Phase 2 Gaucher Disease Trial, Recruiting NCT04637503 Sponsor: Shenzhen Geno-Immune Medical Institute Condition: Gaucher Disease
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Phase 2 Gaucher Disease Trial, Recruiting

NCT04637503
Recruiting Phase 2

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • Your tumor has already received the standard first-line treatment but cannot be surgically removed, has spread to other parts of the body, has continued to grow, or has come back.
  • Your tumor must test positive for certain proteins called GD2, PSMA, and CD276, as confirmed by laboratory testing of tumor tissue or cells.
  • You weigh at least 10 kg (approximately 22 pounds).
  • You are between 1 and 65 years old at the time of joining the trial.
  • You are expected to live for at least 8 weeks.
  • Any serious side effects from previous treatments have mostly resolved or improved to a mild level.
  • It has been at least 1 week since you last received any blood cell growth factor injections.
  • It has been at least 7 days since finishing certain targeted or low-dose treatments.
  • It has been at least 4 weeks since receiving a treatment involving a monoclonal antibody (a specific type of cancer drug).
  • It has been at least 1 week since your last radiation therapy.
  • You are still able to carry out a reasonable amount of daily activity (as measured by a standard health scoring tool).
  • Your heart is pumping blood adequately, as shown by a heart function test.
  • Your blood oxygen level is at least 90% while breathing normal room air.
  • Your liver and kidney test results are within an acceptable range.
  • Your blood cell counts (white cells, infection-fighting cells, and platelets) meet the minimum levels required.
  • If your cancer has spread to the bone marrow, you may still be eligible as long as your blood counts meet the required levels.
  • A parent or legal guardian must sign a consent form on behalf of any patient who requires one.

Who may not be able to join:

  • You have another serious illness or major organ problems (for example, significant heart, lung, or liver disease), beyond certain blood-related side effects.
  • You have cancer that has spread to the brain or spinal cord and has not yet been treated, or has been treated but has not been stable for at least 6 weeks.
  • You have previously been treated with a genetically engineered cell therapy (CAR T-cell therapy) targeting GD2, PSMA, or CD276.
  • You currently have an active HIV, Hepatitis B, or Hepatitis C infection, or any other infection that is not under control.
  • You need to take steroid medications or other drugs that suppress your immune system on a regular basis.
  • There is a sign that the tumor may be blocking or could block your airway.
  • You are unable to follow the requirements of the trial.
  • There are not enough of the specially prepared cells available for your treatment (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Phone: +86 0755-86573763

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 2
Sponsor
Shenzhen Geno-Immune Medical Institute
Registry
ClinicalTrials.gov
Start date
1 June 2026
Est. completion
31 December 2029

Where this trial is recruiting

🇨🇳 China

Primary endpoints

Number of patients with adverse events

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov