Phase 2 Sickle Cell Disease Trial, Recruiting NCT04008368 Sponsor: National Heart, Lung, and Blood Institute (NHLBI) Condition: Sickle Cell Disease
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Phase 2 Sickle Cell Disease Trial, Recruiting

NCT04008368
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:

  • You previously had a stem cell transplant from a related donor to treat sickle cell disease (SCD) or beta thalassemia
  • Your sickle cell disease or beta thalassemia has come back after the transplant, causing symptoms such as pain crises, chest problems, dependence on blood transfusions, or other complications
  • Your body still shows some presence of the donor's cells (confirm with trial site)
  • You are 4 years old or older
  • You have a negative pregnancy test
  • Your heart is pumping with at least 35% efficiency (confirm with trial site)
  • Your lungs are functioning at 35% capacity or above — though this requirement may be skipped for very young children or those who cannot complete the breathing test

For the stem cell donor:

  • The donor must be the same related family member from your original transplant, willing to donate again, medically suitable, and between 2 and 79 years old

Who may not be able to join:

  • You have a low level of physical functioning — meaning you are largely unable to care for yourself or carry out daily activities
  • You have an active, uncontrolled infection (bacterial, viral, or fungal) that is getting worse despite treatment within the past month
  • You currently have a fever or signs of an infection (you may need to wait until you recover)
  • You have a serious illness or organ problem that would make surviving a stem cell transplant unlikely
  • You are currently pregnant or breastfeeding
  • You have a history of a second cancer (other than certain minor skin cancers)
  • Your immune system has developed specific antibodies against the donor's tissue type at a level that could cause problems (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 23 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

Principal Investigator: Courtney F Joseph, M.D., National Heart, Lung, and Blood Institute (NHLBI)

Phone: (301) 402-3595

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
Registry
ClinicalTrials.gov
Start date
24 October 2019
Est. completion
30 January 2030

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection

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: 23 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