYA Biosciences

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NCT06681220Recruiting

Biomarker Directed Trial of Temozolomide and Stenoparib in Relapsed SCLC

Randomized phase 2, multicenter, biomarker directed clinical trial with a safety lead-in to assess the efficacy of Stenoparib plus Temozolomide (TMZ) in relapsed Small Cell Lung Cancer patients. Parti...

Philadelphia, PAPHASE1166 participantsVA Office of Research and Development

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NCT07458347Recruiting

A Phase 1 Dose-escalation Trial of KST-6051 in Participants With Advanced Solid Tumors With Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) Mutation

The main purpose of the trial is to assess whether the trial drug, KST-6051, is safe and tolerable when administered orally to adults with advanced or metastatic solid tumors with certain KRAS mutatio...

Philadelphia, PAPHASE1145 participantsKestrel Therapeutics, Inc.

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NCT06116786Recruiting

A Study of JNJ-86974680 in Participants With Advanced Non-small Cell Lung Cancer

The purpose of this study is to determine a safe and tolerable dose(s) of JNJ-86974680 for further research in combination with cetrelimab and radiation therapy....

Philadelphia, PAPHASE1126 participantsJohnson & Johnson Enterprise Innovation Inc.

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NCT07216105Recruiting

FT836 With or Without Chemotherapy and/or Monoclonal Antibodies, in Participants With Advanced Solid Tumors

This is a phase 1 study of FT836 administered in participants with advanced solid tumors. The primary objectives of the study are to evaluate the safety and tolerability of FT836 with or without pacli...

Philadelphia, PAPHASE1113 participantsFate Therapeutics

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NCT05502523Recruiting

The Impact of Surgical Technique on Circulating Tumor DNA in Stage I-III Non-Small Cell Lung Cancer

This clinical trial compares the effect of pulmonary vein-first surgical technique to pulmonary artery-first surgical technique in decreasing circulating tumor cell deoxyribonucleic acid (ctDNA) in pa...

Philadelphia, PANA100 participantsThomas Jefferson University

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NCT06731907Recruiting

A Study of Pembrolizumab With or Without Chemotherapy in Combination With Additional Treatments for Advanced Non-Small Cell Lung Cancer (NSCLC) (MK-3475-01G/KEYMAKER U01)

Researchers are investigating new treatments for untreated advanced non-small cell lung cancer (NSCLC), which is the most common form of lung cancer and lung cancer that has spread beyond surgical rem...

Philadelphia, PAPHASE290 participantsMerck Sharp & Dohme LLC

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NCT06630806Recruiting

A Study to Investigate the Safety and Efficacy of SAR446523 Injected Subcutaneously in Adult Participants With Relapsed/Refractory Myeloma

This is a first-in-human study of SAR446523 conducted in patients with RRMM. The study consists of two parts: Dose escalation (Part A): In this part, up to several dose levels (DLs) of SAR446523 wil...

Philadelphia, PAPHASE187 participantsSanofi

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NCT07057791Recruiting

Phase II Study of Platinum/Etoposide Plus Ivonescimab for Extensive-Stage Small Cell Lung Cancer

Eligible untreated participants with Extensive Stage Small Cell Lung Cancer (ES-SCLC) who are ≥ 18 years of age will be randomized to receive ivonescimab 10 milligrams per kilogram (mg/kg) or ivonesci...

Philadelphia, PAPHASE260 participantsPrECOG, LLC.

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NCT04332367Recruiting

Carboplatin, Taxane And Ramucirumab for Patients With NSCLC After Pemetrexed or Pembrolizumab Maintenance

The purpose of this study is to determine if the combination of the three anti-cancer drugs carboplatin, paclitaxel, and ramucirumab is helpful in shrinking tumors or delaying tumor growth in particip...

Philadelphia, PAPHASE259 participantsAbramson Cancer Center at Penn Medicine

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NCT07047144Recruiting

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who...

Philadelphia, PAPHASE252 participantsScholar Rock, Inc.

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NCT07276789Recruiting

Efficacy and Safety of a Single Dose of LS301-IT for Fluorescence Intraoperative Molecular Imaging (IMI) for Patients Undergoing Lung Cancer Resection for Non Small Cell Lung Cancer

The aim of this Phase 2 study is to investigate the efficacy and safety of a single dose of LS301-IT, a novel fluorescence imaging agent developed by Integro Theranostics (IT), administered by intrave...

Philadelphia, PAPHASE235 participantsIntegro Theranostics

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NCT05861986Recruiting

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular...

Philadelphia, PAPHASE428 participantsHoffmann-La Roche

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NCT05861999Recruiting

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously rec...

Philadelphia, PAPHASE428 participantsHoffmann-La Roche

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NCT04691817Recruiting

Tocilizumab and Atezolizumab in Adults With Locally Advanced or Metastatic Non-Small Cell Lung Cancer Refractory to 1st Line Immune Checkpoint Inhibitor-Based Therapy

This is a research study to evaluate the safety and effectiveness of tocilizumab in combination with atezolizumab to treat non-small cell lung cancer....

Philadelphia, PAPHASE128 participantsAbramson Cancer Center at Penn Medicine

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NCT04139304Recruiting

A Study of Daratumumab and Dose-Adjusted EPOCH in Plasmablastic Lymphoma

This feasibility trial studies how well daratumumab in combination with dose-adjusted etoposide, prednisone, vincristine sulfate, cyclophosphamide, and doxorubicin hydrochloride (DA-EPOCH) works in tr...

Philadelphia, PAEARLY_PHASE115 participantsAIDS Malignancy Consortium

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NCT06934967Recruiting

Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated w...

Philadelphia, PAPHASE312 participantsNovartis Pharmaceuticals

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NCT07116057Recruiting

MOv19-BBz CAR T Cells in FRa+ Cancers

This is a Phase I open-label clinical trial to assess the safety, feasibility, and preliminary efficacy of intrapleural administration of MOv19-BBz CAR T cells in patients with FRa+ cancers. This stud...

Philadelphia, PAPHASE110 participantsUniversity of Pennsylvania

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NCT03546829Recruiting

Safety Trial of Antimicrobial Therapy and Precision Radiation Therapy in Patients With Oligoprogressive Non-small Cell Lung Cancer

The first phase of this study was an open label, randomized pilot study. Enrollment for this phase is now complete. The second phase is a single arm phase 1 safety study. Patients planned to undergo ...

Philadelphia, PAPHASE110 participantsAbramson Cancer Center at Penn Medicine

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About Spinal Muscular Atrophy Clinical Trials in Philadelphia

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder that causes loss of motor neurons, leading to progressive muscle weakness and atrophy. It is the leading genetic cause of infant death. Revolutionary gene therapies and SMN-enhancing drugs have transformed treatment.

There are currently 21 spinal muscular atrophy clinical trials recruiting participants in Philadelphia, PA. These studies are seeking a combined 2,151 participants. Research is being sponsored by Amgen, Antares Therapeutics, Inc, IDEAYA Biosciences and 15 other organizations. Clinical trial participation is free and participants receive study-related medical care at no cost.

Spinal Muscular Atrophy Clinical Trials in Philadelphia — FAQ

Are there spinal muscular atrophy clinical trials in Philadelphia?

Yes, there are 21 spinal muscular atrophy clinical trials currently recruiting in Philadelphia, PA. Browse the studies on this page to find one that fits.

How do I join a clinical trial in Philadelphia?

Browse the studies listed above, click one that interests you, and complete the free eligibility form. A study coordinator at the Philadelphia research site will contact you about next steps.

Are clinical trials in Philadelphia free?

Yes, clinical trial participation is always free. Study-related treatment, medical tests, and doctor visits are provided at no cost. Many Philadelphia studies also compensate for your time and travel.

What spinal muscular atrophy treatments are being tested?

The 21 active trials in Philadelphia are testing new therapies including novel drugs, biologics, and treatment approaches for spinal muscular atrophy.

Data updated August 27, 2026 from ClinicalTrials.gov