Clinical research and clinical trials

JAMA Internal Medicine has declined to retract a 2000 oxycodone-for-osteoarthritis trial despite a co-author briefly backing the request and litigation showing sponsor Purdue Frederick knew of unreported withdrawal symptoms. The journal’s reasoning turns on a narrow standard — fabrication or falsification — that says little about sponsor-level data omission, a gap research-integrity offices should understand.

BMJ Public Health retracted Saskia Mostert’s COVID-19 vaccine mortality paper on Aug. 11, 2026, roughly two years after first flagging it — after the paper was cited in a June 2026 Senate hearing while still under an unresolved expression of concern.

Intellia’s one-time CRISPR therapy lonvo-z met its primary endpoint in the Phase 3 HAELO trial, cutting hereditary angioedema attacks by 87% versus placebo — a still-investigational result now heading into FDA review.

A gene-burden analysis of 2,811 patients with ataxia, spastic paraplegia, and dystonia identified CD99L2 — previously known only as an immune protein — as a cause of X-linked spastic ataxia, closing a long-standing diagnostic gap.

A ten-year clinical trial led by the University of Pittsburgh and University of Chicago found that intracortical microstimulation can safely and durably restore touch sensation in people with spinal cord injury — the longest human study of a brain-computer interface for sensory feedback to date, jointly funded by the NIH BRAIN Initiative and DARPA.

The FDA has approved datopotamab deruxtecan (Datroway) for unresectable or metastatic triple-negative breast cancer, based on the MSK-led TROPION-Breast02 trial, which showed a roughly six-month overall survival benefit over chemotherapy.

MD Anderson has opened a Phase 1 trial of JV-394, a CAR-T therapy targeting CD94, in patients with relapsed or refractory T-cell and NK-cell lymphomas, a cancer type that has long resisted cell-based immunotherapy.

The FDA has approved atezolizumab (Tecentriq) as adjuvant treatment for muscle-invasive bladder cancer, but only for patients whose blood tests ctDNA-positive after surgery — the first approval to use a blood-based biomarker to gate eligibility for a cancer drug.

Researchers at HUN-REN Biological Research Centre, Szeged (Hungary) have identified surface adhesion proteins that act as ‘molecular anchors,’ letting certain gut bacteriophages bind human intestinal cells without infecting them. The finding, published in Nature Communications, points toward more precisely targeted phage therapies and new intracellular delivery strategies.

Baylor College of Medicine researchers recorded individual hippocampal neurons in epilepsy-surgery patients under general anesthesia and found the brain still distinguishes nouns, verbs, and adjectives, and even predicts upcoming words — evidence of structured language processing without consciousness. Because the data can only be collected while the patient is unconscious, all consent has to be obtained pre-operatively, a genuinely distinct human-subjects-ethics scenario from research where a participant can object in real time.

A two-year Binghamton University study tracking 242 children’s eye movements toward emotional faces found a measurable attention marker linked to depression risk — one that shifts differently depending on whether a child’s mother has a history of major depressive disorder. Because the study collects family mental-health-history data on minors and assesses inherited depression risk, it required heightened IRB review and parental consent, a genuine human-subjects-ethics angle.

A small University of Bristol-led pilot trial found the anti-inflammatory drug tocilizumab produced 54% remission in treatment-resistant depression versus 31% on placebo (NNT=5) over four weeks. The 30-person trial, funded by Wellcome, NIHR, and the BMA Foundation, is explicitly framed by its own authors as proof-of-concept requiring a larger confirmatory trial — and repurposes an approved rheumatoid-arthritis biologic off-label for a psychiatric indication, a genuine human-subjects-ethics angle.

In a Charité-led, double-blind, placebo-controlled trial of 170+ PTSD patients, the THC medication Dronabinol cut nightmare severity nearly twice as much as placebo over ten weeks, and more than a third of treated patients stopped having nightmares entirely. The trial was funded by Bionorica SE, a pharmaceutical manufacturer with a commercial stake in THC-based products — a genuine conflict-of-interest disclosure story — and its at-home, self-administered dosing design in a trauma-exposed population raises real research-ethics protocol questions.

A Stanford-led Nature study finds that blood stem cells send immune cell reinforcements into the aging human brain, forming microglia-like cells in a process traced through shared DNA mutations — a phenomenon not seen in mice or nonhuman primates, and one that upends the long-held idea of the brain as immune-privileged.

A Bundibugyo ebolavirus outbreak (PHEIC since 17 May 2026) has no disease-specific licensed vaccine. WHO is deploying the Zaire-strain Ervebo against it via a Phase 3 trial on cross-protection evidence, skipping Phase 2 — a trial-only, not compassionate-use, response.

FDA granted accelerated approval to Tudriqev (RP1) plus nivolumab for advanced melanoma on Aug 6, 2026 — after two Complete Response Letters on the same IGNYTE single-arm trial. Here is what changed in the evidence review, and what the required confirmatory trial still has to show.

STAT News reported on August 5, 2026 that a child enrolled in a HuidaGene Therapeutics CRISPR trial for Duchenne muscular dystrophy died of ARDS during a severe immune reaction roughly a year before the death was disclosed — a second undisclosed pediatric death in a Chinese gene-editing trial to surface within weeks, this one run under China’s investigator-initiated trial pathway with no independent safety monitoring board.

A 1,154-patient IMPACC cohort study found anellovirus reactivation, not EBV, tracks long-term disability in long COVID — and revises what EBV reactivation actually signals.

The 2026 domestic cyclosporiasis outbreak is CDC’s largest on record. The specialist angle: why routine ova-and-parasite exams miss Cyclospora cayetanensis, and the modified acid-fast staining, UV autofluorescence, and PCR methods that actually confirm it.

On August 5, 2026 the FDA approved Takeda’s oveporexton (Orzeyful), the first orexin receptor agonist for narcolepsy type 1, based on two Phase 3 trials (FirstLight, RadiantLight). DEA scheduling review and commercial availability are still pending.

China banned an unproven Alzheimer’s neck surgery and detained its inventor in 2025. Registered trials of the same procedure continue abroad.

Shanghai Jiao Tong University has opened a formal investigation, and Nature has posted an editor’s note, after Science and Retraction Watch reported that a 6-year-old girl died days after an experimental base-editing gene therapy overseen by neuroscientist Zilong Qiu — a death, along with the family’s roughly $860,000 contribution to the therapy’s development, that was not disclosed in the resulting Nature paper.

FDA’s July 2026 Unified Agenda lists a dozen new proposed rules on clinical trials, Phase 1 IND, DTC ads, and CRL disclosure beyond the IND pilot.

HHS OIG is asking whether the Anti-Kickback Statute needs a new safe harbor for paying clinical trial participants. Comments on the RFI are due August 24, 2026.

FDA shipped Elsa 4.0 and the HALO data platform in May 2026 — an internal AI upgrade, not a sponsor-facing one. Here is what changed and what did not.

EMA’s draft guidance on running clinical trials during public health emergencies closed for comment April 30, 2026. Finalisation is still pending.

FDA’s April 2026 RFI sought input on an AI-enabled early-phase trials pilot. Comment window closed June 29, 2026 — here’s what it covered.

NIH plans to widen All of Us pediatric enrollment to ages 0-12 in 2026, even as the program absorbs a steep funding drop from FY2023 levels.

FDA eased CMC expectations for cell and gene therapy BLAs on Jan 11, 2026 — finalized in May, extended to genome editing in June.

The EU’s proposed Biotech Act (Commission proposal adopted 16 December 2025, not yet enacted) would revise Clinical Trials Regulation Article 93 to align it with GDPR, naming sponsors and investigators as data controllers and permitting cross-trial data reuse — here is what is actually proposed, what the EDPB-EDPS flagged as unresolved, and what remains to be decided.

An FDA warning letter dated July 10, 2026, cites a bioequivalence-trial investigator tied to an Alembic Pharmaceuticals facility for obtaining informed consent after study procedures had already begun. Here is what the letter actually says, and what unverified secondary claims about it do not hold up.

FDA’s Dec. 2025 guidance pushes sponsors to justify restrictive eligibility criteria, distinct from and broader than the FDORA diversity action plan mandate.

ARPA-H’s Advancing Clinical Trial Readiness (ACTR) initiative aims to put a clinical trial site within 30 minutes of 90% of eligible Americans. Here’s what the program covers, its three funding focus areas, and where it stands.

FDA’s Commissioner’s National Priority Voucher (CNPV) pilot promises a one-to-two-month review for drugs meeting national health priorities. Here’s how it differs from statutory Priority Review Vouchers, who has received one, and where the program stands as of FDA’s 2026 public hearing.

Health Canada’s draft Clinical Trials Regulations and its April 2026 ICH E6(R3) adoption mark Canada’s biggest trial-oversight overhaul in decades.

FDA’s June 22, 2026 revised draft guidance clarifies when a single adequate and well-controlled trial plus confirmatory evidence can satisfy the substantial-evidence-of-effectiveness standard for drug and biologic approval, narrowing the traditional two-trial default. Comments are open through September 22, 2026.

FDA published draft guidance Jan. 12, 2026 setting expectations for Bayesian primary-analysis designs in drug/biologic trials. Comments closed March 13, 2026.

CMS and FDA launched RAPID on April 23, 2026, targeting Medicare coverage within 60-90 days of FDA authorization for breakthrough devices, pausing TCET for new candidates.

FDA’s final guidance sets trial-design rules for psychedelic drug studies, addressing functional unblinding and abuse-potential testing.

On July 14, 2026, FDA finalized guidances on generic topical corticosteroid bioequivalence testing and formal FDA meetings for OTC monograph drug sponsors.

CMS’s CY2027 fee schedule proposal narrows, not bans, third-party RPM/RTM billing. Here is the real mechanism, the OIG data behind it, and what it means for clinical trial billing compliance.

AHRQ has issued near-identical “non-award” letters ending continuation funding for dozens of active grants, citing portfolio prioritization. HHS says these are not terminations; grantees and advocacy groups disagree, a distinction with real consequences for appeal rights and sponsored-programs planning.

FDA reopened comment on its master protocol draft guidance in June 2026, adding new basket-trial recommendations. Comments are open through August 24, 2026.

FDA’s proposed Expedited IND Pilot Program would speed first-in-human trials via Qualified Research Institutions; comments now due Aug. 24, 2026.

FDA has initiated two proof-of-concept real-time clinical trials and opened an RFI on a broader RTCT pilot program. Final selection criteria are expected in July 2026, with pilot selections completed in August 2026.

ClinicalTrials.gov is retiring the Classic Protocol Registration and Results System (PRS) in stages through 2026 and 2027, moving every sponsor and registrant onto the Modernized PRS. Here is the confirmed timeline, from the July 2026 cutoff on new records to the Fall 2026 submission deadline, and what to do before each one.