Phase 2 Spinal Muscular Atrophy Trial, Recruiting (NCT07070999)

Phase 2 Spinal Muscular Atrophy Trial, Recruiting NCT07070999 Sponsor: Gemma Biotherapeutics Condition: Spinal Muscular Atrophy

Printed from Voxsanity (voxsanity.com.au) — NCT07070999 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Spinal Muscular Atrophy

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:

Children showing symptoms: Your child has been diagnosed with SMA Type 1 through genetic testing, showing changes in the SMN1 gene and having up to 3 copies of the SMN2 gene.
Children showing symptoms: Your child is between 2 weeks and 12 months old at the time of receiving the treatment, and symptoms began within the first 6 months of life.
Children not yet showing symptoms: Your child is considered at risk for SMA Type 1 based on genetic testing, showing changes in the SMN1 gene and having up to 2 copies of the SMN2 gene.
Children not yet showing symptoms: Your child is between 2 weeks and just under 5 months old (under 150 days) at the time of receiving the treatment.

Who may not be able to join:

Your child has a suspected or confirmed active viral infection, including HIV, Hepatitis B, Hepatitis C, or HTLV.
Your child has needed a breathing tube (tracheotomy with a breathing machine) or has had blood oxygen levels below 95%.
Your child is currently taking, or has taken within the last 3 months, medicines that suppress the immune system (such as steroids, cyclosporine, methotrexate, or similar drugs).
Your child has recently taken part in another SMA treatment trial that the trial doctor believes could make gene therapy unsafe for them.
Your child has previously received any gene therapy, a bone marrow transplant, or an organ transplant.
Your child has severe curvature of the spine (scoliosis).
Your child has a known allergy or bad reaction to prednisolone, steroid medicines, or their ingredients.

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: May Orfali, MD, Gemma Biotherapeutics

Phone: 267-718-7654

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: NCT07070999

Sponsor: Gemma Biotherapeutics

Phase: Phase 2

Status: Recruiting

Condition: Spinal Muscular Atrophy

Trial contact: 267-718-7654

More information: https://clinicaltrials.gov/study/NCT07070999

Eligibility summary (AI generated)

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

Who might be able to join this trial:

Children showing symptoms: Your child has been diagnosed with SMA Type 1 through genetic testing, showing changes in the SMN1 gene and having up to 3 copies of the SMN2 gene.
Children showing symptoms: Your child is between 2 weeks and 12 months old at the time of receiving the treatment, and symptoms began within the first 6 months of life.
Children not yet showing symptoms: Your child is considered at risk for SMA Type 1 based on genetic testing, showing changes in the SMN1 gene and having up to 2 copies of the SMN2 gene.
Children not yet showing symptoms: Your child is between 2 weeks and just under 5 months old (under 150 days) at the time of receiving the treatment.

Who may not be able to join:

Your child has a suspected or confirmed active viral infection, including HIV, Hepatitis B, Hepatitis C, or HTLV.
Your child has needed a breathing tube (tracheotomy with a breathing machine) or has had blood oxygen levels below 95%.
Your child is currently taking, or has taken within the last 3 months, medicines that suppress the immune system (such as steroids, cyclosporine, methotrexate, or similar drugs).
Your child has recently taken part in another SMA treatment trial that the trial doctor believes could make gene therapy unsafe for them.
Your child has previously received any gene therapy, a bone marrow transplant, or an organ transplant.
Your child has severe curvature of the spine (scoliosis).
Your child has a known allergy or bad reaction to prednisolone, steroid medicines, or their ingredients.

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

Gemma Biotherapeutics

Registry

ClinicalTrials.gov

Start date

6 January 2026

Est. completion

1 December 2028

Where this trial is recruiting

🇧🇷 Brazil

Primary endpoints

Number of participants with treatment-related adverse events (AEs) and serious adverse events (SAEs) at Grade 3 or higher as characterized by CTCAEv5.0; Number of Participants with Clinically Significant Changes in Physical Functions; Number of Participants with Clinically Significant Changes in Neurological Functions; Number of Participants with Clinically Significant Changes in Vital signs; Change in electrocardiogram results; Change in serum cardiac troponin I levels; Number of Participants with Clinically Significant Laboratory Abnormalities as Measured Using Hematology, Chemistry and Coag...

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