Gene Mutations Clinical Trials

6 recruitingLast updated: May 13, 2026

There are 6 actively recruiting gene mutations clinical trials across 8 countries. Studies span Phase 1, Phase 2, Phase 3. Top locations include Atlanta, Georgia, United States, Boston, Massachusetts, United States, Chapel Hill, North Carolina, United States. Updated daily from ClinicalTrials.gov.


Gene Mutations Trials at a Glance

6 actively recruiting trials for gene mutations are listed on ClinicalTrialsFinder across 6 cities in 8 countries. The largest study group is Phase 1 with 2 trials, with the heaviest enrollment activity in Atlanta, Boston, and Chapel Hill. Lead sponsors running gene mutations studies include Inozyme Pharma, Lomond Therapeutics Holdings, Inc., and M.D. Anderson Cancer Center.

Browse gene mutations trials by phase

Treatments under study

About Gene Mutations Clinical Trials

Looking for clinical trials for Gene Mutations? There are currently 1 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Gene Mutations trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Gene Mutations clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 16 of 6 trials

Recruiting
Phase 1

Revumenib in Combination With 7+3 + Midostaurin in AML

Acute Myeloid LeukemiaAMLLeukemia+2 more
Richard Stone, MD22 enrolled2 locationsNCT06313437
Recruiting
Phase 1

Dose Escalation and Expansion Study to Evaluate the Safety, PK, PD and Efficacy of ZE46-0134 in Adults With FLT3 Mutated or Spliceosome Mutated Relapsed or Refractory Acute Myeloid Leukemia

AML With Gene Mutations
Lomond Therapeutics Holdings, Inc.150 enrolled23 locationsNCT06366789
Recruiting
Phase 2

CPX-351 and Ivosidenib for the Treatment of IDH1 Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Myelodysplastic SyndromeMyeloproliferative NeoplasmRecurrent Acute Myeloid Leukemia+2 more
M.D. Anderson Cancer Center30 enrolled1 locationNCT04493164
Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Multiple Endocrine Neoplasia Type 1Multiple Endocrine Neoplasia Type 2Rare Disorders+337 more
Sanford Health20,000 enrolled2 locationsNCT01793168
Recruiting
Phase 2

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Arterial calcificationPseudoxanthoma ElasticumEctonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency+2 more
Inozyme Pharma200 enrolled5 locationsNCT06462547
Recruiting
Phase 3

Evaluating the Effects of Tasimelteon Vs. Placebo in Delayed Sleep-Wake Phase Disorder (DSWPD) and the CRY1Δ11 Variant

Sleep Wake DisordersSleep Disorders, Circadian RhythmChronobiology Disorders+1 more
Vanda Pharmaceuticals60 enrolled1 locationNCT06701396