Phase 2 Sickle Cell Disease Trial, Recruiting (NCT04774536)

Phase 2 Sickle Cell Disease Trial, Recruiting NCT04774536 Sponsor: Mark Walters, MD Condition: Sickle Cell Disease

Printed from Voxsanity (voxsanity.com.au) — NCT04774536 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Sickle Cell Disease

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 are between 12 and 34 years old (at the time of signing consent)
You have sickle cell disease of the HbSS type (the most common form) and have experienced one or more of the following serious complications:
- Two or more episodes of a lung complication called "acute chest syndrome" in the past 2 years, even while receiving treatment such as asthma therapy or hydroxyurea
- Four or more severe pain crises in the past 2 years, even while on a pain management plan or hydroxyurea, where each crisis required strong painkiller treatment through a drip or injection for more than 24 hours in hospital, or at least 2 emergency visits within 72 hours both needing injected painkillers
- Two or more episodes where the spleen suddenly trapped a large amount of blood, causing it to enlarge and your blood count to drop significantly, in the past 2 years
- Repeated episodes of a painful erection lasting at least 4 hours, occurring at least twice in the last 12 months or 3 times in the last 24 months, that has not responded to medical treatment
- At least one episode where the liver suddenly trapped blood, causing liver pain, enlargement, and a rapid drop in blood count, in the past 2 years
- A leg ulcer that has not healed despite treatment, within the past 2 years
Your heart is working well enough, with the heart's pumping function above a certain level as measured by a heart scan (confirm with trial site)
Your lungs are working well enough, with blood oxygen levels of at least 85% and lung capacity above a certain level on breathing tests (confirm with trial site)
Your kidneys are working well enough, based on blood and urine tests (confirm with trial site)
Your liver is working well enough, based on blood tests; if liver iron levels are high on an MRI scan, additional tests will be done to check for serious scarring, and you may still be eligible depending on results (confirm with trial site)
Your blood clotting levels are within an acceptable range, unless you are already taking a blood-thinning medication for prevention (confirm with trial site)
You are able and willing to give written consent (or a legal guardian can do so on your behalf) and are able to follow the study requirements

Who may not be able to join:

You have an uncontrolled bacterial, viral, or fungal infection in the 6 weeks before joining the trial that is getting worse despite treatment
You have HIV, test positive for HIV antibodies, or have active hepatitis B or C
You have previously had a stem cell or bone marrow transplant
You have previously had a solid organ transplant (such as a kidney or liver transplant)
You have taken part in another clinical trial involving an experimental or unapproved drug or device within the 3 months before joining
You are pregnant or currently breastfeeding
You are a female who could become pregnant and are not willing or able to use two effective methods of contraception, or practice complete abstinence, from the time of consent until 12 months after the stem cell treatment (confirm with trial site)
You are male and are not willing or able to use effective barrier contraception (such as condoms) or practice complete abstinence from the time of consent until 12 months after the stem cell treatment
You have previously had a stroke, or you are currently receiving regular blood transfusions to prevent a first stroke or silent brain injury
You have a brother or sister whose tissue type (HLA) is a full match and who is willing and able to donate bone marrow to you
You have a condition or health issue that the study doctor believes could put you at risk or affect the quality of the study results (confirm with trial site)
Your sickle cell disease is not the HbSS type (the most common form)
A bone marrow test shows signs of a blood cell disorder called myelodysplastic syndrome (MDS), or certain gene changes linked to blood cancers (confirm with trial site)
Genetic testing has found a harmful mutation linked to blood or bone marrow cancers, or a gene change that experts on the study panel believe may be harmful (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

Principal Investigator: Mark Walters, MD, UCSF Benioff Children's Hospital Oakland

Phone: (510) 428-3374

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.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT04774536

Sponsor: Mark Walters, MD

Phase: Phase 2

Status: Recruiting

Condition: Sickle Cell Disease

Trial contact: (510) 428-3374

More information: https://clinicaltrials.gov/study/NCT04774536

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

You are between 12 and 34 years old (at the time of signing consent)
You have sickle cell disease of the HbSS type (the most common form) and have experienced one or more of the following serious complications:
- Two or more episodes of a lung complication called "acute chest syndrome" in the past 2 years, even while receiving treatment such as asthma therapy or hydroxyurea
- Four or more severe pain crises in the past 2 years, even while on a pain management plan or hydroxyurea, where each crisis required strong painkiller treatment through a drip or injection for more than 24 hours in hospital, or at least 2 emergency visits within 72 hours both needing injected painkillers
- Two or more episodes where the spleen suddenly trapped a large amount of blood, causing it to enlarge and your blood count to drop significantly, in the past 2 years
- Repeated episodes of a painful erection lasting at least 4 hours, occurring at least twice in the last 12 months or 3 times in the last 24 months, that has not responded to medical treatment
- At least one episode where the liver suddenly trapped blood, causing liver pain, enlargement, and a rapid drop in blood count, in the past 2 years
- A leg ulcer that has not healed despite treatment, within the past 2 years
Your heart is working well enough, with the heart's pumping function above a certain level as measured by a heart scan (confirm with trial site)
Your lungs are working well enough, with blood oxygen levels of at least 85% and lung capacity above a certain level on breathing tests (confirm with trial site)
Your kidneys are working well enough, based on blood and urine tests (confirm with trial site)
Your liver is working well enough, based on blood tests; if liver iron levels are high on an MRI scan, additional tests will be done to check for serious scarring, and you may still be eligible depending on results (confirm with trial site)
Your blood clotting levels are within an acceptable range, unless you are already taking a blood-thinning medication for prevention (confirm with trial site)
You are able and willing to give written consent (or a legal guardian can do so on your behalf) and are able to follow the study requirements

Who may not be able to join:

You have an uncontrolled bacterial, viral, or fungal infection in the 6 weeks before joining the trial that is getting worse despite treatment
You have HIV, test positive for HIV antibodies, or have active hepatitis B or C
You have previously had a stem cell or bone marrow transplant
You have previously had a solid organ transplant (such as a kidney or liver transplant)
You have taken part in another clinical trial involving an experimental or unapproved drug or device within the 3 months before joining
You are pregnant or currently breastfeeding
You are a female who could become pregnant and are not willing or able to use two effective methods of contraception, or practice complete abstinence, from the time of consent until 12 months after the stem cell treatment (confirm with trial site)
You are male and are not willing or able to use effective barrier contraception (such as condoms) or practice complete abstinence from the time of consent until 12 months after the stem cell treatment
You have previously had a stroke, or you are currently receiving regular blood transfusions to prevent a first stroke or silent brain injury
You have a brother or sister whose tissue type (HLA) is a full match and who is willing and able to donate bone marrow to you
You have a condition or health issue that the study doctor believes could put you at risk or affect the quality of the study results (confirm with trial site)
Your sickle cell disease is not the HbSS type (the most common form)
A bone marrow test shows signs of a blood cell disorder called myelodysplastic syndrome (MDS), or certain gene changes linked to blood cancers (confirm with trial site)
Genetic testing has found a harmful mutation linked to blood or bone marrow cancers, or a gene change that experts on the study panel believe may be harmful (confirm with trial site)

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

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Phase 2

Sponsor

Mark Walters, MD

Registry

ClinicalTrials.gov

Start date

22 August 2025

Est. completion

1 March 2030

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Incidence of adverse events and grade 3 or higher serious adverse events, using the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE)

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