A Phase 2 interventional study of Valproic Acid and Placebo in Retinitis Pigmentosa, sponsored by Foundation Fighting Blindness. Completed at 6 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2017-12-02.
Sponsored by Foundation Fighting Blindness · Phase 2, Interventional, and Treatment
The objectives of this study are to evaluate the efficacy of Valproic Acid (VPA) to both slow the progression of visual function loss and/or to restore visual function in patients with Autosomal Dominant Retinitis Pigmentosa (RP) and to collect safety and tolerability information.
Retinitis Pigmentosa (RP) is an incurable and untreatable group of heterogeneous retinal degenerative diseases that cause severe visual loss. There is currently no therapeutic that substantially slows the progression of this disease, and certainly none that can restore vision in RP patients. The Valproic Acid (VPA) study is designed as a six-site, interventional, prospective, randomized, placebo controlled, double-blinded study of 90 participants to evaluate the efficacy of oral Valproic Acid to both slow the progression of visual function loss and/or to restore visual function in patients with an Autosomal Dominant RP genetic mutation and to collect safety and tolerability information. Patients that participate in the study will be randomized to either placebo or VPA in a 1:1 ratio.
278 studies on the registry are indexed under Retinitis Pigmentosa; 72 are open to participants now.
This study's enrollment of 90 is above the median of 27 across 179 interventional studies indexed under Retinitis Pigmentosa.
Browse Retinitis Pigmentosa studies →Foundation Fighting Blindness is the lead sponsor of 3 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects who receive valproic acid
Drug: Valproic Acid
Subjects who receive placebo
Drug: Placebo
One to four 250mg softgels by mouth daily (dose determined by body weight)
Also known as: Valproate
Dosage per subject weight- same schedule as the active comparator
Mean Change in Visual Field Area From Baseline to 52 Weeks--III4e Isopter
Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the III4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
Time frame: baseline to week 52
Mean Change in Visual Field Area From Baseline to 52 Weeks--I4e Isopter
Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the I4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
Time frame: baseline to week 52
Static Perimetry by Treatment Arm--Full Field Hill of Vision
Mean change from baseline at week 52 for Full field Hill of Vision (Static perimetry)
Time frame: baseline to week 52
Static Perimetry Volume--30 Degree Hill of Vision
Mean Change from baseline to week 52 for Static Perimetry Volume --30 Degree Hill of Vision. Full field static perimetry protocol was followed using the Octopus 900 (Haag-Streit) for a single session for each eye.
Time frame: baseline to week 52
Mean Change From Baseline in Best Corrected Visual Acuity
Mean change in best corrected visual acuity as assessed by ETDRS (Early Treatment Diabetic Retinopathy Study) method from baseline to week 52
Time frame: baseline to week 52
| Milestone | Valproic Acid | Placebo |
|---|---|---|
| Started | 46 | 44 |
| Completed | 37 | 42 |
| Not completed | 9 | 2 |
| Withdrew: Death | 1 | 0 |
| Withdrew: Lost to follow-up | 3 | 1 |
| Withdrew: Adverse event | 2 | 1 |
| Withdrew: Withdrawal by subject | 3 | 0 |
Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the III4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
| Visual field area (degrees squared) | Placebo--Right Eye | Placebo--Left Eye | Valproic Acid--Right Eye | Valproic Acid--Left Eye |
|---|---|---|---|---|
| Mean Change in Visual Field Area From Baseline to 52 Weeks--III4e Isopter | -122.9 ± 543.60 | -112.0 ± 584.63 | -293.7 ± 736.56 | -237.1 ± 691.76 |
Mean change in visual field area from baseline to 52 weeks. Visual field area is measured with semi-automated kinetic perimetry (SKP) using the Octopus 900 (Haag-Streit) with the I4e target size for each eye and done at least twice to ensure reliable sessions; the visual field area measurements are averaged over the two sessions. Analysis performed with linear mixed model
| Visual field area (degrees squared) | Placebo--Right Eye | Placebo--Left Eye | Valproic Acid--Right Eye | Valproic Acid--Left Eye |
|---|---|---|---|---|
| Mean Change in Visual Field Area From Baseline to 52 Weeks--I4e Isopter | 80.9 ± 406.2 | 115.7 ± 696.89 | 5.3 ± 759.46 | 19.5 ± 703.83 |
Mean change from baseline at week 52 for Full field Hill of Vision (Static perimetry)
| db-steridians | Placebo--Right Eye | Placebo--Left Eye | Valproic Acid--Right Eye | Valproic Acid--Left Eye |
|---|---|---|---|---|
| Static Perimetry by Treatment Arm--Full Field Hill of Vision | -0.3 ± 2.55 | -1.4 ± 3.27 | -0.2 ± 5.11 | -0.6 ± 4.68 |
Mean Change from baseline to week 52 for Static Perimetry Volume --30 Degree Hill of Vision. Full field static perimetry protocol was followed using the Octopus 900 (Haag-Streit) for a single session for each eye.
| db-steridans | Placebo--Right Eye | Placebo--Left Eye | Valproic Acid--Right Eye | Valproic Acid--Left Eye |
|---|---|---|---|---|
| Static Perimetry Volume--30 Degree Hill of Vision | -0.3 ± 0.61 | -0.3 ± 0.73 | -0.2 ± 1.07 | -0.2 ± 0.96 |
Mean change in best corrected visual acuity as assessed by ETDRS (Early Treatment Diabetic Retinopathy Study) method from baseline to week 52
| letters read correctly | Valproic Acid -- Right Eye | Valproic Acid--Left Eye | Placebo --Right Eye | Placebo --Left Eye |
|---|---|---|---|---|
| Mean Change From Baseline in Best Corrected Visual Acuity | -1.4 ± 5.21 | 0.0 ± 3.41 | 0.2 ± 4.41 | 1.3 ± 5.05 |
Collected over Treatment emergent adverse events are defined as those that occur between the first dose of study drug and the last dose of study drug plus 7 days.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | 0/44 (0%) | 2/44 (4.5%) | 41/44 (93.2%) |
| Valproic Acid | 1/46 (2.2%) | 3/46 (6.5%) | 45/46 (97.8%) |
| Event | Placebo | Valproic Acid |
|---|---|---|
| Lens DislocationEye disorders | 1/44 | 0/46 |
| Atrial FibrilationCardiac disorders | 1/44 | 0/46 |
| OsteoarthritisMusculoskeletal and connective tissue disorders | 0/44 | 1/46 |
| Road Traffic AccidentsInjury, poisoning and procedural complications | 0/44 | 1/46 |
| Immunodeficiency common variableImmune system disorders | 0/44 | 1/46 |
| Event | Placebo | Valproic Acid |
|---|---|---|
| NasopharyngitisInfections and infestations | 9/44 | 3/46 |
| NauseaGastrointestinal disorders | 3/44 | 8/46 |
| DyspepsiaGastrointestinal disorders | 0/44 | 8/46 |
| HeadacheNervous system disorders | 4/44 | 8/46 |
| InfluenzaInfections and infestations | 7/44 | 1/46 |
| SinusitisInfections and infestations | 3/44 | 7/46 |
| Ammonia IncreasedInvestigations | 1/44 | 7/46 |
| FatigueGeneral disorders | 3/44 | 6/46 |
| Vision BlurredEye disorders | 5/44 | 3/46 |
| DiarrheaGastrointestinal disorders | 1/44 | 4/46 |
| Age, Continuous(years) | Placebo | Valproic Acid | Total |
|---|---|---|---|
| Mean | 51.6 ± 10.9 | 49.3 ± 12.3 | 50.4 ± 11.6 |
| Sex: Female, Male(Participants) | Placebo | Valproic Acid | Total |
|---|---|---|---|
| Female | 20 | 24 | 44 |
| Male | 24 | 22 | 46 |
| Ethnicity (NIH/OMB)(Participants) | Placebo | Valproic Acid | Total |
|---|---|---|---|
| Hispanic or Latino | 6 | 5 | 11 |
| Not Hispanic or Latino | 38 | 41 | 79 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Placebo | Valproic Acid | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 1 |
| White | 43 | 44 | 87 |
| More than one race | 0 | 1 | 1 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Genetic Mutations(Participants) | Placebo | Valproic Acid | Total |
|---|---|---|---|
| RHO | 22 | 19 | 41 |
| PRPF31 | 5 | 9 | 14 |
| RP1 | 4 | 9 | 13 |
| PRPF8 | 3 | 1 | 4 |
| PRPH2 | 2 | 2 | 4 |
| NR2E3 | 0 | 2 | 2 |
| RHO and PRPH2 | 2 | 0 | 2 |
| SNRNP200/ASCC3L1 | 2 | 0 | 2 |
| TOPORS | 0 | 2 | 2 |
| IMPDH1 | 1 | 0 | 1 |
| KLHL7 | 1 | 0 | 1 |
| NR2E3 and TOPORS | 0 | 1 | 1 |
| RHO and ROM1 | 0 | 1 | 1 |
The record gives a start of Nov 2010, a primary completion of Dec 2015 and a completion of Dec 2015. It does not say whether these dates are actual or estimated.
Results were first posted in Dec 2017, 2 years after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Oct 2017. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Valproic Acid
Foundation Fighting Blindness